ong Term Extension Trial of setmelanotide (RM-493) for patients who have completed a trial of Setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway

Phase 1

• Conditions: Treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2017-005006-35-FR
• Lead Sponsor: Rhythm Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-03-20
• Last Update Posted: 1 February 2020

Recruitment & Eligibility

• Status: A
• Sex: Not specified
• Target Recruitment: 100

Inclusion Criteria

1. Patients aged 6 or older that have completed participation on active drug and demonstrated adequate safety in a previous setmelanotide study for obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.
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. Female participants of child-bearing potential must 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), postmenopausal for at least 12 months (and confirmed with a screening FSH level in the postmenopausal lab range), or have delayed pubertal development and failure to have achieved menarche, do not require contraception during the study. Any female participant in this latter category of having failed to reach menarche upon study entry and who now suspects this status may have changed should promptly inform the investigator and undergo pregnancy testing.

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 80
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. 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.
2. Pregnant and/or breastfeeding women are excluded.
3. 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.
4. A Patient Health Questionnaire-9 (PHQ-9) score of = 15.
5. Any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS). Any lifetime
history of a suicide attempt, or any suicidal behavior in the last month.
6. Current, severe stable restrictive or obstructive lung disease as a consequence of extreme obesity, evidence of significant heart failure (NYHA Class 3 or greater), or oncologic disease, if these were 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.
7. History of significant liver disease or liver injury, or current liver assessment for a cause of abnormal liver tests [as indicated by abnormal liver function tests, alanine transaminase (ALT), aspartate transaminase (AST),
alkaline phosphatase, or serum bilirubin (> 2.0 x upper limit of normal (ULN) for any of these tests)] for an
etiology other than non-alcoholic 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 will be exclusionary, but the presence of NAFLD would not be exclusionary.
8. History or presence of impaired renal function as indicated by clinically significant abnormal creatinine,
blood urea nitrogen (BUN), or urinary constituents (e.g., albuminuria) or moderate to severe renal
dysfunction as defined by the Cockroft Gault equation <30 mL/min if 17 years or older or by Schwartz if 16 years or less.
9. History or close family history (parents or siblings) of skin cancer or melanoma, or patient history of ocularcutaneous albinism.
10. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions, 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 pretreatment biopsy results are of concern, the patient may need to be excluded from the study.
11. Volunteer is, in the opinion of the Study Investigator, not suitable to participate in the study.
12. Significant hypersensitivity to study drug.
13. Inability to comply with injection regimen.
14. Patients who have been placed in an institution through an official or court order, as well as those who are dependent on the sponsor, Investigator or study site.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified