Safety and Efficacy of PTH-IA

Recruiting Phase 1 Phase 2 Interventional Study
Jansen's Metaphyseal Chondrodysplasia
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
3 – 100
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
12 (estimated)
Sponsor
National Institute of Dental and Craniofacial Research (NIDCR) · NIH
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About This Trial
Jansen s Metaphyseal Chondrodysplasia (JMC) is a very rare disorder with only approximately 30 people known to have the disease worldwide. It is caused by parathyroid hormone 1 receptor (PTH1R) variants leading to constitutive activation of the receptor for parathyroid hormone (PTH) and parathyroid hormone-related peptide (PTHrP). PTH1R is predominantly expressed in the kidneys and bone and growth…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Willingness of participant and/or guardian to sign a written informed consent form, which must be obtained prior to initiation of study procedures. 2. Period 1: Adu…
Contacts

Olivia J de Jong, C.R.N.P.

(240) 595-2764

olivia.dejong@nih.gov

CONTACT

Alison M Boyce, M.D.

(301) 827-4802

alison.boyce@nih.gov

CONTACT