Protocol Details
MEASuRE: Metreleptin Effectiveness and Safety Registry
This study is currently recruiting participants.
Summary
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) |
None |
Condition(s) |
Lipodystrophy |
Investigational Drug(s) |
None |
Investigational Device(s) |
None |
Intervention(s) |
None |
Supporting Site |
National Institute of Diabetes and Digestive and Kidney Diseases |
Background:
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.
Eligibility
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.
Citations:
Chan JL, Oral EA. Clinical classification and treatment of congenital and acquired lipodystrophy. Endocr Pract. 2010 Mar-Apr;16(2):310-23. doi: 10.4158/EP09154.RA.
Akinci B, Onay H, Demir T, Ozen S, Kayserili H, Akinci G, Nur B, Tuysuz B, Nuri Ozbek M, Gungor A, Yildirim Simsir I, Altay C, Demir L, Simsek E, Atmaca M, Topaloglu H, Bilen H, Atmaca H, Atik T, Cavdar U, Altunoglu U, Aslanger A, Mihci E, Secil M, Saygili F, Comlekci A, Garg A. Natural History of Congenital Generalized Lipodystrophy: A Nationwide Study From Turkey. J Clin Endocrinol Metab. 2016 Jul;101(7):2759-67. doi: 10.1210/jc.2016-1005. Epub 2016 May 4.
Meehan CA, Cochran E, Kassai A, Brown RJ, Gorden P. Metreleptin for injection to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. Expert Rev Clin Pharmacol. 2016;9(1):59-68. doi: 10.1586/17512433.2016.1096772. Epub 2015 Oct 14.
Contacts:
Clinical Trials Number:
NCT02325674