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Protocol Details

MEASuRE: Metreleptin Effectiveness and Safety Registry

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts

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: 7 mo
Max Age: N/A

Referral Letter Required

Yes

Population Exclusion(s)

None

Special Instructions

Currently Not Provided

Keywords

Metreleptin;
Myalept;
Generalized Lipodystrophy

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.

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Eligibility

INCLUSION CRITERIA:

-Patients who are treated with metreleptin as part of clinical care i.e., those:

--who provide written consent before and/or upon enrollment into registry, or

--are treated with metreleptin through commercial supply but before enrollment into registry, or

--are continuing treatment with metreleptin through commercial supplies but coming off the metreleptin clinical studies

EXCLUSION CRITERIA:

-Patients receiving metreleptin currently as part of a clinical trial or through expanded access including compassionate use.

-Patients currently treated with an investigational agent as part of a clinical trial.


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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.

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Contacts:

Principal Investigator

Referral Contact

For more information:

Elaine K. Cochran, C.R.N.P.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)



Megan S. Startzell, R.N.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institutes of Health
Building 10
Room 5-5750
10 Center Drive
Bethesda, Maryland 20892
(301) 402-6371
megan.startzell@nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: 1-866-411-1010
PRPL@cc.nih.gov

Clinical Trials Number:

NCT02325674

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