Status:
APPROVED_FOR_MARKETING
An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
Lead Sponsor:
Rhythm Pharmaceuticals, Inc.
Conditions:
Bardet-Biedl Syndrome (BBS)
Obesity
Eligibility:
All Genders
6+ years
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 patie...
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 ...
Eligibility Criteria
Inclusion
- Patients who have not taken part in any previous setmelanotide clinical trial must fulfill all of the following study's eligibility criteria:
- 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
Exclusion
- 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.
Key Trial Info
Start Date :
Trial Type :
EXPANDED_ACCESS
End Date :
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT05183802
Last Update
August 10 2022
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