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

August 8, 2022 updated by: Rhythm Pharmaceuticals, Inc.
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.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

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.

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Rhythm Pharmaceuticals, Inc.

Investigators

  • Study Chair: Linda Shapiro, M.D., Ph.D., Chief Medical Officer, Rhythm Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

November 8, 2021

First Submitted That Met QC Criteria

January 7, 2022

First Posted (ACTUAL)

January 11, 2022

Study Record Updates

Last Update Posted (ACTUAL)

August 10, 2022

Last Update Submitted That Met QC Criteria

August 8, 2022

Last Verified

August 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RM-493-036

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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