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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03013543
Recruitment Status : Completed
First Posted : January 6, 2017
Last Update Posted : December 27, 2022
Sponsor:
Information provided by (Responsible Party):
Rhythm Pharmaceuticals, Inc.

Study Description
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Brief Summary:
The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight, hunger assessments and other factors in patients with rare genetic disorders of obesity.

Condition or disease Intervention/treatment Phase
Genetic Obesity Obesity Obesity Due to Melanocortin 4 Receptor Deficiency Drug: Setmelanotide Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 213 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
Actual Study Start Date : February 10, 2017
Actual Primary Completion Date : March 1, 2022
Actual Study Completion Date : March 1, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Setmelanotide
Setmelanotide subcutaneous injection once daily
 Drug: Setmelanotide
RM-493 once daily subcutaneous injection
Other Name: RM-493


Outcome Measures
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Primary Outcome Measures :
  1. Effect of Setmelanotide on Body Weight Reduction [ Time Frame: Baseline to 3 months ]
    The proportion of patients in each subgroup of RGDO who achieve at least 5% body weight reduction from baseline at ~3 months treatment with setmelanotide.


Secondary Outcome Measures :
  1. Change and percentage change in body weight [ Time Frame: Baseline to 3 months ]
    Change and percentage change from baseline in body weight

  2. Change in daily most hunger scores [ Time Frame: Baseline to 3 months ]
    Change in the weekly average of the daily hunger score from Baseline at 3 months

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

  4. Mean percent change in BMI [ Time Frame: Baseline to 3 months ]
    Mean percent change in body mass index from baseline at 3 months

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

  6. Mean change in BMI-Z score in patients ≥12 to <18 years old [ Time Frame: Baseline to 3 months ]
    Mean change in BMI-Z score in patients ≥12 to <18 years old from Baseline at 3 months


Eligibility Criteria
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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03013543


Locations
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Sponsors and Collaborators
Rhythm Pharmaceuticals, Inc.
Investigators
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Study Chair: David Meeker, MD Rhythm Pharmaceuticals, Inc.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Rhythm Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03013543    
Other Study ID Numbers: RM-493-014
First Posted: January 6, 2017    Key Record Dates
Last Update Posted: December 27, 2022
Last Verified: December 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rhythm Pharmaceuticals, Inc.:
Pro-opiomelanocortin (POMC) deficiency obesity
LepR deficiency obesity
Smith-Magenis Syndrome
 MC4R deficiency obesity
SRC1 deficiency obesity
SH2B1 deficiency obesity
Additional relevant MeSH terms:
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Genetic Diseases, Inborn
Obesity
Overweight
 Overnutrition
Nutrition Disorders
Body Weight