Long Term Extension Trial of Setmelanotide


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.

Key Inclusion Criteria:

1. Patients aged 2 or older (or aged >2 years as per local regulations) who have
completed participation in a previous setmelanotide trial and demonstrated adequate
safety and meaningful clinical benefit (efficacy)

2. Patient and/or parent or guardian is able to communicate with the investigator,
understand and sign the written informed consent/assent, and comply with the trial

3. Agree to use a highly effective form of contraception throughout the trial

Key Exclusion Criteria:

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)

3. Current, clinically significant disease

4. Documented diagnosis of schizophrenia, bipolar disorder, personality disorder, major
depressive disorder, or other psychiatric disorder(s)

5. Suicidal ideation, attempt or behavior

6. History of significant liver disease

7. Moderate to severe renal dysfunction as defined by a glomerular filtration rate (GFR)
<30 mL/min.

8. History or close family history of melanoma or patient history of oculocutaneous

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 5/31/23. Questions regarding updates should be directed to the study team contact.

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


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