Ignite Creation Date:
2025-12-25 @ 3:39 AM
Ignite Modification Date:
2026-03-02 @ 3:45 PM
Study NCT ID:
NCT05183802
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2022-08-10
First Post:
2021-11-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
Sponsor:
Rhythm Pharmaceuticals, Inc.
Organization:
Study Overview
Official Title:
An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
Status:
APPROVED_FOR_MARKETING
Status Verified Date:
2022-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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.
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.
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: