Phase I open-label rhPTH(1-84) PK/PD in Pediatric Hypoparthyroidism
Research type
Research Study
Full title
An Open-label, Phase 1, Study to Determine the Pharmacokinetic/Pharmacodynamic Profiles of rhPTH(1-84) Administered Twice Daily Subcutaneously to Pediatric Subjects with Hypoparathyroidism
IRAS ID
273514
Contact name
Raja Padidela
Contact email
Sponsor organisation
Shire Human Genetic Therapies, Inc.
Eudract number
2019-001524-35
Clinicaltrials.gov Identifier
P/136/2019, EMA Decision number of Paediatric Investigation Plan
Duration of Study in the UK
0 years, 8 months, 1 days
Research summary
Parathyroid hormone is important for maintaining blood calcium levels. It does this by converting vitamin D to active-vitamin D which absorbs calcium from diet. In a condition called hypoparathyroidism (low parathyroid hormone levels) blood calcium levels can drop to dangerously low levels. Standard treatment is oral calcium and active-vitamin D-analoges. Some patients don’t respond to standard treatment or develop nephrocalcinosis (calcium deposition in the kidneys) which can affect kidney function.
This is a Phase I open label study to evaluate the safety and Pharmacokinetic/pharmacodynamic effects of rhPTH (1-84) (the study drug) in paediatric and adolescent participants with hypoparathyroidism.
rhPTH (1-84), recombinant (grown in a cell) human parathyroid hormone, has been conditionally approved in the EU
for adult patients who are non-responsive to standard treatment. rhPTH (1-84) has not been studied in paediatric
populations. It is hoped that this study will support dose selection for a larger study to follow, which will test safety and
efficacy over a 6-month period.
This study will consist of at least 14 participants split into two cohorts with age categories 12-18 years-old and 0-12 years-old (there will be at least 1 participant aged 2-years-old or younger). The dosing will be staggered for the first 3 participants in each cohort.
rhPTH (1-84) will be administered via 2 subcutaneous injections, 12-hours-apart, with participants above 10kg
receiving 0.36μg/kg body weight up to 50μg and 0.54μg/kg body weight for participants under 10kg. The treatment will
be performed over a 24-hour inpatient visit with a follow up visit performed no longer than 32-days after the last dose.
Throughout the inpatient stay, blood and urine tests will measure Pharmacokinetic/pharmacodynamic of rhPTH (1-84).
Safety will be tested through blood tests, ECG’s, vital signs and physical examinations.
Shire Human Genetic Therapies, Inc is the sponsor for this study.REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
20/YH/0149
Date of REC Opinion
17 Jun 2020
REC opinion
Further Information Favourable Opinion