Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Patients With Specific Gene Defects in the MC4R Pathway
Bardet-Biedl Syndrome, POMC Deficiency
About this trial
This is an interventional treatment trial for Bardet-Biedl Syndrome focused on measuring Melanocortin-4 Receptor Pathway, Genetic Obesity, Hunger, Hyperphagia
Eligibility Criteria
Key Inclusion Criteria:
- Biallelic or heterozygous POMC/PCSK1 or LEPR (PPL) genetic variants or Bardet-Biedl syndrome (BBS), for which they are being treated with QD setmelanotide.
- 6 years or older at screening.
- Taking the setmelanotide QD formulation for at least 6 months in the RM-493-022 study with acceptable safety and tolerability, and dose level.
- Patient and/or parent or guardian is able to communicate well with the Investigator, to understand and comply with the requirements of the study and is able to understand and sign the written informed consent/assent.
- Use of a highly effective form of contraception throughout the study and for 90 days following the study.
Key Exclusion Criteria:
- HbA1C >9.0% at screening.
- Anti-obesity medications within 3 months prior to starting the Run-in Period.
- History of significant liver disease or liver injury.
- Glomerular filtration rate <30 mL/min.
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
- Major psychiatric disorders.
- Any suicidal ideation or behavior, or any lifetime history of a suicide attempt.
- Significant hypersensitivity to any excipient in the study drug.
- Inability to comply with the QW and QD injection regimens.
- Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing, with the exception of a setmelanotide clinical trial.
Other protocol defined Inclusion/Exclusion criteria may apply.
Sites / Locations
- Honor Health Research Institute
- Marshfield Clinic Research Institute
- Alberta Health Services
- Charité - Universitätsmedizin Berlin, Campus Virchow-Klinikum
- Erasmus MC
- UPR Medical Sciences Campus
- Addenbrooke's Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Experimental
Experimental
Experimental
Experimental
Placebo Comparator
Placebo Comparator
Setmelanotide subcutaneous injection Weekly 20 mg
Setmelanotide subcutaneous injection Weekly 30 mg
Setmelanotide subcutaneous injection Daily 2 mg
Setmelanotide subcutaneous injection Daily 3 mg
Placebo subcutaneous injection Daily
Placebo subcutaneous injection Weekly
Patients on 2mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period, and all will be assigned to this arm in the open label period.
Patients on 3mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period, and all will be assigned to this arm in the open label period.
Patients on 2mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period.
Patients on 3mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period.
Patients will be randomized 1:1 to receive either QD or QW placebo during the double blind period (and setmelanotide in the other formulation).
Patients will be randomized 1:1 to receive either QD or QW placebo during the double blind period (and setmelanotide in the other formulation).