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

Primary Purpose

Bardet-Biedl Syndrome (BBS), Obesity

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
Setmelanotide, administered subcutaneously [SC], once daily.
Sponsored by
Rhythm Pharmaceuticals, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Bardet-Biedl Syndrome (BBS)

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All Sexes

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.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    November 8, 2021
    Last Updated
    August 8, 2022
    Sponsor
    Rhythm Pharmaceuticals, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05183802
    Brief Title
    An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
    Official Title
    An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2022
    Overall Recruitment Status
    Approved for marketing
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Rhythm Pharmaceuticals, Inc.

    4. Oversight

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Bardet-Biedl Syndrome (BBS), Obesity

    7. Study Design

    8. Arms, Groups, and Interventions

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

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    6 Years
    Eligibility Criteria
    Patients who have not taken part in any previous setmelanotide clinical trial must fulfill all of the following study's eligibility criteria: Inclusion 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 Criteria: 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.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Linda Shapiro, M.D., Ph.D.
    Organizational Affiliation
    Chief Medical Officer, Rhythm Pharmaceuticals
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Links:
    URL
    https://www.rhythmtx.com/policy-on-expanded-access-to-investigational-drugs/
    Description
    Policy on Expanded Access to Investigational Drugs

    Learn more about this trial

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

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