Recombinant human parathyroid hormone for hypoparathyroidism in adults
Research type
Research Study
Full title
A Randomized, Double-blind, Placebo-controlled, Adaptive Study to Evaluate Symptom Improvement and Metabolic Control Among Adult Subjects With Symptomatic Hypoparathyroidism Treated With Recombinant Human Parathyroid Hormone [rhPTH(1-84)]
IRAS ID
227579
Contact name
William Fraser
Contact email
Sponsor organisation
Shire Human Genetic Therapies, Inc.
Eudract number
2017-000284-32
Duration of Study in the UK
3 years, 1 months, 24 days
Research summary
Summary of Research
Recombinant Human Parathyroid Hormone (rhPTH(1-84)) is a hormone treatment identical to the natural hormone present in humans. rhPTH(1-84) is thought to treat hypoparathyroidism whilst reducing the risk of kidney complications, which potentially results from standard therapy consisting of calcium and vitamin D supplementation.
This study will evaluate symptom improvement and metabolic control among adults with hypoparathyroidism treated with the study treatment, rhPTH(1-84), compared with standard therapy.
Between 118 and 150 patients will take part in this study which will consist of 3 phases: screening, treatment and follow up. During screening, patients will undergo a physical examination, ECG, X-ray of the left wrist and left hand to confirm whether or not the bone growth process has stopped in participants 25 years of age or younger, blood and urine laboratory tests, and will be assessed for eligibility. Eligible patients will be assigned at random to receive either rhPTH(1-84) or placebo (a control that looks like the study drug) via subcutaneous injection (injection under the skin). In addition, all patients will be given vitamin D and calcium supplements (the current standard of care treatment). Participants will not know which treatment group they have been assigned to for the duration of the study.
The study treatment will be administered daily for a total of 26 weeks, after which participants will be followed up weekly for 4 weeks following end of treatment. Participants will be asked to visit the clinic at least 12 and not more than 19 times during the course of the study, over a maximum duration of approximately 33 weeks. Study procedures include physical examinations, vital signs, ECGs, blood and urine tests, patient diaries, neurocognitive assessments and patient questionnaires.
Shire Human Genetic Therapies, Inc. is the Sponsor.
Summary of Results
Treatment with Natpar(a) over the course of the trial resulted in superior improvements in symptoms of Hypoparathyroid and metabolic control in subjects with symptomatic Hypoparathyroid compared with placebo. The safety profile of Natpa(a) was consistent with the data from previous studies with Natpar(a), and no new safety signals were identified.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
17/EE/0463
Date of REC Opinion
5 Feb 2018
REC opinion
Further Information Favourable Opinion