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PCSK1 Deficiency Obesity clinical trials

View clinical trials related to PCSK1 Deficiency Obesity.

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NCT ID: NCT04966741 Completed - Clinical trials for Bardet-Biedl Syndrome

Setmelanotide in Pediatric Patients With Rare Genetic Diseases of Obesity

Start date: February 16, 2022
Phase: Phase 3
Study type: Interventional

This is a phase 3 open-label, one-arm, clinical study to evaluate the efficacy, safety and tolerability of setmelanotide over 1 year of treatment, in pediatric patients aged 2 to <6 years with obesity due to either biallelic variants of the POMC, PCSK1 or LEPR genes or Bardet-Biedl Syndrome (BBS).

NCT ID: NCT03621007 Completed - Clinical trials for PCSK1 Deficiency Obesity

An Observational,Prospective Natural History Study of Early-Onset Extreme Obesity Due to Bi-Allelic Loss-of-Function Mutations in the POMC, PCSK1 or LEPR Genes

NHS
Start date: August 6, 2019
Phase:
Study type: Observational

This is an observational study. There are no protocol-defined visits, although patients are expected to have routine office visits approximately every 6 months. Upon signing of informed consent/assent and study enrollment, historical data will be abstracted from the patient's medical chart. The patient will then be observed prospectively for up to 5 years, with additional data collected from routine healthcare encounters and direct-to-patient questionnaires (where local laws allow), including laboratory tests, physical exam and patient reported outcomes/quality of life measures. Patients will be consented/assented to provide blood samples for biomarker assessments, DNA sequencing and archiving.