Long Term Extension Trial of Setmelanotide

Overview

About this study

The purpose of this extension study of up to an additional 2 years duration beyond the index trial for patients who have completed a previous study of setmelanotide for genetic obesity disorders upstream of the MC4 receptor in the melanocortin-leptin pathway. Since continued assessments of the safety and efficacy of setmelanotide are the same in this extension protocol regardless of the disease studied in the index protocol, all patients can be followed in this single extension protocol. Nevertheless, the analysis of each individual disease will be performed separately with the ability to combine data from the original index protocol and this extension protocol on a disease specific basis. For patients who qualify and consent, the study will start immediately on the completion of their index protocol such that there are no gaps in setmelanotide therapy , if possible. Efforts should be made to allow the last visit of a prior index trial or an index trial extension phase and the initial visit of this comprehensive long-term extension trial to occur on the same day. Patients will continue taking the same dose of setmelanotide that they were taking when they finished the index protocol. Dose changes can be made for safety or efficacy. Patients will be assessed in the clinic approximately every 3 months for vital signs, adverse events, concomitant medications, weight, waist circumference, and hunger score. Safety laboratories will be collected at each visit and additional assessments will be done on a yearly basis. In general assessments will be less frequent and burdensome than was required in their index protocol.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • 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), post-menopausal 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.

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 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 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.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Andres Acosta, M.D., Ph.D.

Closed for enrollment

Contact information:

Elizabeth Manggaard CCRP

(507) 538-5861

Bungum.Lisa2@mayo.edu

More information

Publications

Publications are currently not available
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