Long Term Extension Trial of Setmelanotide

  • STATUS
    Recruiting
  • End date
    Mar 27, 2023
  • participants needed
    150
  • sponsor
    Rhythm Pharmaceuticals, Inc.
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Updated on 27 February 2021
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Summary

This is a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in patients who have completed a previous clinical trial on treatment with setmelanotide for obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.

Description

The purpose of this protocol is to continue the assessment of setmelanotide treatment in patients who have successfully completed all critical study evaluations in a prior trial (index protocol) of setmelanotide for the treatment of obesity associated with genetic defects in the leptin-melanocortin pathway upstream of the MC4 receptor. The primary objectives of this extension trial are to explore the long-term safety and tolerability of setmelanotide for up to 5 years or until drug is otherwise available through authorized use. Patients can enter this protocol immediately upon completion of their index protocol such that dosing of setmelanotide continues without gaps in therapy.

Details
Condition Obesity Associated With Defects in Leptin-melanocortin Pathway, Obesity Associated With Defects in Leptin-melanocortin Pathway, Obesity Associated With Defects in Leptin-melanocortin Pathway, Obesity Associated With Defects in Leptin-melanocortin Pathway, Obesity Associated With Defects in Leptin-melanocortin Pathway, Obesity Associated With Defects in Leptin-melanocortin Pathway, Obesity Associated With Defects in Leptin-melanocortin Pathway
Treatment Setmelanotide
Clinical Study IdentifierNCT03651765
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 Obesity Associated With Defects in Leptin-melanocortin Pathway?
Do you have any of these conditions: Do you have Obesity Associated With Defects in Leptin-melanocortin Pathway??
Do you have any of these conditions: Do you have Obesity Associated With Defects in Leptin-melanocortin Pathway??
Do you have any of these conditions: Do you have Obesity Associated With Defects in Leptin-melanocortin Pathway??
Do you have any of these conditions: Do you have Obesity Associated With Defects in Leptin-melanocortin Pathway??
Do you have any of these conditions: Do you have Obesity Associated With Defects in Leptin-melanocortin Pathway??
Patients aged 6 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
Note: The index study may have a primary endpoint relied on efficacy, safety
or tolerability. Patient will be eligible for extension study if the Primary
Investigator believes the patient exhibited a clinically meaningful benefit
(i,e, efficacy) to setmelanotide treatment, and would benefit from continued
treatment, after discussion with the Sponsor
\. 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
\. 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
All patients must agree to follow requirements for contraception outlined in
the protocol

Exclusion Criteria

Pregnant and/or breastfeeding women
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
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
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
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
A Patient Health Questionnaire-9 (PHQ-9) score of 15
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
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
Moderate to severe renal dysfunction as defined by a glomerular filtration rate (GFR) <30 mL/min
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
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