An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)

NCT05183802 | Unknown

• 1 other identifier: RM-493-036
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

An open-label, single-arm, multicenter, Expanded Access Protocol \[EAP\] designed to provide treatment access to setmelanotide (3 mg, administered subcutaneously \[SC\], once daily) for eligible patients with BBS who have no alternative treatment options. All patients will continue to receive setmelanotide at the discretion of the Treating Physician and while they are deriving clinical benefit.

Conditions

Bardet-Biedl Syndrome (BBS); Obesity

Interventions

Setmelanotide, administered subcutaneously [SC], once daily. DRUG

Dosing based on age, and titrated from starting dose to target dose.

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinical diagnosis of BBS
• Males and females aged ≥6 years
• Obesity (≥30 kg/m2) for patients ≥18 years old or weight \>97th percentile for age and sex on growth chart assessment for patients aged 6 to 17 years
• Female participants of child-bearing potential must be confirmed as non-pregnant
• Patients with significant neuropsychiatric disorders such as depression should be carefully evaluated regarding the benefit/risk ratio for the use of setmelanotide
• Patients unable to enroll in another clinical trial with setmelanotide as determined by the treating physician

You may not qualify if:

• Moderate to severe renal dysfunction defined as a glomerular filtration rate (GFR) \<60 mL/min/1.73m2
• History or close family history (parents or siblings) of skin cancer (excluding non-invasive basal or squamous cell lesion) melanoma, or patient history of ocular cutaneous albinism
• Inability to comply with a daily injection regimen
• Patients who have taken part in any previous setmelanotide clinical trial, have met all of the study's eligibility criteria, and have shown meaningful clinical benefit, defined as at least a 5% reduction in body weight for patients aged ≥18 years or a 5% reduction in BMI in patients aged \<18 years from baseline through the end of the preceding study, will be eligible.

Sponsors & Collaborators

• Rhythm Pharmaceuticals, Inc.: lead

Related Links

• Policy on Expanded Access to Investigational Drugs

MeSH Terms

Conditions

Bardet-Biedl Syndrome; Obesity

Interventions

setmelanotide

Condition Hierarchy (Ancestors)

Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Retinitis Pigmentosa; Eye Diseases, Hereditary; Eye Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Linda Shapiro, M.D., Ph.D.

  Chief Medical Officer, Rhythm Pharmaceuticals

  STUDY CHAIR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2021
• First Posted: January 11, 2022
• Last Updated: August 10, 2022

Record last verified: 2022-08