NL-OMON49642已完成2 期
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity - Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with obesity
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •1. Patients with the following genotypes and/or clinical assessment:
- •a. POMC/PCSK1/LEPR heterozygous
- •b. POMC/PCSK1/LEPR compound heterozygous (two different mutations in gene) or
- •homozygous deficiency obesity
- •c. POMC/PCSK1/LEPR composite heterozygous (two or more mutations in two or more
- •genes) deficiency obesity
- •d. Smith-Magenis Syndrome (SMS)
- •e. SH2B1 deficiency obesity
- •f. Chromosomal rearrangement of the 16p11.2 locus causing obesity
- •g. CPE compound heterozygous or homozygous deficiency obesity
- •h. Leptin deficiency obesity with loss of response to metreleptin
- •i. SRC1 deficiency obesity
- •j. MC4R deficiency obesity
- •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.
- •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
- •4. 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.
- •5. 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.
- •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.
排除标准
- •1. 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.
- •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). 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.
- •3. 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.
- •4. 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.
- •5. 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.
- •6. 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.
- •7. HbA1c >9.0% at Screening
- •8. 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 S
研究者
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