Setmelanotide Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
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|ClinicalTrials.gov Identifier: NCT03013543|
Recruitment Status : Completed
First Posted : January 6, 2017
Last Update Posted : December 27, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Genetic Obesity Obesity Obesity Due to Melanocortin 4 Receptor Deficiency||Drug: Setmelanotide||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||213 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Setmelanotide subcutaneous injection once daily
RM-493 once daily subcutaneous injection
Other Name: RM-493
- 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.
- Change and percentage change in body weight [ Time Frame: Baseline to 3 months ]Change and percentage change from baseline in body weight
- 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
- Change in waist circumference [ Time Frame: Baseline to 3 months ]Change from baseline in waist circumference
- Mean percent change in BMI [ Time Frame: Baseline to 3 months ]Mean percent change in body mass index from baseline at 3 months
- 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
- 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
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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|
Patients with the following genotypes and/or clinical assessment:
- POMC/PCSK1/LEPR heterozygous - not currently enrolling new patients
- 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
- Smith-Magenis Syndrome (SMS)
- SH2B1 deficiency obesity
- Chromosomal rearrangement of the 16p11.2 locus causing obesity
- 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 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.
- 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
- Female participants of child-bearing 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 patients 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
- HbA1c >9.0% at Screening
- History of significant liver disease
- Glomerular filtration rate (GFR) <30 mL/min at Screening.
- History or close family history of melanoma or patient 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.
- Patients 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.
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
|Study Chair:||David Meeker, MD||Rhythm Pharmaceuticals, Inc.|
|Responsible Party:||Rhythm Pharmaceuticals, Inc.|
|Other Study ID Numbers:||
|First Posted:||January 6, 2017 Key Record Dates|
|Last Update Posted:||December 27, 2022|
|Last Verified:||December 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Pro-opiomelanocortin (POMC) deficiency obesity
LepR deficiency obesity
MC4R deficiency obesity
SRC1 deficiency obesity
SH2B1 deficiency obesity
Genetic Diseases, Inborn