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Completed

NCT Number: NCT03013543

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

The purpose of the study was to determine the effect of setmelanotide (RM-493) on weight, hunger assessments, and other factors in participants with rare genetic disorders of obesity.

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Key information

Conditions

Genetic Obesity Abnormalities, Multiple Body Weight Chromosome Disorders Chronobiology Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Nervous System Diseases Nutrition Disorders Nutritional and Metabolic Diseases Obesity Obesity Due to Melanocortin 4 Receptor Deficiency Overnutrition Overweight Pathological Conditions, Signs and Symptoms Signs and Symptoms Smith-Magenis Syndrome

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Alberta, Edmonton, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants with the following genotypes and/or clinical assessment:
  • POMC/PCSK1/LEPR heterozygous - not currently enrolling new participants
  • POMC/PCSK1/LEPR compound heterozygous (two different mutations in gene) or homozygous deficiency obesity
  • POMC/PCSK1/LEPR composite heterozygous (two or more mutations in two or more genes) deficiency obesity
  • SMS
  • SH2B1 deficiency obesity
  • Chromosomal rearrangement of the 16p11.2 locus causing obesity
  • Carboxypeptidase E (CPE) compound heterozygous or homozygous deficiency obesity
  • Leptin deficiency obesity with loss of response to metreleptin
  • SRC1 deficiency obesity
  • MC4R deficiency obesity
  • Age 6 years and above
  • Obese, defined as Body Mass Index (BMI) ≥ 30 kilogram per meter square (kg/m^2) for participants ≥16 years of age or BMI≥ 95th percentile for age and gender for participants 6 up to 16 years of age.
  • Participant 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
  • Female participants of childbearing potential must be confirmed non-pregnant, and agree to use contraception as outlined in the protocol.
  • Male participants with female partners of childbearing potential must agree to a double barrier method if they become sexually active during the study. Male participants must not donate sperm during and for 90 days following their participation in the study.

Key Exclusion Criteria:

  • 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.
  • 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).
  • Gastric bypass surgery within the previous six months or any prior gastric bypass surgery resulting in >10% weight loss durably maintained
  • Diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s)
  • Suicidal ideation, attempt or behavior
  • Clinically significant pulmonary, cardiac, or oncologic disease
  • hemoglobin A1c (HbA1c) > 9.0% at Screening
  • History of significant liver disease
  • Glomerular filtration rate (GFR) < 30 milliliter/minute (mL/min) at Screening.
  • History or close family history of melanoma or participant history of oculocutaneous albinism
  • Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
  • Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
  • Participants previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
  • Inability to comply with QD injection regimen.
  • Females who are breastfeeding or nursing.

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

Setmelanotide

Drug

RM-493 QD SC injection

Other names: RM-493

Primary outcomes

  1. Number of Participants With ≥ 5% Reduction in Body Weight From Baseline After 3 Months of Setmelanotide Treatment

    Time frame: Baseline to Month 3

Secondary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Time frame: From first dose up to Month 16

    An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE (also referred to as an adverse experience) could be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. TEAEs were defined as AEs reported after dosing on Day 1.

  2. Change From Baseline in Body Weight After 3 Months of Setmelanotide Treatment

    Time frame: Baseline, Month 3

  3. Percent Change From Baseline in Body Weight After 3 Months of Setmelanotide Treatment

    Time frame: Baseline, Month 3

  4. Change From Baseline in Daily Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged ≥ 12 Years

    Time frame: Baseline, Month 3

    The mean change in daily hunger questionnaire scores for participants ≥ 12 years of age with obesity in treatment with setmelanotide was evaluated. On the Daily Hunger Questionnaire, each of the 3 items (average hunger in the last 24 hours, most/worst hunger in the last 24 hours, and morning hunger) was assessed daily and scored separately using a numeric rating score for each from 0 to 10, with 0 = not hungry at all and 10 = hungriest possible.

  5. Change From Baseline in Daily Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged < 12 Years

    Time frame: Baseline, Month 3

    The mean change in daily hunger questionnaire scores for participants < 12 years of age with obesity in treatment with setmelanotide was evaluated. Hunger was assessed daily using a Daily Hunger Questionnaire with a pictorial (smiley face) version of the Likert rating scale with scores ranged from 0 to 4, with 0 = not hungry at all and 4 = hungriest possible.

  6. Number of Participants With Shifts From Baseline in Global Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged ≥ 12 Years

    Time frame: Baseline, Month 3

    For participants ≥ 12 years of age, the following question was asked using the Global Hunger Questionnaire: Overall, how would you rate the hunger you experience now? Possible responses were: No hunger; Mild hunger; Moderate hunger; Severe hunger; and Not answered.

  7. Number of Participants With Shifts From Baseline in Global Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged < 12 Years

    Time frame: Baseline, Month 3

    For participants < 12 years of age, the following question was asked to parents or caregivers of participants using the Global Hunger Questionnaire: How hungry is your child acting now? Possible responses were: Not hungry at all; A little hungry; Moderately hungry; Extremely hungry; and Not answered.

  8. Percent Change From Baseline in Waist Circumference After 3 Months of Setmelanotide Treatment

    Time frame: Baseline, Month 3

    Waist circumference (cm) was measured according to the National Heart, Lung, and Blood Institute (NHLBI) criteria. All measurements were single measures. Waist circumference was measured when participants were in fasting condition and at approximately the same time at each visit.

Sponsors and collaborators

Lead sponsor

Rhythm Pharmaceuticals, Inc.

Industry

Registry information

Official study title

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity

Important dates

Study start
2017
Primary completion
2022
Study completion
2022
First posted
Jan 6, 2017
Registry last updated
Aug 18, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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