Clinical trials: PCSK1 Deficiency Obesity
POMC Deficiency Obesity, 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
This is an observational study. There are no protocol-defined visits, although patients are expected to have routine office visits approximately every 6 months.
- Conditions
- POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
- Trial name
- NHS (NCT03621007)
- Where
- 3 sites in Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Pro-opiomelanocortin (POMC) Deficiency Obesity
Setmelanotide for the Treatment of Early-Onset Pro-Opiomelanocortin (POMC) Deficiency Obesity
To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with pro-opiomelanocortin (POMC) deficiency or proprotein convertase subtilisin/kexin type…
- Trial name
- NCT02896192
- Testing
- Setmelanotide
- Where
- 7 sites in United States, Belgium and 5 more
- Volunteers
- 15 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Bardet-Biedl Syndrome, POMC Deficiency Obesity
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
This is a phase 3 open-label, clinical study to evaluate the efficacy, safety and tolerability of setmelanotide over 1 year of treatment, in pediatric participants aged 2 to <6 years with obesity due to either biallelic variants…
- Conditions
- Bardet-Biedl Syndrome, POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
- Trial name
- NCT04966741
- Testing
- Setmelanotide
- Where
- 6 sites in United States, Australia and 2 more
- Volunteers
- 12 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant)
An Open-Label Study of Diazoxide Choline in Patients With Genetic Obesities
This is a phase 2 open-label, one-arm, clinical study to evaluate the efficacy and safety of DCCR (diazoxide choline) extended-release tablets over 1 year of treatment, in patients with genetic obesities.
- Conditions
- SH2B1 Deficiency Obesity, Obesity Associated With PCSK1 Mutation (rs6232 Variant), SIM1 Deficiency Obesity
- Trial name
- NCT05532020
- Testing
- DCCR (diazoxide choline) extended-release tablets
- Sponsor
- Soleno Therapeutics, Inc.
POMC Deficiency Obesity, PCSK1 Deficiency Obesity
A Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Genetic Obesity
The purpose of the study was to assess the efficacy of LB54640 in participants with rare genetic disorders of obesity
- Conditions
- POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
- Trial name
- NCT06041841
- Testing
- LB54640
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 4 people
- Sponsor
- LG Chem
Obesity, Childhood, Hyperphagia
Early Genetic Identification of Obesity
TITLE: Whole genetic approach in Early Genetic Identification of Obesity (WEGIO) DESIGN: Multicenter epidemiological study STUDY POPULATION: Participants at risk for a syndromic or a monogenic genetic obesity, incl.
- Conditions
- Obesity, Childhood, Hyperphagia, Retinopathy, Syndactyly
- Trial name
- WEGIO (NCT06239064)
- Testing
- Genetic testing via blood collection
- Where
- 37 sites in Germany
- Volunteers
- 1,000 people
- Sponsor
- Rolfs Consulting und Verwaltungs-GmbH (RCV)