PaTHway: TransCon PTH in Adults with Hypoparathyroidism
Research type
Research Study
Full title
PaTHway TRIAL: A Phase 3, 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
289603
Contact name
Richard Quinton
Contact email
Sponsor organisation
Ascendis Pharma Bone Diseases A/S
Eudract number
2020-003380-26
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
IND, 133469
Duration of Study in the UK
3 years, 9 months, 27 days
Research summary
This is a phase 3 study assessing the safety, tolerability and effectiveness of TransCon PTH (study medication) in patients with Hypoparathyroidism. The study consists of 3 periods; a Screening Period (approx. 4 weeks), Blinded Treatment Period (where participants may receive TransCon PTH or a placebo, approx. 26 weeks) and an Extension Period where participants will receive ION373 TransCon PTH (approx. 156 weeks).
Hypoparathyroidism is a disease that changes the reduction of secretion or activity of the parathyroid hormone (PTH) which leads to decreased blood levels of calcium (hypocalcaemia) and increased levels of blood phosphate (hyperphosphatemia). It can be acquired post-surgery, auto-immune (when the body’s immune system attacks its own tissues), genetic or idiopathic (without known reason). The US prevalence rate is estimated to range from 60,000-115,000 people, thus meeting orphan disease status.
The intended effect of TransCon PTH is to reduce the symptoms of hypoparathyroidism, improve quality of life (QOL) and lower the long-term risk of renal damage, without adverse effect on bone. Currently, standard of care is active vitamin D and high doses of calcium, sometimes leading to renal damage, kidney disease and low bone turnover.
Approximately 76 participants (male and female, aged 18 and over) are planned to be enrolled in this study globally and participants will visit the study site at least 34 times. Participants will take part for approximately 190 weeks (about 3 ½ years), and will undergo procedures such as completing questionnaires, physical exams, X-rays, ECGs and urine and blood sample collections.
REC name
South Central - Hampshire B Research Ethics Committee
REC reference
20/SC/0428
Date of REC Opinion
21 Jan 2021
REC opinion
Further Information Favourable Opinion