Efficacy and Safety of TransCon Parathyroid Hormone (PTH) in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial
An erratum to this article was published (J Clin Endocrinol Metab, 17 Mar 2025 ↗)
Authors' conclusion
At week 52 of the PaTHway trial, TransCon PTH showed sustained efficacy, safety, and tolerability in adults with hypoparathyroidism.
Study context
- Population
- Adults with hypoparathyroidism, 21 sites across North America and Europe78 completed week 52
- Participants
- 82 adults
- Comparison
- TransCon PTH once daily in the open-label extension (OLE)
- Outcome
- Multicomponent: normal serum calcium (8.3-10.6 mg/dL) and independence from conventional therapy (≤600 mg/day elemental calcium and no active vitamin D)
- Follow-up
- Week 52
Methodological details52-week results, 26-week double-blind, placebo-controlled period, 156-week open-label extension, multicentre
- 52-week results
- 26-week double-blind, placebo-controlled period
- 156-week open-label extension
- Multicentre
Main finding
As reported in the abstract
Multicomponent efficacy endpoint: normal serum calcium (8.3-10.6 mg/dL) and independence from conventional therapy
Met the multicomponent efficacy endpoint
81%
63/78
Further findings
Independence from conventional therapy · week 52
- Achieved independence from conventional therapy
- 95% (74/78)
- Required active vitamin D
- 0
Patient-reported outcomes
- Sustained improvements in quality of life, physical functioning, and well-being
BMD Z-scores · baseline to week 52
- Mean BMD Z-scores decreased toward age- and sex-matched norms
Safety
24-hour urine calcium excretion · baseline to week 52
- Mean (SD) at baseline, mg/day
- 376 (168)
- Mean (SD) at week 52, mg/day
- 195 (114)
Treatment-emergent adverse events
- Led to trial discontinuation during the OLE
- 0
- Most were mild or moderate
Original publication
Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial. J Clin Endocrinol Metab. Published 8 October 2024. doi:10.
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