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

NCTNCT05183802Other idRM-493-036

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Approved for marketing
Phase: Phase not stated
Enrollment: Not stated
Sponsor: Rhythm Pharmaceuticals, Inc. · Industry
Design: Not stated
Countries: Not stated
Start: Not stated
Primary completion: Not stated
Completion: Not stated
First posted: 11 Jan 2022
Last update posted: 10 Aug 2022
Conditions: Bardet-Biedl Syndrome (BBS) · Obesity

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

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

Arms0

The registry lists no arms for this trial.

Primary outcomes0

Outcomes the sponsor listed as primary.

The registry lists no primary outcome.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: from 6 Years
Healthy volunteers: Not stated

Inclusion and exclusion criteria, as posted

Patients who have not taken part in any previous setmelanotide clinical trial must fulfill all of the following study's eligibility criteria: Inclusion Criteria: 1. Clinical diagnosis of BBS 2. Males and females aged ≥6 years 3. 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 4. Female participants of child-bearing potential must be confirmed as non-pregnant 5. Patients with significant neuropsychiatric disorders such as depression should be carefully evaluated regarding the benefit/risk ratio for the use of setmelanotide 6. Patients unable to enroll in another clinical trial with setmelanotide as determined by the treating physician Exclusion Criteria: 1. Moderate to severe renal dysfunction defined as a glomerular filtration rate (GFR) \<60 mL/min/1.73m2 2. 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 3. 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.

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