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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

NCT03621007 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 8

Last updated 2021-06-30

No results posted yet for this study

Summary

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.

Conditions

  • POMC Deficiency Obesity
  • PCSK1 Deficiency Obesity
  • LEPR Deficiency Obesity

Sponsors & Collaborators

  • Rhythm Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Murray Stewart, MD · Rhythm Pharmceuticals, Inc

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-08-06
Primary Completion
2021-01-22
Completion
2021-01-22

Countries

  • Turkey (Türkiye)

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03621007 on ClinicalTrials.gov