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...
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| Published in | The journal of clinical endocrinology and metabolism Vol. 107; no. 1; pp. e372 - e385 |
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| Main Authors | , , , , , , , , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Oxford University Press
01.01.2022
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| Subjects | |
| Online Access | Get full text |
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| 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. |
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| 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 |
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| 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 |
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| 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 |
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| 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 |
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| 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 |
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| 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... |
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| 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 |
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