PaTH Forward
Research type
Research Study
Full title
PaTH Forward: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial with an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults with Hypoparathyroidism.
IRAS ID
264178
Contact name
Richard Quinton
Contact email
Sponsor organisation
Ascendis Pharma Bone Diseases A/S
Eudract number
2018-004815-33
Duration of Study in the UK
1 years, 5 months, 31 days
Research summary
Hypoparathyroidism (HP) is the reduction of secretion or activity of parathyroid hormone (PTH) which leads to decreased blood levels of calcium (hypocalcemia) and increased levels of blood phosphate (hyperphosphatemia). To improve hypocalcaemia, current standard of care (SOC) for HP includes active vitamin D and calcium.
In the absence of a true PTH replacement therapy that replaces active PTH within the normal physiologic range 24 hours per day, chronic HP therapy has been challenging. Chronic administration of active vitamin D and calcium may cause long-term morbidity and mortality, so replacing the missing hormone in HP patients has long been desired.
TransCon PTH is a sustained-release parathyroid hormone that is injected subcutaneously (under the skin) once a day. The purpose of this study is to assess the effectiveness of daily TransCon PTH on blood and urine calcium levels, and on the amount of active vitamin D and calcium doses required to manage the symptoms of HP.
The study will enroll worldwide approximately 40 male and female adults with HP. After enrolment, the patients will be assigned to either TransCon PTH or placebo at a dose of 15, 18 or 21 mcg/day for a 4-week period (Blinded Treatment Period). During this period adjustments will be made to the vitamin D and calcium supplement doses based on blood test results and symptoms.
After the end of this 4-week period, patients will be assigned to open-label treatment with TransCon PTH for a period of 54 weeks (Extension Period).
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
19/NE/0244
Date of REC Opinion
8 Oct 2019
REC opinion
Further Information Favourable Opinion