Bardet-Biedl Syndrome (BBS)NCT05183802
An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
A study testing Setmelanotide, administered subcutaneously [SC], once daily. in people with Bardet-Biedl Syndrome (BBS) and Obesity, run by Rhythm Pharmaceuticals, Inc..
Trial 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. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Bardet-Biedl Syndrome (BBS), Obesity
- What is being tested?
- Setmelanotide, administered subcutaneously [SC], once daily.
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
About this study
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.
Read the 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.
- Conditions
- Bardet-Biedl Syndrome (BBS), Obesity
- Being tested
- Setmelanotide, administered subcutaneously [SC], once daily.
- Medical subject headings
- Bardet-Biedl Syndrome, Obesity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Setmelanotide, administered subcutaneously [SC], once daily.Drug
Dosing based on age, and titrated from starting dose to target dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bardet-Biedl Syndrome (BBS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Linda Shapiro, M.D., Ph.D., Study chair, Chief Medical Officer, Rhythm Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05183802
- Sponsor's protocol ID
- RM-493-036
- Status last verified
- Aug 2022
- First submitted
- 8 Nov 2021
- Last updated
- 10 Aug 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
- ClinicalTrials.gov ID
- NCT05183802
- Acronym / sponsor ID
- RM-493-036
- Conditions
- Bardet-Biedl Syndrome (BBS), Obesity
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 11 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Setmelanotide, administered subcutaneously [SC], once daily.Dosing based on age, and titrated from starting dose to target dose. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2022.
- Policy on Expanded Access to Investigational DrugsLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Linda Shapiro, M.D., Ph.D. study chair
Chief Medical Officer, Rhythm Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A study testing Setmelanotide, administered subcutaneously [SC], once daily. in people with Bardet-Biedl Syndrome (BBS) and Obesity, run by Rhythm Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Aug 2022.