PaTH Forward: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism
Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism. This work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults...
Saved in:
Published in | The journal of clinical endocrinology and metabolism Vol. 107; no. 1; pp. e372 - e385 |
---|---|
Main Authors | , , , , , , , , , , , , , , , , , , |
Format | Journal Article |
Language | English |
Published |
United States
Oxford University Press
01.01.2022
|
Subjects | |
Online Access | Get full text |
Cover
Loading…
Abstract | Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism.
This work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism.
This phase 2, randomized, double-blind, placebo-controlled 4-week trial with open-label extension enrolled 59 individuals with hypoparathyroidism. Interventions included TransCon PTH 15, 18, or 21 µg PTH(1-34)/day or placebo for 4 weeks, followed by a 22-week extension during which TransCon PTH dose was titrated (6-60 µg PTH[1-34]/day).
By Week 26, 91% of participants treated with TransCon PTH achieved independence from standard of care (SoC, defined as active vitamin D = 0 μg/day and calcium [Ca] ≤ 500 mg/day). Mean 24-hour urine Ca (uCa) decreased from a baseline mean of 415 mg/24h to 178 mg/24h by Week 26 (n = 44) while normal serum Ca (sCa) was maintained and serum phosphate and serum calcium-phosphate product fell within the normal range. By Week 26, mean scores on the generic 36-Item Short Form Health Survey domains increased from below normal at baseline to within the normal range. The Hypoparathyroidism Patient Experience Scale symptom and impact scores improved through 26 weeks. TransCon PTH was well tolerated with no treatment-related serious or severe adverse events.
TransCon PTH enabled independence from oral active vitamin D and reduced Ca supplements (≤ 500 mg/day) for most participants, achieving normal sCa, serum phosphate, uCa, serum calcium-phosphate product, and demonstrating improved health-related quality of life. These results support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism. |
---|---|
AbstractList | Context: Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism. Objective: This work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism. Methods: This phase 2, randomized, double-blind, placebo-controlled 4-week trial with open-label extension enrolled 59 individuals with hypoparathyroidism. Interventions included TransCon PTH 15, 18, or 21 [micro]g PTH(1-34)/day or placebo for 4 weeks, followed by a 22-week extension during whichTransCon PTH dose was titrated (6-60 [micro]g PTH[1-34]/day). Results: ByWeek 26, 91% of participants treated withTransCon PTH achieved independence from standard of care (SoC, defined as active vitamin D = 0 [micro]g/day and calcium [Ca] [less than or equal to] 500 mg/day). Mean 24-hour urine Ca (uCa) decreased from a baseline mean of 415 mg/24h to 178 mg/24h by Week 26 (n = 44) while normal serum Ca (sCa) was maintained and serum phosphate and serum calcium-phosphate product fell within the normal range. By Week 26, mean scores on the generic 36-Item Short Form Health Survey domains increased from below normal at baseline to within the normal range. The Hypoparathyroidism Patient Experience Scale symptom and impact scores improved through 26 weeks. TransCon PTH was well tolerated with no treatment-related serious or severe adverse events. Conclusion: TransCon PTH enabled independence from oral active vitamin D and reduced Ca supplements ([less than or equal to] 500 mg/day) for most participants, achieving normal sCa, serum phosphate, uCa, serum calcium-phosphate product, and demonstrating improved health-related quality of life. These results support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism. Key Words: hypoparathyroidism, prodrug, parathyroid hormone, PTH(1-34), replacement therapy, TransCon PTH CONTEXTHypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism. OBJECTIVEThis work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism. METHODSThis phase 2, randomized, double-blind, placebo-controlled 4-week trial with open-label extension enrolled 59 individuals with hypoparathyroidism. Interventions included TransCon PTH 15, 18, or 21 µg PTH(1-34)/day or placebo for 4 weeks, followed by a 22-week extension during which TransCon PTH dose was titrated (6-60 µg PTH[1-34]/day). RESULTSBy Week 26, 91% of participants treated with TransCon PTH achieved independence from standard of care (SoC, defined as active vitamin D = 0 μg/day and calcium [Ca] ≤ 500 mg/day). Mean 24-hour urine Ca (uCa) decreased from a baseline mean of 415 mg/24h to 178 mg/24h by Week 26 (n = 44) while normal serum Ca (sCa) was maintained and serum phosphate and serum calcium-phosphate product fell within the normal range. By Week 26, mean scores on the generic 36-Item Short Form Health Survey domains increased from below normal at baseline to within the normal range. The Hypoparathyroidism Patient Experience Scale symptom and impact scores improved through 26 weeks. TransCon PTH was well tolerated with no treatment-related serious or severe adverse events. CONCLUSIONTransCon PTH enabled independence from oral active vitamin D and reduced Ca supplements (≤ 500 mg/day) for most participants, achieving normal sCa, serum phosphate, uCa, serum calcium-phosphate product, and demonstrating improved health-related quality of life. These results support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism. Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism. This work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism. This phase 2, randomized, double-blind, placebo-controlled 4-week trial with open-label extension enrolled 59 individuals with hypoparathyroidism. Interventions included TransCon PTH 15, 18, or 21 µg PTH(1-34)/day or placebo for 4 weeks, followed by a 22-week extension during which TransCon PTH dose was titrated (6-60 µg PTH[1-34]/day). By Week 26, 91% of participants treated with TransCon PTH achieved independence from standard of care (SoC, defined as active vitamin D = 0 μg/day and calcium [Ca] ≤ 500 mg/day). Mean 24-hour urine Ca (uCa) decreased from a baseline mean of 415 mg/24h to 178 mg/24h by Week 26 (n = 44) while normal serum Ca (sCa) was maintained and serum phosphate and serum calcium-phosphate product fell within the normal range. By Week 26, mean scores on the generic 36-Item Short Form Health Survey domains increased from below normal at baseline to within the normal range. The Hypoparathyroidism Patient Experience Scale symptom and impact scores improved through 26 weeks. TransCon PTH was well tolerated with no treatment-related serious or severe adverse events. TransCon PTH enabled independence from oral active vitamin D and reduced Ca supplements (≤ 500 mg/day) for most participants, achieving normal sCa, serum phosphate, uCa, serum calcium-phosphate product, and demonstrating improved health-related quality of life. These results support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism. Abstract Context Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism. Objective This work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism. Methods This phase 2, randomized, double-blind, placebo-controlled 4-week trial with open-label extension enrolled 59 individuals with hypoparathyroidism. Interventions included TransCon PTH 15, 18, or 21 µg PTH(1-34)/day or placebo for 4 weeks, followed by a 22-week extension during which TransCon PTH dose was titrated (6-60 µg PTH[1-34]/day). Results By Week 26, 91% of participants treated with TransCon PTH achieved independence from standard of care (SoC, defined as active vitamin D = 0 μg/day and calcium [Ca] ≤ 500 mg/day). Mean 24-hour urine Ca (uCa) decreased from a baseline mean of 415 mg/24h to 178 mg/24h by Week 26 (n = 44) while normal serum Ca (sCa) was maintained and serum phosphate and serum calcium-phosphate product fell within the normal range. By Week 26, mean scores on the generic 36-Item Short Form Health Survey domains increased from below normal at baseline to within the normal range. The Hypoparathyroidism Patient Experience Scale symptom and impact scores improved through 26 weeks. TransCon PTH was well tolerated with no treatment-related serious or severe adverse events. Conclusion TransCon PTH enabled independence from oral active vitamin D and reduced Ca supplements (≤ 500 mg/day) for most participants, achieving normal sCa, serum phosphate, uCa, serum calcium-phosphate product, and demonstrating improved health-related quality of life. These results support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism. |
Audience | Academic |
Author | Schwarz, Peter Smith, Alden Karpf, David B Khan, Aliya A Ahmed, Intekhab Rubin, Mishaela Pihl, Susanne Hofbauer, Lorenz Mourya, Sanchita Rejnmark, Lars Vokes, Tamara Brod, Meryl Palermo, Andrea Marcocci, Claudio Pagotto, Uberto Eriksen, Erik Clarke, Bart Shu, Aimee D Markova, Denka |
AuthorAffiliation | 14 Ascendis Pharma Inc , Palo Alto, California 94301 , USA 15 Ascendis Pharma A/S , 2900 Hellerup , Denmark 5 Section of Endocrinology, Diabetes and Metabolism, University of Chicago , Chicago, Illinois 60637 , USA 3 Metabolic Bone Disease Unit, Columbia University , New York, New York 10027 , USA 12 Oslo University Hospital, Institute of Clinical Medicine, 0372 Oslo, Norway 4 Department of Endocrinology, Rigshospitalet, Copenhagen and Faculty of Health Sciences, Copenhagen University , 2200 Copenhagen N , Denmark 2 Department of Endocrinology and Internal Medicine, Aarhus University Hospital , 8200 Aarhus N , Denmark 13 The Brod Group , Mill Valley, California 94941 , USA 10 Division of Endocrinology and Diabetes Prevention and Care, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Department of Medical and Surgical Sciences (DIMEC), Alma Mater Studiorum University of Bologna , 40126 Bologna , Italy 7 Thomas Jefferson University , Philadelphia, Pennsylvania 19107 , USA 8 Universitätsklinikum |
AuthorAffiliation_xml | – name: 3 Metabolic Bone Disease Unit, Columbia University , New York, New York 10027 , USA – name: 15 Ascendis Pharma A/S , 2900 Hellerup , Denmark – name: 12 Oslo University Hospital, Institute of Clinical Medicine, 0372 Oslo, Norway – name: 8 Universitätsklinikum Dresden , 01307 Dresden , Germany – name: 10 Division of Endocrinology and Diabetes Prevention and Care, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Department of Medical and Surgical Sciences (DIMEC), Alma Mater Studiorum University of Bologna , 40126 Bologna , Italy – name: 14 Ascendis Pharma Inc , Palo Alto, California 94301 , USA – name: 1 Department of Endocrinology and Metabolism and Geriatrics, McMaster University , Hamilton, Ontario L8S 4L8 , Canada – name: 9 University of Pisa , 56126 Pisa , Italy – name: 6 Division of Endocrinology, Diabetes, Metabolism, and Nutrition, Mayo Clinic , Rochester, Minnesota 55905 , USA – name: 4 Department of Endocrinology, Rigshospitalet, Copenhagen and Faculty of Health Sciences, Copenhagen University , 2200 Copenhagen N , Denmark – name: 7 Thomas Jefferson University , Philadelphia, Pennsylvania 19107 , USA – name: 11 Unit of Endocrinology and Diabetes, Campus Bio-Medico University , 00128 Rome , Italy – name: 5 Section of Endocrinology, Diabetes and Metabolism, University of Chicago , Chicago, Illinois 60637 , USA – name: 13 The Brod Group , Mill Valley, California 94941 , USA – name: 2 Department of Endocrinology and Internal Medicine, Aarhus University Hospital , 8200 Aarhus N , Denmark |
Author_xml | – sequence: 1 givenname: Aliya A surname: Khan fullname: Khan, Aliya A organization: Department of Endocrinology and Metabolism and Geriatrics, McMaster University, Hamilton, Ontario L8S 4L8, Canada – sequence: 2 givenname: Lars surname: Rejnmark fullname: Rejnmark, Lars organization: Department of Endocrinology and Internal Medicine, Aarhus University Hospital, 8200 Aarhus N, Denmark – sequence: 3 givenname: Mishaela surname: Rubin fullname: Rubin, Mishaela organization: Metabolic Bone Disease Unit, Columbia University, New York, New York 10027, USA – sequence: 4 givenname: Peter surname: Schwarz fullname: Schwarz, Peter organization: Department of Endocrinology, Rigshospitalet, Copenhagen and Faculty of Health Sciences, Copenhagen University, 2200 Copenhagen N, Denmark – sequence: 5 givenname: Tamara surname: Vokes fullname: Vokes, Tamara organization: Section of Endocrinology, Diabetes and Metabolism, University of Chicago, Chicago, Illinois 60637, USA – sequence: 6 givenname: Bart surname: Clarke fullname: Clarke, Bart organization: Division of Endocrinology, Diabetes, Metabolism, and Nutrition, Mayo Clinic, Rochester, Minnesota 55905, USA – sequence: 7 givenname: Intekhab surname: Ahmed fullname: Ahmed, Intekhab organization: Thomas Jefferson University, Philadelphia, Pennsylvania 19107, USA – sequence: 8 givenname: Lorenz surname: Hofbauer fullname: Hofbauer, Lorenz organization: Universitätsklinikum Dresden, 01307 Dresden, Germany – sequence: 9 givenname: Claudio orcidid: 0000-0001-6568-8588 surname: Marcocci fullname: Marcocci, Claudio organization: University of Pisa, 56126 Pisa, Italy – sequence: 10 givenname: Uberto surname: Pagotto fullname: Pagotto, Uberto organization: Division of Endocrinology and Diabetes Prevention and Care, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Department of Medical and Surgical Sciences (DIMEC), Alma Mater Studiorum University of Bologna, 40126 Bologna, Italy – sequence: 11 givenname: Andrea orcidid: 0000-0002-1143-4926 surname: Palermo fullname: Palermo, Andrea organization: Unit of Endocrinology and Diabetes, Campus Bio-Medico University, 00128 Rome, Italy – sequence: 12 givenname: Erik surname: Eriksen fullname: Eriksen, Erik organization: Oslo University Hospital, Institute of Clinical Medicine, 0372 Oslo, Norway – sequence: 13 givenname: Meryl surname: Brod fullname: Brod, Meryl organization: The Brod Group, Mill Valley, California 94941, USA – sequence: 14 givenname: Denka surname: Markova fullname: Markova, Denka organization: Ascendis Pharma Inc , Palo Alto, California 94301, USA – sequence: 15 givenname: Alden surname: Smith fullname: Smith, Alden organization: Ascendis Pharma Inc , Palo Alto, California 94301, USA – sequence: 16 givenname: Susanne surname: Pihl fullname: Pihl, Susanne organization: Ascendis Pharma A/S, 2900 Hellerup, Denmark – sequence: 17 givenname: Sanchita surname: Mourya fullname: Mourya, Sanchita organization: Ascendis Pharma Inc , Palo Alto, California 94301, USA – sequence: 18 givenname: David B surname: Karpf fullname: Karpf, David B organization: Ascendis Pharma A/S, 2900 Hellerup, Denmark – sequence: 19 givenname: Aimee D orcidid: 0000-0003-1801-5289 surname: Shu fullname: Shu, Aimee D organization: Ascendis Pharma Inc , Palo Alto, California 94301, USA |
BackLink | https://www.ncbi.nlm.nih.gov/pubmed/34347093$$D View this record in MEDLINE/PubMed |
BookMark | eNptkk1vEzEQhi1URNPClSOyxIVDt_XXrtcckEKgBKkSEQoSN2t27U2MvHZqJ0Xpr8dVQgVS5YM99jOv39HMGToJMViEXlNySRklV713wY5XZgVdLeUzNKFK1JWkSp6gCSGMVkqyn6foLOdfhFAhav4CnXLBhSSKT9DdApZzfB3Tb0jmPZ7i7xBMHN29NRf4U9x13lYfyx8lWnjobRerWQzbFL23Bi_WkC1meJkceByHcoCQC4AXRdUFPDU7v8Xz_SZuIMF2vU_RGZfHl-j5AD7bV8f9HP24_ryczaubb1--zqY3VS9IK6qOEc7awfCWMGYFY7TuwLBGCVKqB0mE5JywQfGmHtRge0slpTVlDVe0AcnP0YeD7mbXjdb0tlgHrzfJjZD2OoLT_78Et9areKfbphVCtUXg3VEgxdudzVs9utxb7yHYuMua1XVLlBCMFvTtAV2Bt9qFIRbF_gHXU1n8t7KRqlCXT1BlGTu6vjR3cOX-qYQ-xZyTHR7dU6IfZkAfZkAfZ6AkvPm35kf8b9P5H5-hroU |
CitedBy_id | crossref_primary_10_1007_s00431_022_04772_6 crossref_primary_10_1007_s11914_023_00790_x crossref_primary_10_1007_s12020_024_03807_2 crossref_primary_10_1016_j_ando_2023_04_001 crossref_primary_10_1002_jbmr_4659 crossref_primary_10_1002_jbmr_4716 crossref_primary_10_1002_jbmr_4726 crossref_primary_10_1007_s12020_022_03292_5 crossref_primary_10_1007_s12020_023_03443_2 crossref_primary_10_1002_jbmr_4673 crossref_primary_10_20945_2359_3997000000549 crossref_primary_10_1210_clinem_dgae121 crossref_primary_10_1002_jbmr_4566 crossref_primary_10_1002_jbmr_4676 crossref_primary_10_1007_s12020_021_02978_6 crossref_primary_10_3803_EnM_2024_1916 crossref_primary_10_20945_2359_3997000000554 crossref_primary_10_1007_s12020_024_03784_6 crossref_primary_10_1016_S2588_932X_23_00028_1 crossref_primary_10_3390_ijms222312975 crossref_primary_10_1093_jbmrpl_ziae045 crossref_primary_10_1002_jbmr_4691 crossref_primary_10_1210_jcemcr_luad136 crossref_primary_10_1002_jbmr_4780 |
Cites_doi | 10.1056/NEJM197711032971804 10.1002/jbmr.4016 10.1056/NEJM197308162890710 10.1007/s00198-018-04819-1 10.1056/NEJM197711102971906 10.1210/jc.2017-01471 10.1210/jc.2015-4135 10.1210/jc.2019-01010 10.1002/jbmr.470 10.1210/jc.2012-1808 10.1210/jc.2015-3910 10.1007/s12020-017-1377-3 10.1016/j.ecl.2018.07.001 10.1002/jbmr.3824 10.4158/EP13328.OR 10.1056/NEJM198904133201506 10.1007/BF03346763 10.1002/jbmr.452 |
ContentType | Journal Article |
Copyright | The Author(s) 2021. Published by Oxford University Press on behalf of the Endocrine Society. COPYRIGHT 2022 Oxford University Press The Author(s) 2021. Published by Oxford University Press on behalf of the Endocrine Society. 2021 |
Copyright_xml | – notice: The Author(s) 2021. Published by Oxford University Press on behalf of the Endocrine Society. – notice: COPYRIGHT 2022 Oxford University Press – notice: The Author(s) 2021. Published by Oxford University Press on behalf of the Endocrine Society. 2021 |
DBID | CGR CUY CVF ECM EIF NPM AAYXX CITATION 7X8 5PM |
DOI | 10.1210/clinem/dgab577 |
DatabaseName | Medline MEDLINE MEDLINE (Ovid) MEDLINE MEDLINE PubMed CrossRef MEDLINE - Academic PubMed Central (Full Participant titles) |
DatabaseTitle | MEDLINE Medline Complete MEDLINE with Full Text PubMed MEDLINE (Ovid) CrossRef MEDLINE - Academic |
DatabaseTitleList | MEDLINE - Academic MEDLINE CrossRef |
Database_xml | – sequence: 1 dbid: NPM name: PubMed url: https://proxy.k.utb.cz/login?url=http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed sourceTypes: Index Database – sequence: 2 dbid: EIF name: MEDLINE url: https://proxy.k.utb.cz/login?url=https://www.webofscience.com/wos/medline/basic-search sourceTypes: Index Database |
DeliveryMethod | fulltext_linktorsrc |
Discipline | Medicine |
EISSN | 1945-7197 |
EndPage | e385 |
ExternalDocumentID | A702287679 10_1210_clinem_dgab577 34347093 |
Genre | Multicenter Study Clinical Trial, Phase II Randomized Controlled Trial Research Support, Non-U.S. Gov't Journal Article |
GeographicLocations | Denmark |
GeographicLocations_xml | – name: Denmark |
GroupedDBID | --- -~X .55 .XZ 08P 0R~ 18M 29K 2WC 34G 354 39C 4.4 48X 53G 5GY 5RS 5YH 8F7 AABZA AACZT AAIMJ AAPQZ AAPXW AARHZ AAUAY AAVAP AAWTL ABBLC ABJNI ABLJU ABMNT ABNHQ ABOCM ABPMR ABPPZ ABPQP ABPTD ABQNK ABWST ABXVV ACGFO ACGFS ACPRK ACUTJ ACYHN ADBBV ADGKP ADGZP ADHKW ADQBN ADRTK ADVEK AELWJ AEMDU AENEX AENZO AETBJ AEWNT AFCHL AFFZL AFGWE AFOFC AFRAH AFXAL AGINJ AGKRT AGQXC AGUTN AHMBA AJEEA ALMA_UNASSIGNED_HOLDINGS APIBT ARIXL ASPBG ATGXG AVWKF AZFZN BAWUL BAYMD BCRHZ BEYMZ BPHCQ BSWAC BTRTY BVXVI C45 CDBKE CGR CS3 CUY CVF D-I DAKXR DIK E3Z EBS ECM EIF EMOBN ENERS F5P FECEO FHSFR FLUFQ FOEOM FOTVD FQBLK GAUVT GJXCC GX1 H13 HZ~ H~9 IAO IHR INH ITC KOP KQ8 KSI KSN L7B M5~ MHKGH MJL N9A NLBLG NOMLY NOYVH NPM O9- OAUYM OBH OCB ODMLO OFXIZ OGEVE OHH OJZSN OK1 OPAEJ OVD OVIDX P2P P6G PQQKQ PROAC REU ROX ROZ TEORI TJX TLC TR2 TWZ VVN W8F WOQ X7M YBU YFH YHG YOC YSK ZY1 ~02 ~H1 AAYXX CITATION AABJS AABMN ABSAR ABXZS ACIMA ADEIU AIKOY AIMBJ ALXQX ASMCH AZQFJ BYORX CASEJ DPPUQ ZA5 7X8 5PM KBUDW |
ID | FETCH-LOGICAL-c4084-b20328fd38022e42215bad26940121a70473302f9365f9fece171151263916a73 |
ISSN | 0021-972X |
IngestDate | Tue Sep 17 21:03:40 EDT 2024 Sat Aug 17 03:53:36 EDT 2024 Thu Feb 22 23:40:17 EST 2024 Sat Dec 16 00:20:51 EST 2023 Thu Sep 26 15:40:37 EDT 2024 Sat Sep 28 08:26:02 EDT 2024 |
IsDoiOpenAccess | true |
IsOpenAccess | true |
IsPeerReviewed | true |
IsScholarly | true |
Issue | 1 |
Keywords | prodrug parathyroid hormone hypoparathyroidism TransCon PTH replacement therapy PTH(1-34) |
Language | English |
License | The Author(s) 2021. Published by Oxford University Press on behalf of the Endocrine Society. This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs licence (https://creativecommons.org/licenses/by-nc-nd/4.0/), which permits non-commercial reproduction and distribution of the work, in any medium, provided the original work is not altered or transformed in any way, and that the work is properly cited. For commercial re-use, please contact journals.permissions@oup.com |
LinkModel | OpenURL |
MergedId | FETCHMERGED-LOGICAL-c4084-b20328fd38022e42215bad26940121a70473302f9365f9fece171151263916a73 |
Notes | ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-1 ObjectType-News-3 content type line 23 |
ORCID | 0000-0003-1801-5289 0000-0002-1143-4926 0000-0001-6568-8588 |
OpenAccessLink | https://pubmed.ncbi.nlm.nih.gov/PMC8684498 |
PMID | 34347093 |
PQID | 2558094421 |
PQPubID | 23479 |
ParticipantIDs | pubmedcentral_primary_oai_pubmedcentral_nih_gov_8684498 proquest_miscellaneous_2558094421 gale_infotracmisc_A702287679 gale_infotracacademiconefile_A702287679 crossref_primary_10_1210_clinem_dgab577 pubmed_primary_34347093 |
PublicationCentury | 2000 |
PublicationDate | 2022-01-01 |
PublicationDateYYYYMMDD | 2022-01-01 |
PublicationDate_xml | – month: 01 year: 2022 text: 2022-01-01 day: 01 |
PublicationDecade | 2020 |
PublicationPlace | United States |
PublicationPlace_xml | – name: United States – name: US |
PublicationTitle | The journal of clinical endocrinology and metabolism |
PublicationTitleAlternate | J Clin Endocrinol Metab |
PublicationYear | 2022 |
Publisher | Oxford University Press |
Publisher_xml | – name: Oxford University Press |
References | Reichel (2021121823120492600_CIT0004) 1989; 320 Rubin (2021121823120492600_CIT0019) 2011; 26 Ware (2021121823120492600_CIT0010) Karpf (2021121823120492600_CIT0008) 2020; 35 Maruish (2021121823120492600_CIT0011) Winer (2021121823120492600_CIT0017) 2012; 97 Holten-Andersen (2021121823120492600_CIT0009) 2019; 34 Büttner (2021121823120492600_CIT0022) 2017; 58 Eastell (2021121823120492600_CIT0018) 2019; 30 Sikjaer (2021121823120492600_CIT0020) 2011; 26 Haussler (2021121823120492600_CIT0003) 1977; 297 Cusano (2021121823120492600_CIT0023) 2018; 47 Brod (2021121823120492600_CIT0013) 2021 Bilezikian (2021121823120492600_CIT0006) 2016; 101 Vokes (2021121823120492600_CIT0012) 2018; 103 Mitchell (2021121823120492600_CIT0005) 2012; 97 Center for Drug Evaluation and Research (2021121823120492600_CIT0007) 2014 DeLuca (2021121823120492600_CIT0001) 1973; 289 Cusano (2021121823120492600_CIT0016) 2013; 36 Hadker (2021121823120492600_CIT0021) 2014; 20 Mannstadt (2021121823120492600_CIT0014) 2019; 104 Rubin (2021121823120492600_CIT0015) 2016; 101 Haussler (2021121823120492600_CIT0002) 1977; 297 |
References_xml | – volume: 297 start-page: 974 issue: 18 year: 1977 ident: 2021121823120492600_CIT0002 article-title: Basic and clinical concepts related to vitamin D metabolism and action (first of two parts) publication-title: N Engl J Med. doi: 10.1056/NEJM197711032971804 contributor: fullname: Haussler – volume: 35 start-page: 1430 issue: 8 year: 2020 ident: 2021121823120492600_CIT0008 article-title: A randomized double-blind placebo-controlled first-in-human phase 1 trial of TransCon PTH in healthy adults publication-title: J Bone Miner Res. doi: 10.1002/jbmr.4016 contributor: fullname: Karpf – volume: 97 start-page: 391 issue: 2 year: 2012 ident: 2021121823120492600_CIT0017 article-title: Synthetic human parathyroid hormone 1-34 replacement therapy: a randomized crossover trial comparing pump versus injections in the treatment of chronic hypoparathyroidism publication-title: J Clin Endocrinol Metab. contributor: fullname: Winer – volume: 289 start-page: 359 issue: 7 year: 1973 ident: 2021121823120492600_CIT0001 article-title: The kidney as an endocrine organ for the production of 1,25-dihydroxyvitamin D 3, a calcium-mobilizing hormone publication-title: N Engl J Med. doi: 10.1056/NEJM197308162890710 contributor: fullname: DeLuca – volume: 30 start-page: 667 issue: 3 year: 2019 ident: 2021121823120492600_CIT0018 article-title: Bone turnover markers to explain changes in lumbar spine BMD with abaloparatide and teriparatide: results from ACTIVE publication-title: Osteoporos Int. doi: 10.1007/s00198-018-04819-1 contributor: fullname: Eastell – volume: 297 start-page: 1041 issue: 19 year: 1977 ident: 2021121823120492600_CIT0003 article-title: Basic and clinical concepts related to vitamin D metabolism and action (second of two parts) publication-title: N Engl J Med. doi: 10.1056/NEJM197711102971906 contributor: fullname: Haussler – volume-title: User’s manual for the SF-36v2 Health Survey. ident: 2021121823120492600_CIT0011 contributor: fullname: Maruish – volume: 103 start-page: 722 issue: 2 year: 2018 ident: 2021121823120492600_CIT0012 article-title: Recombinant human parathyroid hormone effect on health-related quality of life in adults with chronic hypoparathyroidism publication-title: J Clin Endocrinol Metab. doi: 10.1210/jc.2017-01471 contributor: fullname: Vokes – start-page: 5(1):70 year: 2021 ident: 2021121823120492600_CIT0013 article-title: Psychometric validation of the hypoparathyroidism patient experience scales (HPES) publication-title: J Patient Rep Outcomes. contributor: fullname: Brod – volume: 101 start-page: 2742 issue: 7 year: 2016 ident: 2021121823120492600_CIT0015 article-title: Therapy of hypoparathyroidism with PTH(1-84): a prospective six year investigation of efficacy and safety publication-title: J Clin Endocrinol Metab. doi: 10.1210/jc.2015-4135 contributor: fullname: Rubin – volume: 104 start-page: 5136 issue: 11 year: 2019 ident: 2021121823120492600_CIT0014 article-title: Safety and efficacy of 5 years of treatment with recombinant human parathyroid hormone in adults with hypoparathyroidism publication-title: J Clin Endocrinol Metab. doi: 10.1210/jc.2019-01010 contributor: fullname: Mannstadt – volume: 26 start-page: 2358 issue: 10 year: 2011 ident: 2021121823120492600_CIT0020 article-title: The effect of adding PTH(1-84) to conventional treatment of hypoparathyroidism: a randomized, placebo-controlled study publication-title: J Bone Miner Res. doi: 10.1002/jbmr.470 contributor: fullname: Sikjaer – year: 2014 ident: 2021121823120492600_CIT0007 contributor: fullname: Center for Drug Evaluation and Research – volume: 97 start-page: 4507 issue: 12 year: 2012 ident: 2021121823120492600_CIT0005 article-title: Long-term follow-up of patients with hypoparathyroidism publication-title: J Clin Endocrinol Metab. doi: 10.1210/jc.2012-1808 contributor: fullname: Mitchell – volume: 101 start-page: 2313 issue: 6 year: 2016 ident: 2021121823120492600_CIT0006 article-title: Management of hypoparathyroidism: present and future publication-title: J Clin Endocrinol Metab. doi: 10.1210/jc.2015-3910 contributor: fullname: Bilezikian – volume: 58 start-page: 14 issue: 1 year: 2017 ident: 2021121823120492600_CIT0022 article-title: Quality of life in patients with hypoparathyroidism receiving standard treatment: a systematic review publication-title: Endocrine. doi: 10.1007/s12020-017-1377-3 contributor: fullname: Büttner – volume: 47 start-page: 759 issue: 4 year: 2018 ident: 2021121823120492600_CIT0023 article-title: Signs and symptoms of hypoparathyroidism publication-title: Endocrinol Metab Clin North Am. doi: 10.1016/j.ecl.2018.07.001 contributor: fullname: Cusano – volume: 34 start-page: 2075 issue: 11 year: 2019 ident: 2021121823120492600_CIT0009 article-title: Design and preclinical development of TransCon PTH, an investigational sustained-release PTH replacement therapy for hypoparathyroidism publication-title: J Bone Miner Res. doi: 10.1002/jbmr.3824 contributor: fullname: Holten-Andersen – volume: 20 start-page: 671 issue: 7 year: 2014 ident: 2021121823120492600_CIT0021 article-title: Understanding the burden of illness associated with hypoparathyroidism reported among patients in the PARADOX study publication-title: Endocr Pract. doi: 10.4158/EP13328.OR contributor: fullname: Hadker – volume: 320 start-page: 980 issue: 15 year: 1989 ident: 2021121823120492600_CIT0004 article-title: The role of the vitamin D endocrine system in health and disease publication-title: N Engl J Med. doi: 10.1056/NEJM198904133201506 contributor: fullname: Reichel – volume: 36 start-page: 1121 issue: 11 year: 2013 ident: 2021121823120492600_CIT0016 article-title: Use of parathyroid hormone in hypoparathyroidism publication-title: J Endocrinol Invest. doi: 10.1007/BF03346763 contributor: fullname: Cusano – volume-title: Med Care ident: 2021121823120492600_CIT0010 contributor: fullname: Ware – volume: 26 start-page: 2727 issue: 11 year: 2011 ident: 2021121823120492600_CIT0019 article-title: PTH(1-84) administration reverses abnormal bone-remodeling dynamics and structure in hypoparathyroidism publication-title: J Bone Miner Res. doi: 10.1002/jbmr.452 contributor: fullname: Rubin |
SSID | ssj0014453 |
Score | 2.551407 |
Snippet | Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34)... Abstract Context Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting... Context: Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of... CONTEXTHypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of... |
SourceID | pubmedcentral proquest gale crossref pubmed |
SourceType | Open Access Repository Aggregation Database Index Database |
StartPage | e372 |
SubjectTerms | Adult Adults Aged Alfacalcidol Calcifediol Calcium - administration & dosage Calcium - blood Care and treatment Clinical trials Delayed-Action Preparations - administration & dosage Delayed-Action Preparations - adverse effects Double-Blind Method Drug Administration Schedule Female Health surveys Hormone Replacement Therapy - adverse effects Hormone Replacement Therapy - methods Hormone therapy Humans Hypoparathyroidism Hypoparathyroidism - blood Hypoparathyroidism - complications Hypoparathyroidism - diagnosis Hypoparathyroidism - drug therapy Male Middle Aged Online Only Parathyroid hormone Parathyroid Hormone - administration & dosage Parathyroid Hormone - adverse effects Parathyroid Hormone - blood Patient Reported Outcome Measures Phosphates Placebos - administration & dosage Placebos - adverse effects Prodrugs - administration & dosage Prodrugs - adverse effects Quality of Life Treatment Outcome Type 2 diabetes Vitamin D Vitamin D - administration & dosage Vitamin D - blood |
Title | PaTH Forward: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism |
URI | https://www.ncbi.nlm.nih.gov/pubmed/34347093 https://search.proquest.com/docview/2558094421 https://pubmed.ncbi.nlm.nih.gov/PMC8684498 |
Volume | 107 |
hasFullText | 1 |
inHoldings | 1 |
isFullTextHit | |
isPrint | |
link | http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1db9MwFLXKkBAvaHyXDWQkJB66QJo4ccJbmSgVYqgandS3yE4cmqlNpq5Fan8iv4p7YydN1j0MXqIq_mjie-J7bZ17TMi7AL2OH0gLbC8tFgj4pEIVW4kjhReDS1ellt7ZD390wb5NvWmn86fBWlqv5Id4e2teyf9YFe6BXTFL9h8sW3cKN-A32BeuYGG43snGYzEZ9YbFEpmvOsX8XORJsci2egsTomM5V9ZnCCV1uIh75rKwTjU_fQ7B5ngGbqzn9Cbl6R0QOZbOCyr0xtA3boagQEdvtLkqSpXw2WZZZEmlO3i5w1pDgqLOtlTwMDAr5Tudp4VaAermVXuc6Wd6C3YwzzZit7N6ri7zheFxfxfLBiNfZobvf418_9qr_IxnMAzbNufYbGc4TmM7o0ov6FshL89YBwelZ-WQeRbvayJvPW3r03Jb-NSTsHL1aUDGoStXHwq05yxgtQsWxjFR8O0Nk19CeuZMmZYu9w1_WbMYcf0EfUS6h8i0v0fuOzz0kF76dVrTjWDdahRRzesZAVHMoNLtP5r2rQDpZpjQiJPaHN5GUDQ5JI_MaoYONDQfk47Kn5AHZ4av8ZT8RoRSg9BPdEB3-DyhTXSe0H1s0hKb1KElNmmR0gqbFLBJs5yW2KT72HxGLoZfJqcjy5z0YcXMDpglHZR1TBMXE78VcyAOlSLBJGuUHBTcZtx1bScNXd9Lw1TFqs_7GKv6mDcuuPucHORFrl4S6jEIsFEkk0PgJUNfgBsVgbR5KqC5Ul3yvhre6EoLukS3GxJq4uhHiBkY4liYhBX4H9RMiwYctaO4z8MuOW7VhBk6bhW_rewXYRHSGnNVrK8jWM8HdsiY0--SF9qe9UO5zGXcDt0u4S1L1xVQGL5dkmezUiA-8APGwuDVnV_1iDzcfYvH5GC1XKvXEGyv5JsSxX8B-BDXwg |
link.rule.ids | 230,315,786,790,891,27955,27956 |
linkProvider | Geneva Foundation for Medical Education and Research |
openUrl | ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=PaTH+Forward%3A+A+Randomized%2C+Double-Blind%2C+Placebo-Controlled+Phase+2+Trial+of+TransCon+PTH+in+Adult+Hypoparathyroidism&rft.jtitle=The+journal+of+clinical+endocrinology+and+metabolism&rft.au=Khan%2C+Aliya+A&rft.au=Rejnmark%2C+Lars&rft.au=Rubin%2C+Mishaela&rft.au=Schwarz%2C+Peter&rft.date=2022-01-01&rft.issn=0021-972X&rft.eissn=1945-7197&rft.volume=107&rft.issue=1&rft.spage=e372&rft.epage=e385&rft_id=info:doi/10.1210%2Fclinem%2Fdgab577&rft.externalDBID=n%2Fa&rft.externalDocID=10_1210_clinem_dgab577 |
thumbnail_l | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=0021-972X&client=summon |
thumbnail_m | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=0021-972X&client=summon |
thumbnail_s | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=0021-972X&client=summon |