A Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway

• Status: Enrolling by invitation
• Trial end date: 2022-11-01
• Target enrollment: 0
• Participant gender: All

Summary

Phase 3 trial to compare the weekly and daily formulations of setmelanotide in patients with genetic defects in the melanocortin-4 receptor pathway.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Rhythm Pharmaceuticals, Inc.
• Treatments: alpha-MSH

Criteria

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.

Exclusion Criteria (partial list):

• 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.

• Patient is not suitable, in the opinion of the Investigator, to participate in the study.

• 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.