Treatment of Hypoparathyroidism in children 12 to 17 years old
Research type
Research Study
Full title
An Open-label, Single-dose Study to Determine the Pharmacokinetic/Pharmacodynamic Profile of Parathyroid Hormone (rDNA) Administered Subcutaneously at a Dose of 50 μg in Subjects with Hypoparathyroidism Who Are 12 to Less Than 18 Years of Age
IRAS ID
191274
Contact name
Syed Faisal Ahmed
Contact email
Sponsor organisation
NPS Pharmaceuticals, Inc.
Eudract number
2015-003108-22
Duration of Study in the UK
0 years, 5 months, 3 days
Research summary
Hypoparathyroidism means the parathyroid gland(s) in the body are not making enough parathyroid hormone (PTH). If the body does not produce enough PTH it causes an abnormally low level of calcium in the blood (hypocalcaemia) and an abnormally high level of phosphorus in the blood (hyperphosphataemia).
Main symptoms are various neuromuscular issues including numbness, difficulty in concentrating and cardiac arrhythmias.
Current treatment for hypoparathyroidism involves taking large amounts of Vitamin D and large amounts of calcium. Because hypoparathyroidism is a chronic condition, people with the condition will most likely need to keep taking the calcium and vitamin D for the rest of their lives.
A new treatment, recombinant human parathyroid hormone (rDNA) has been approved in the USA for adults and it is currently under development in the EU. It is an exact copy of the human parathyroid hormone produced with a specific manufacturing process. The sponsor is currently researching this experimental drug to treat hypoparathyroidism in children aged 12 to less than 18 years old.
As this is an initial phase of new drug development, the Sponsor would like to ensure that it is safe and well tolerated in children and adolescents as well as evaluating the amount of the study drug that will be present in the participant’s blood after the administration of a single subcutaneous (under the skin) dose.
Participation in the study will last a maximum of 123 days and it will require the participants to spend three nights in the hospital. During the study physical examinations e.g. height/weight, vital signs e.g. pulse and blood pressure, blood/urine samples and an ECG (to measure the activity of the heart) will be undertaken.
This study is planned to take place at 5 sites in Europe and will involve approximately 10 participants aged 12 to less than 18 years old.
REC name
West of Scotland REC 1
REC reference
15/WS/0250
Date of REC Opinion
8 Dec 2015
REC opinion
Further Information Favourable Opinion