This study is currently recruiting participants.
Number
16-DK-0173
Sponsoring Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Recruitment Detail
Type: Participants currently recruited/enrolled Gender: Male & Female Min Age: 6 mo Max Age: 98 Years
Referral Letter Required
Yes
Population Exclusion(s)
None
Keywords
Metreleptin; Myalept; Generalized Lipodystrophy; Natural History
Recruitment Keyword(s)
Condition(s)
Lipodystrophy
Investigational Drug(s)
Investigational Device(s)
Intervention(s)
Supporting Site
National Institute of Diabetes and Digestive and Kidney Diseases
Generalized lipodystrophy (GL) is a rare metabolic disease. People with GL have a lack of fatty tissue. Myalept is a drug that is approved by the Food and Drug Administration (FDA) to treat GL. It is also called metreleptin. The FDA is making researchers do a registry study for Myalept. They will observe people who have taken the drug to see how safe and effective it is. No medication will be given in the study and no tests will be done.
Objective:
To collect data about the long-term safety and effectiveness of Myalept in clinical practice.
Eligibility:
People who are planning to start taking Myalept or have taken at least one dose of Myalept.
Design:
Participants will sign a consent document.
All data will be collected by the study team at the NIH and/or at the participant s local doctor or his/her staff. This may include data from:
Physical exams
Lab tests
Procedures and other tests
Participants will answer questions about their health. They may also be asked about:
Personal and family medical history.
Medicines and therapies
Illnesses
Hospitalizations
If a participant becomes pregnant during the study, data may be collected about the pregnancy and the child s health.
Participants will have follow-up visits every year. These may be at NIH or local.
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INCLUSION CRITERIA: -Patients who are treated with metreleptin as part of clinical care i.e., those: --who provide written consent before enrolment into registry, and --are treated with metreleptin through commercial supply at the time or before enrolment into registry, or --are coming off the metreleptin clinical studies and continuing or restarting treatment with metreleptin through commercial supplies. EXCLUSION CRITERIA: -Patients currently treated with an investigational agent as part of a clinical trial.
-Patients who are treated with metreleptin as part of clinical care i.e., those:
--who provide written consent before enrolment into registry, and
--are treated with metreleptin through commercial supply at the time or before enrolment into registry, or
--are coming off the metreleptin clinical studies and continuing or restarting treatment with metreleptin through commercial supplies.
EXCLUSION CRITERIA:
-Patients currently treated with an investigational agent as part of a clinical trial.
Principal Investigator
Referral Contact
For more information: