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

  • STATUS
    Recruiting
  • End date
    Aug 27, 2021
  • participants needed
    150
  • sponsor
    Rhythm Pharmaceuticals, Inc.
Updated on 27 February 2021
Investigator
Olga Ohayon
Primary Contact
NIH Hatfield Clinical Research Center (9.7 mi away) Contact
+54 other location
body mass index
other disease
genetic disorders

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.

Details
Condition Smith-Magenis syndrome, Hereditary motor and sensory neuropathy, Alstrom Syndrome, Bardet-Biedl Syndrome, Retinitis Pigmentosa, Neuropathy, Leptin Receptor Deficiency Obesity, Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Alström Syndrome, Alström Syndrome, Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Alström Syndrome, Alström Syndrome, Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder), Pro-opiomelanocortin (POMC) Deficiency Obesity (Heterozygous or Epigenetic), Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder)
Treatment Setmelanotide
Clinical Study IdentifierNCT03013543
SponsorRhythm Pharmaceuticals, Inc.
Last Modified on27 February 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age greater than or equal to 6 yrs?
Gender: Male or Female
Do you have any of these conditions: Hereditary motor and sensory neuropathy or Alström Syndrome or Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder) or Alstrom Syndrome or Neu...?
Do you have any of these conditions: Alström Syndrome or Hereditary motor and sensory neuropathy or Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder) or Retinitis Pigmentosa or...?
Do you have any of these conditions: Smith-Magenis syndrome or Obesity Due to Melanocortin 4 Receptor Deficiency (Disorder) or Hereditary motor and sensory neuropathy or Leptin Receptor D...?
Do you have any of these conditions: Leptin Receptor Deficiency Obesity or Neuropathy or Retinitis Pigmentosa or Alstrom Syndrome or Smith-Magenis syndrome or Bardet-Biedl Syndrome or Obe...?
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 - not currently enrolling new patients
Smith-Magenis Syndrome (SMS)
SH2B1 deficiency obesity - not currently enrolling new patients
Chromosomal rearrangement of the 16p11.2 locus causing obesity - not currently enrolling new patients
CPE compound heterozygous or homozygous deficiency obesity
Leptin deficiency obesity with loss of response to metreleptin
SRC1 deficiency obesity - not currently enrolling new patients
MC4R deficiency obesity - not currently enrolling new patients
Note: The specific genotype for all patients must be reviewed by the Sponsor
prior to study enrollment to confirm that the patient meets Inclusion
Criterion #1. In addition, enrollment of patients in some subgroups may be
prioritized by the Sponsor in order to ensure enrollment of patients with (1)
well described, loss-of-function genetic mutations, (2) a variety of genetic
variants, or (3) genetic variants likely to respond to setmelanotide
\. 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
\. Study participant and/or parent or guardian is able to communicate well
with the Investigator, 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. Female
participants of non-childbearing potential, defined as surgically sterile
(status post hysterectomy, bilateral oophorectomy, or bilateral tubal
ligation), post-menopausal for at least 12 months (and confirmed with a
screening Follicle-Stimulating Hormone [FSH] level in the post-menopausal lab
range), and failure to have achieved menarche, do not require contraception
during the study
\. Male participants with female partners of childbearing potential must
agree to a doublebarrier 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

Exclusion Criteria

Recent intensive (within 2 months) diet and/or exercise regimen with or without the use of weight loss agents including herbal medications 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). Note:Glucagon-like peptide-1 (GLP-1) receptor agonists may be used up to the dose approved for the treatment of diabetes mellitus (e.g., liraglutide up to a daily dose of 1.8 mg) as long as (1) is it not being prescribed for the treatment of obesity, (2) the dose has been stable for at least three months prior to enrollment, (3) the patient has not experienced weight loss during the previous three months, AND (4) the patient intends to keep the dose stable throughout the course of the study
Gastric bypass surgery within the previous six months or any prior gastric bypass surgery resulting in >10% weight loss durably maintained from the baseline pre-operative weight with no evidence of weight regain. Specifically, patients may be considered if surgery was not successful, or resulted in <10% weight loss compared to pre-operative baseline weight or clear evidence of weight regain after an initial response to bariatric surgery. All patients with a history of bariatric surgery must be discussed with and receive approval from the Sponsor prior to enrollment
Diagnosis of schizophrenia, bipolar disorder, personality disorder or other psychiatric disorder(s) that the Investigator believes will interfere significantly with study compliance
Neurocognitive disorders affecting ability to consent will not be
disqualifying as long as an appropriate guardian able to give consent has been
appointed
\. A PHQ-9 score of 15 or any suicidal ideation of type 4 or 5 on the C-SSRS
during Screening, any lifetime history of a suicide attempt, or any suicidal
behavior in the last month. Note: Patients who are unable to complete the
PHQ-9 or C-SSRS due to significant neurocognitive defects may be allowed to
enroll in the study, as long as in the opinion of the Primary Investigator
there are no clinical signs or symptoms of suicidal behavior
\. Current, clinically significant pulmonary, cardiac, or oncologic disease
considered severe enough to interfere with the study and/or confound the
results. Any patient with a potentially clinically significant disease should
be reviewed with the Sponsor to determine eligibility
\. HbA1c >9.0% at Screening
\. History of significant liver disease or abnormal liver tests on Screening
(i.e. > 1.5 x upper limit of normal [ULN] for alanine transaminase [ALT]
aspartate transaminase [AST], alkaline phosphatase, or serum bilirubin). Note
Patients entering the study with SRC1 haploinsufficiency obesity must be
evaluated during the Screening Period for hepatic fibrosis by appropriate
imaging techniques (e.g., transient elastography or magnetic resonance
elastography). Any patient with moderate or greater fibrosis (e.g., the
equivalent of a METAVIR score 2) will be excluded from the study. Note: A
patient with a diagnosis of non-alcoholic fatty liver disease (NAFLD) or non-
alcoholic steatohepatitis (NASH) may be allowed to enroll in the study, after
consultation with the Sponsor. Other significant liver disease, such as
cirrhosis, are exclusionary
\. Glomerular filtration rate (GFR) <30 mL/min at Screening
\. History or close family history (parents or siblings) of skin cancer or
melanoma (not including non-invasive/infiltrative basal or squamous cell
lesion), or patient history of ocular-cutaneous albinism
\. Significant dermatologic findings relating to melanoma or pre-melanoma
skin lesions (excluding non-invasive basal or squamous cell lesion)
determined as part of a comprehensive skin evaluation performed by a qualified
dermatologist during Screening
Any concerning lesions identified during the Screening Period will be biopsied
and results known to be benign prior to enrollment. If the pre-treatment
biopsy results are of concern, the patient may need to be excluded from the
study
\. Patient is, in the opinion of the Study Investigator, not suitable to
participate in the study
\. 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
\. Significant hypersensitivity to any excipient in the study drug
\. Inability to comply with QD injection regimen
\. Females who are breastfeeding or nursing
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