A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH in Adults With Hypoparathyroidism
Summary
During the first four weeks of the trial, participants were randomly assigned to one of four groups: three groups received fixed doses of TransCon PTH and one group received placebo. TransCon PTH or placebo were administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors know who has been assigned to each group. After the four weeks, participants continued in the trial as part of a long-term extension period. During the extension, all participants received TransCon PTH, with the dose adjusted to their individual needs. This was a global trial that was conducted in the United States, Canada, Germany, Denmark, Italy and Norway.
Timeline
- Start
- 2019-08-27
- Primary completion
- 2020-03-06
- Completion
- 2025-04-17
Outcome
Missed primary endpoint
registry analysis (superiority test); TransCon PTH 15 mcg vs Placebo; p = 0.2635; t-test, 2 sided NCT04009291 ↗
registry analysis (superiority test); TransCon PTH 18 mcg vs Placebo; p = 0.6999; t-test, 2 sided NCT04009291 ↗
registry analysis (superiority test); TransCon PTH 21 mcg vs Placebo; p = 0.1394; t-test, 2 sided NCT04009291 ↗
Publications
- Background Rubin MR, Clarke BL, Hofbauer LC, Khan A, Schwarz P, Vokes T, Ahmed I, Palermo A, Cetani F, Pagotto U, Zhao C, Ominsky MS, Lai B, Ukena J, Shu AD, Rejnmark L. Palopegteriparatide for Adults with Chronic Hypoparathyroidism: Skeletal Dynamics Through 3 yr of the Phase 2 paTH Forward Trial. J Bone Miner Res. 2026 Feb 4:zjag013. doi: 10.1093/jbmr/zjag013. Online ahead of print.
- Background Rubin M, Palermo A, Vokes T, Khan AA, Schwarz P, Cetani F, Pagotto U, Tsourdi E, Sikjaer T, Pfeiffer KM, Brod M, McLeod LD, Zhao C, Noori W, Shu AD, Smith AR. Patient-reported outcomes in palopegteriparatide-treated adults with hypoparathyroidism: PaTH Forward trial extension. J Clin Endocrinol Metab. 2026 Mar 17;111(4):945-952. doi: 10.1210/clinem/dgaf653.
- Results Khan AA, Rejnmark L, Rubin M, Schwarz P, Vokes T, Clarke B, Ahmed I, Hofbauer L, Marcocci C, Pagotto U, Palermo A, Eriksen E, Brod M, Markova D, Smith A, Pihl S, Mourya S, Karpf DB, Shu AD. PaTH Forward: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism. J Clin Endocrinol Metab. 2022 Jan 1;107(1):e372-e385. doi: 10.1210/clinem/dgab577.
- Khan AA, Rubin MR, Schwarz P, Vokes T, Shoback DM, Gagnon C, Palermo A, Marcocci C, Clarke BL, Abbott LG, Hofbauer LC, Kohlmeier L, Pihl S, An X, Eng WF, Smith AR, Ukena J, Sibley CT, Shu AD, Rejnmark L. Efficacy and Safety of Parathyroid Hormone Replacement With TransCon PTH in Hypoparathyroidism: 26-Week Results From the Phase 3 PaTHway Trial. J Bone Miner Res. 2023 Jan;38(1):14-25. doi: 10.1002/jbmr.4726. Epub 2022 Nov 12.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | TransCon PTH | Unknown | 15 ug | Subcutaneous |
| Subject | TransCon PTH | Unknown | 18 ug | Subcutaneous |
| Subject | TransCon PTH | Unknown | 21 ug | Subcutaneous |