Setmelanotide Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity

• Register: ClinicalTrials.gov
• Last refreshed on: 9 January 2023
• Main ID: NCT03013543
• Date of registration: 03/01/2017
• Prospective Registration: Yes
• Primary sponsor: Rhythm Pharmaceuticals, Inc.
• Public title: Setmelanotide Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
• Scientific title: Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
• Date of first enrolment: February 10, 2017
• Target sample size: 213
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03013543
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Canada, France, Germany, Greece, Israel, Netherlands, Spain, United Kingdom, United States

Contacts

• Name: David Meeker, MD
• Affiliation: Rhythm Pharmaceuticals, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patients with the following genotypes and/or clinical assessment:

1. POMC/PCSK1/LEPR heterozygous - not currently enrolling new patients

2. POMC/PCSK1/LEPR compound heterozygous (two different mutations in gene) or
homozygous deficiency obesity

3. POMC/PCSK1/LEPR composite heterozygous (two or more mutations in two or more
genes) deficiency obesity

4. Smith-Magenis Syndrome (SMS)

5. SH2B1 deficiency obesity

6. Chromosomal rearrangement of the 16p11.2 locus causing obesity

7. CPE compound heterozygous or homozygous deficiency obesity

8. Leptin deficiency obesity with loss of response to metreleptin

9. SRC1 deficiency obesity

10. MC4R deficiency obesity

2. Age 6 years and above

3. Obese, defined as Body Mass Index (BMI) = 30 kg/m2 for patients =16 years of age or
BMI= 95th percentile for age and gender for patients 6 up to 16 years of age.

4. Patient and/or parent or guardian is able to understand and comply with the
requirements of the study and is able to understand and sign the written informed
consent/assent

5. Female participants of child-bearing potential must be confirmed non-pregnant, and
agree to use contraception as outlined in the protocol.

6. Male participants with female partners of childbearing potential must agree to a
double barrier method if they become sexually active during the study. Male patients
must not donate sperm during and for 90 days following their participation in the
study.

Key Exclusion Criteria:

1. Recent intensive (within 2 months) diet and/or exercise regimen with or without the
use of weight loss agents that has resulted in > 2% weight loss.

2. Use of any medication that is approved to treat obesity within three months of first
dose of study drug (e.g., orlistat, lorcaserin, phentermine-topiramate,
naltrexone-bupropion).

3. Gastric bypass surgery within the previous six months or any prior gastric bypass
surgery resulting in >10% weight loss durably maintained

4. Diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive
disorder, or other psychiatric disorder(s)

5. Suicidal ideation, attempt or behavior

6. Clinically significant pulmonary, cardiac, or oncologic disease

7. HbA1c >9.0% at Screening

8. History of significant liver disease

9. Glomerular filtration rate (GFR) <30 mL/min at Screening.

10. History or close family history of melanoma or patient history of oculocutaneous
albinism

11. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.

12. Participation in any clinical study with an investigational drug/device within 3
months prior to the first day of dosing.

13. Patients previously enrolled in a clinical study involving setmelanotide or any
previous exposure to setmelanotide.

14. Inability to comply with QD injection regimen.

15. Females who are breastfeeding or nursing.

Other protocol defined Inclusion/Exclusion criteria may apply.



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Genetic Obesity

Obesity

Obesity Due to Melanocortin 4 Receptor Deficiency

Intervention(s)

Drug: Setmelanotide

Primary Outcome(s)

Effect of Setmelanotide on Body Weight Reduction [Time Frame: Baseline to 3 months]

Secondary Outcome(s)

Change and percentage change in body weight [Time Frame: Baseline to 3 months]

Mean change in BMI-Z score in patients <12 years old [Time Frame: Baseline to 3 months]

Change in daily most hunger scores [Time Frame: Baseline to 3 months]

Mean change in BMI-Z score in patients =12 to <18 years old [Time Frame: Baseline to 3 months]

Change in waist circumference [Time Frame: Baseline to 3 months]

Mean percent change in BMI [Time Frame: Baseline to 3 months]

Secondary ID(s)

RM-493-014

Source(s) of Monetary Support

Please refer to primary and secondary sponsors