ong Term Extension Trial of setmelanotide for patients who havecompleted a trial of Setmelanotide for the treatment of obesity associated withgenetic defects.
- Conditions
- Patients with obesity caused by genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway. Some rare genetic disorders include, but not limited to:obesity, including: Leptin Receptor (LepR) Deficiency Obesity POMC Heterozygous Deficiency Obesity PCSK deficiency obesity Bardet-Biedl syndromeAlström syndromeTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2017-005006-35-GR
- Lead Sponsor
- Rhythm Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 220
1. Patients aged 2 or older that have completed participation on active drug and demonstrated adequate safety and meaningful clinical benefit (efficacy) in a previous setmelanotide study for obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.Meaningful clinical benefit is defined as follows:
• Patients up to 18 years of age that have completed participation on
active drug and demonstrated adequate safety and at least 2% body
mass index (BMI) reduction or reduction in BMI Z score of 0.2
compared to baseline.
• Patients over 18 years of age should show reduction of 3% weight
compared to baseline.
2. 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 to understand and sign the written informed consent/assent.
The patient must assent/consent to participate in the trial.
3. Patient must meet one of the following requirements:
• If a female of childbearing potential, defined as fertile, following
menarche and until becoming post-menopausal unless permanently
sterile (hysterectomy, bilateral salpingectomy, or bilateral
oophorectomy), must use a highly effective form of contraception as
outlined in the protocol
• If a female 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 FSH level in the post-menopausal laboratory
range), contraception is not required during the study. - Any female
participant in this latter category of having failed to Younger female
patients who have not reached menarche upon study entry, will be
assessed for Tanner staging and at first menarche will be required to
comply with contraception requirements and pregnancy testing as
outlined in the protocol.
• If a male with female partner(s) of childbearing potential, must agree
to a double barrier method if they become sexually active during the
study. Furthermore, male patients must not donate sperm during and for
90 days following their participation in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 83
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3
1.Pregnant and/or breastfeeding women
2.Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion), determined as part of a screening comprehensive skin evaluation performed by a qualified dermatologist. 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.
3. Patient is, in the opinion of the Study Investigator, not suitable to participate in the study.
In addition, any patient who experiences a gap in treatment of at least one month between completing the Index study and Screening for this study, should have the following exclusion criteria evaluated:
4. Current, clinically significant disease, if severe enough to interfere with the study and/or would confound the results. Any such patients should be discussed with the Sponsor prior to inclusion
5. Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the investigator believes will interfere significantly with study compliance.
6. A Patient Health Questionnaire-9 (PHQ-9) score of = 15.
7. Any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale (CSSRS). 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 enrolled in the study, as long as in the opinion of the Primary Investigator there are no clinical signs or symptoms of suicidal behavior.
8. History of significant liver disease or liver injury, or a current liver assessment due to abnormal liver tests (as indicated by abnormal liver function tests, alanine transaminase [ALT], aspartate transaminase [AST], alkaline phosphatase, or serum bilirubin >1.5× the upper limit of normal [ULN] for any of these tests) for an etiology other than nonalcoholic fatty liver disease (NAFLD). Thus, any underlying etiology besides NAFLD, including diagnosed non-alcoholic steatohepatitis (NASH), other causes of hepatitis, or history of hepatic cirrhosis is exclusionary, but the presence of NAFLD is not be exclusionary.
9. Moderate to severe renal dysfunction as defined by a glomerular filtration rate (GFR)
<30 mL/min/1.73 m2.
10. 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method