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Open-Label Study of Setmelanotide in Hypothalamic 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04725240
Recruitment Status : Recruiting
First Posted : January 26, 2021
Last Update Posted : November 26, 2021
Sponsor:
Information provided by (Responsible Party):
Rhythm Pharmaceuticals, Inc.

Brief Summary:
Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in patients with hypothalamic obesity (HO).

Condition or disease Intervention/treatment Phase
Hypothalamic Obesity Drug: Setmelanotide Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-Label 20-Week Study to Evaluate the Safety and Efficacy of Setmelanotide in Subjects With Hypothalamic Obesity
Actual Study Start Date : June 7, 2021
Estimated Primary Completion Date : March 31, 2022
Estimated Study Completion Date : March 31, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Setmelanotide
Dosage: 1.0, 2.0, 3.0 mg daily for patients 6 to <16 years of age, and 2.0 to 3.0 mg daily for patients ≥16 years of age. All patients will receive study treatment for 16 weeks.
Drug: Setmelanotide
Investigational product: Setmelanotide for Subcutaneous (SC) injection.




Primary Outcome Measures :
  1. Change in Body Weight Related Measure [ Time Frame: 16 weeks ]
    Proportion of patients with ≥5% reduction from baseline in BMI after 16 weeks of setmelanotide treatment compared to a historic control of <5% in this patient population.


Secondary Outcome Measures :
  1. Change in body weight related composite measures [ Time Frame: 16 weeks ]
    Composite proportion of patients aged ≥6 to <18 years with ≥0.2 reduction of BMI Z-score and patients aged ≥18 years with 5% reduction of body weight from baseline after 16 weeks of setmelanotide.

  2. Change in body weight related measure in children and adolescents [ Time Frame: 16 weeks ]
    Proportion of patients aged ≥6 to <18 years with ≥0.2 reduction of BMI Z-score from baseline after 16 weeks of setmelanotide.

  3. Change in body weight related measure in adults [ Time Frame: 16 weeks ]
    Proportion of patients aged ≥18 years with ≥5% reduction of body weight from baseline after 16 weeks of setmelanotide.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Patients must meet all of the following criteria to be eligible for study participation:

  • Patient has documented evidence of HO, including:

    • Recent evidence of hypothalamic injury on magnetic resonance imaging (MRI); AND
    • Diagnosis of craniopharyngioma or other non-malignant brain tumor affecting the hypothalamic region; AND
    • Has undergone surgery, or chemotherapy, or radiation ≥6 months and ≤15 years before Screening.
  • Patient has either unilateral hypothalamic lesions or bilateral hypothalamic lesions, as assessed by MRI.
  • Aged 6 to 40 years.
  • Obesity, documented by a BMI ≥35 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and gender for patients 6 to <18 years of age.
  • Documented increase in BMI (change from pre-surgery baseline in BMI z-score ≥0.2 for patients <18 years of age or BMI >5% for patients >18 years of age) either during the first 6 months following surgery or within 1 year before surgery AND still present at Screening.
  • More than 6 months after the end of post-tumor treatment, including chemotherapy, surgery, or radiation.
  • Highly effective contraception throughout the study and for 90 days following the study.
  • Ability to communicate well with the Investigator, understand and comply with the requirements of the study, and understand and sign the written informed consent, or, for patients aged <18 years, a parent/legal guardian that can sign.
  • If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening.

Exclusion Criteria:

Patients meeting any of the following criteria are not eligible for study participation:

  • Weight gain >5% in the previous 3 months.
  • Weight loss ≥2% in the previous 3 months.
  • Note: Dietary and/or exercise regimens, with or without the use of medications, supplements, or herbal treatments associated with weight loss are allowed if:
  • the regimen and/or dose has been stable for at least 3 months prior to randomization
  • the patient has not experienced weight loss ≥2% during the previous 3 months, AND
  • the patient intends to keep the regimen and/or dose stable throughout the course of the study.
  • Bariatric surgery or procedure within the last 6 months.
  • Diagnosis of severe psychiatric disorders
  • HbA1c >10.0% at Screening.
  • Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results.
  • Glomerular filtration rate (GFR) <30mL/min/1.73m^2 during Screening.
  • Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
  • History or close family history (parents or siblings) of skin cancer or melanoma
  • Participation in any clinical study with an investigational drug/device within 3 months, prior to the first setmelanotide dose.
  • Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
  • Inability to comply with once daily (QD) injection regimen.
  • Pregnant and/or breastfeeding, or desiring to become pregnant during this trial.
  • Cognitive impairment that, in the Investigator's opinion, precludes participation to the study and completions of study procedures or questionnaires.
  • Patient is, in Investigator's opinion, otherwise not suitable to participate in the study.

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): NCT04725240


Contacts
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Contact: Sarah Pilley, BS/MS 857-264-4281 spilley@rhythmtx.com
Contact: Emma Jost-Price, BA ejostprice@rhythmtx.com

Locations
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United States, California
Rady Children's Hospital Recruiting
San Diego, California, United States, 92123
Contact: Marla Hashiguchi    858-966-8940    mhashiguchi@rchsd.org.com   
Principal Investigator: Michael Gottschalk, MD         
United States, Florida
University of Florida Not yet recruiting
Gainesville, Florida, United States, 32610-0296
Contact: Rebeccah Mercado    407-383-8919    rebeccahmercado@ufl.edu   
Principal Investigator: Jennifer Miller, MD         
United States, Minnesota
Children's Minnesota Recruiting
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus    651-220-5730    brittany.machus@childrensmn.org   
Principal Investigator: Jennifer Abuzzahab, MD         
United States, Tennessee
Vanderbilt University School of Medicine Recruiting
Nashville, Tennessee, United States, 37232
Contact: Laura Maynard    615-875-4274    laura.e.maynard@vumc.org   
Principal Investigator: Ashley Shoemaker, MD         
United States, Washington
Seattle Children's Research Institute Recruiting
Seattle, Washington, United States, 98101
Contact: Sue Kearns    206-987-1758    sue.kearns@seattlechildrens.org   
Principal Investigator: Christian Roth, MD         
Sponsors and Collaborators
Rhythm Pharmaceuticals, Inc.
Investigators
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Study Chair: Linda Shapiro, MD, PhD Rhythm Pharmaceuticals, Inc.
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Responsible Party: Rhythm Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04725240    
Other Study ID Numbers: RM-493-030
First Posted: January 26, 2021    Key Record Dates
Last Update Posted: November 26, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Obesity
Overnutrition
Nutrition Disorders
Overweight
Body Weight