NCT05183802

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

• Status: Approved For Marketing
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Rhythm Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: —

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.

Detailed description

This is an open-label, multicenter, EAP, designed to provide treatment access to setmelanotide for eligible patients who have BBS and who have no alternative treatment options. Patients will undergo clinical and safety assessments before setmelanotide initiation, and then at 3 monthly intervals - or more frequently if clinically indicated - following initiation of setmelanotide and until treatment is completed or discontinued. All patients must have a 4 week (30 day) follow up safety evaluation after treatment is stopped. The end of the program is defined as the last expected evaluation of the last patient or the date of reimbursement and commercialization of setmelanotide in these indications, whichever comes first.

Conditions

• Bardet-Biedl Syndrome (BBS)
• Obesity

Interventions

Type	Name	Description
DRUG	Setmelanotide, administered subcutaneously [SC], once daily.	Dosing based on age, and titrated from starting dose to target dose.

Timeline

• First posted: 2022-01-11
• Last updated: 2022-08-10