Skip to main content
MedPath Trial logoMedPath TrialMedPath Trial Home
Clinical Trials/NL-OMON54472
NL-OMON54472RecruitingPhase 2

A 2-Stage (Open-Label Followed by Randomized Double-Blind, Placebo-Controlled Stage), Phase 2 Trial of Setmelanotide in Patients with Specific Gene Variants in the Melanocortin-4 Receptor Pathway - RM-493-034

Rhythm Pharmaceuticals, Inc.0 sites11 target enrollmentStarted: TBDLast updated:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
Conditions

Trial Snapshot

Phase
Phase 2
Status
Recruiting
Enrollment
11
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional

Eligibility Criteria

Ages
2 to 64 (—)

Inclusion Criteria

  • •1. Patients must have a pre-identified genetic variant in an established MC4R
  • •pathway gene that contributes to obesity
  • •Note: Genetic testing requirements and a list of genes which have variants that
  • •are eligible for enrollment into the trial are provided in Appendix 1 of the
  • •2. Patients between the ages of 6 and 65, inclusive, at the time of signing
  • •Informed Consent or Assent.
  • •3. Patients with obesity, defined as BMI >=40 kg/m2 for patients >=18 years of
  • •age or BMI >=97th percentile for age and gender for patients 6 to <18 years of
  • •age based on the US Centers for Disease Control and Prevention criteria.
  • •4. Patient and/or parent or guardian is able to communicate well with the
  • •Investigator, to understand and comply with the requirements of the trial
  • •(including the QD injection regimen and all other trial procedures) and is able
  • •to understand and sign the written informed consent/assent. Patients who are
  • •unable to comply with all trial procedures due to cognitive limitations or any
  • •other reason should not be enrolled.
  • •5. Patient must meet one of the following requirements:
  • •Female participants of childbearing potential, defined as fertile, following
  • •menarche and until becoming post-menopausal unless permanently sterile
  • •(hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), must be
  • •confirmed non-pregnant and agree to use a highly effective form of
  • •contraception throughout the trial and for 90 days following the trial.
  • •Highly effective forms of contraception are detailed below and in Section 8.9.7
  • •of the protocol:
  • •Combined (estrogen and progestin) hormonal contraception associated with
  • •inhibition of ovulation (i.e., oral, intravaginal, or transdermal)
  • •Progestin-only hormonal contraception associated with inhibition of ovulation
  • •(oral, implantable, or injectable)
  • •Intrauterine device (IUD)
  • •Intrauterine hormone-releasing system
  • •Bilateral tubal occlusion
  • •Vasectomy/vasectomized partner (provided that the vasectomized partner is the
  • •sole sexual partner of the female participant, and the vasectomized partner has
  • •received medical assessment of surgical success)
  • •Sexual abstinence, only if it is the preferred and usual lifestyle of the
  • •Female participants of non-childbearing potential, defined as: permanently
  • •sterile (status post hysterectomy, bilateral oophorectomy, or bilateral
  • •salpingectomy), or post-menopausal for at least 12 months (and confirmed with a
  • •screening follicle-stimulating hormone level in the post-menopausal lab range)
  • •and do not require contraception during the trial.
  • •Younger female patients who have not achieved sexual maturity at trial entry
  • •will be assessed for Tanner staging and required to comply with contraception
  • •requirements at first menarche.
  • •Male participants with female partners of childbearing potential must agree to
  • •use a highly effective method contraception if they become sexually active
  • •during the trial or within 90 days following their participation in the trial.
  • •Male patients must also not donate sperm during and for 90 days following their
  • •participation in the trial.
  • •6. Symptoms or behaviors of hyperphagia persistent during the patient*s life,
  • •including manifestations in childhood, as determined by the Investigator at

Exclusion Criteria

  • •1. Patients with the following genetic variants: biallelic Bardet-Biedl
  • •Syndrome (BBS); biallelic Alström Syndrome 1 (ALMS1); homozygous, heterozygous,
  • •or compound heterozygous variants in MC4R, POMC, PCSK1, LEPR, nuclear receptor
  • •coactivator 1 (NCOA1; steroid receptor coactivator-1 [SRC1]) or SRC homology 2
  • •B adapter protein 1 (SH2B1) genes as well as 16p11.2 chromosomal deletions that
  • •include the SH2B1 gene.
  • •2. Weight loss >2% in the previous 3 months.
  • •Patients will not be excluded for using regimens for weight maintenance or to
  • •prevent weight gain, such as dietary and/or exercise regimens, or medications,
  • •supplements or herbal treatments (e.g., orlistat, lorcaserin, phentermine,
  • •topiramate, naltrexone, bupropion, glucagon-like peptide-1 [GLP-1] receptor
  • •agonists, etc.), provided:
  • •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,
  • •the patient intends to keep the regimen and/or dose stable throughout the
  • •course of the trial.
  • •3. Bariatric surgery or procedure (e.g., gastric bypass/band/sleeve, duodenal
  • •switch, gastric balloon, intestinal barrier, etc.) within the last 6 months.
  • •All patients with a history of bariatric surgery or procedures must be
  • •discussed with, and receive approval from, the Sponsor prior to enrollment.
  • •4. Documented diagnosis of current unstable major psychiatric disorder(s)
  • •(e.g., major depressive disorder, bipolar disorder, schizophrenia, etc.) or
  • •documented worsening psychiatric condition that required changes in treatment
  • •regimen within the previous 2 years, or other psychiatric related risks that
  • •the Investigator believes may interfere with trial compliance or patient safety.
  • •5. Clinically significant depression or suicidality, as defined by: any
  • •suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale
  • •(C-SSRS) during Screening, any suicide attempt during the patient*s lifetime,
  • •any suicidal behavior in the last month, or a Patient Health Questionnaire-9
  • •(PHQ-9) score of >=15 during Screening process.
  • •Note: Patients who are unable to complete the PHQ-9 or C-SSRS due to
  • •significant neurocognitive impairment may be enrolled in the trial provided
  • •that there are no clinical signs or symptoms of significant depression or
  • •suicidal behavior in the opinion of the Investigator.
  • •6. Current, clinically significant pulmonary, cardiac, endocrine/metabolic,
  • •hepatic, or oncologic disease considered severe enough to interfere with the
  • •trial and/or confound the results. Any patient with a potentially clinically
  • •significant disease should be reviewed with the Sponsor to determine
  • •eligibility.
  • •7. Significant features of, or meeting the diagnostic criteria for, a genetic
  • •syndrome that is associated with obesity.
  • •Note: Although some of the genetic variants that are eligible to be enrolled
  • •into this trial are associated with specific syndromes, the intent of this
  • •trial is not to enroll children with significant cognitive impairment or other
  • •significant co-morbidities. Patients with eligible genetic variants, but who
  • •otherwise do not exhibit the syndrome, are eligible for enrollment.
  • •8. HbA1C >10.0% at Screening.
  • •9. History of significant liver disease other than non-alcoholic fatty liver
  • •disease (NAFLD) or

Investigators

Similar Trials

Active, not recruiting
Phase 1
Study of setmelanotide in patients with obseity with specific genetic defectsSpecific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certainmessenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
EUCTR2021-002855-12-NLRhythm Pharmaceuticals Inc.165
Active, not recruiting
Phase 1
Study of setmelanotide in obese patients with specific genetic defectsSpecific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certainmessenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
EUCTR2021-002855-12-DERhythm Pharmaceuticals Inc.165
Active, not recruiting
Phase 1
Study of setmelanotide in patients with obesity with specific genetic defectsSpecific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certainmessenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
EUCTR2021-002855-12-GRRhythm Pharmaceuticals Inc.165
Active, not recruiting
Phase 1
Study of setmelanotide in obese patients with specific genetic defectsSpecific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certain messenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
EUCTR2021-002855-12-ESRhythm Pharmaceuticals Inc.500
Active, not recruiting
Phase 1
Study of setmelanotide in obese patients with specific genetic defectsSpecific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certainmessenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
EUCTR2021-002855-12-FRRhythm Pharmaceuticals Inc.500