Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
Diabetes · Lipodystrophy · Hyperlipidemia
De qué trata este estudio
El registro no identifica actualmente este estudio como reclutando. Consulta su estado antes de planificar una evaluación.
- Tipo de estudio
- De intervención
- Fase
- Fase 2
- Rango de edad
- 6 meses - 98 años
- Sexo admitido
- Todos los sexos
- Voluntarios sanos
- No aceptados
- Participantes
- 29 Real
Estos son puntos de partida, no una decisión de elegibilidad. El equipo debe revisar todos los requisitos.
Las explicaciones y la navegación están en español. Los textos proporcionados por el registro, incluidos títulos, resúmenes y criterios, se conservan en su idioma original. Puedes pedir orientación en español a WCR.
Acerca de la investigación
Background:
\- Partial lipodystrophy can cause high blood fat levels and resistance to insulin. This can lead to health problems including diabetes. Researchers have found that the drug metreleptin improves health in people with this disease.
Objective:
\- To test the safety and effectiveness of metreleptin.
Eligibility:
- People age 6 months and older with partial lipodystrophy who
- have received metreleptin through NIH studies and shown improvement AND
- cannot get metreleptin other ways.
Design:
- Participants will come to NIH approximately every 6 months during year one, then every 1 2 years. Financial assistance may be available for travel within the U.S.
- At visits, participants will get a supply of metreleptin to take home for daily injections, or it can be shipped to them inside the U.S. They will have:
- plastic catheter placed in an arm vein.
- blood tests, urine collection, and physical exam.
- oral glucose tolerance test, drinking a sweet liquid.
- ultrasound of the heart, liver, uterus, and ovaries. A gel and a probe are placed on the skin and pictures are taken of the organs.
- echocardiogram, which takes pictures of the heart with sound waves.
- Resting Metabolic Rate taken. A plastic hood is worn over the head while the oxygen they breathe is measured.
- Participants will have up to 3 DEXA scan x-rays per year.
- Participants may have:
- annual bone x-rays.
- liver biopsies every few years. A needle will be inserted into the liver to obtain a small piece. Participants will sign a separate consent for this.
- Participants must be seen regularly by their local doctors and have blood tests at least every 3-6 months at home.
Leer la descripción completa
Leptin is an adipocyte-derived hormone that can be thought of as a signal from adipose tissue to the rest of the body conveying information about long-term nutritional status. Patients with lipodystrophy have leptin deficiency secondary to lack of adipose tissue. The combination of leptin deficiency and ectopic lipid deposition in patients with lipodystrophy leads to metabolic complications including severe insulin resistance and diabetes, hypertriglyceridemia, non-alcoholic steatohepatitis, and polycystic ovarian syndrome. Between 2000 and 2014, the NIDDK IRP conducted an open-label clinical trial of the recombinant human leptin analog, metreleptin, in patients with generalized and partial forms of lipodystrophy. This study showed that metreleptin ameliorates metabolic and endocrine abnormalities in lipodystrophy, including reducing food intake, improving insulin resistance and diabetes, reducing ectopic lipid, and normalizing reproduction. Based on these data, metreleptin was approved by the FDA in February, 2014, for patients with generalized, but not partial, lipodystrophy. Our data have shown, however, that a subgroup of patients with partial lipodystrophy do gain medical benefit from metreleptin.
The purpose of this study is twofold:
- To provide access to metreleptin to patients with partial lipodystrophy who have previously received and derived benefit from metreleptin through NIH studies (protocols 02-DK-0022 and 13-DK-0057).
- To continue to collect data on the long-term efficacy of metreleptin in ameliorating the metabolic complications of partial lipodystrophy.
Metreleptin will be given at doses of less than or equal to 0.24 mg/kg/day, adjusted based on body weight and metabolic control. Patients will be seen approximately once per year at NIH for evaluation, and potentially less frequently for those who are medically stable and have difficulty traveling to NIH. Laboratory evaluation will be obtained more frequently by the patient s home providers as clinically indicated. The primary outcomes of the study are improvements in serum triglycerides and hemoglobin A1c levels. Secondary outcomes include measures of steatohepatitis and ectopic lipid, body composition, bone mineral density and bone mineral metabolism, and pituitary and reproductive function.
Metreleptin is supplied by Chiesi USA, Inc. Neither the NIH nor Chiesi USA, Inc. can guarantee that leptin will be available indefinitely and/or after the study ends.
¿Quién podría participar?
La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.
- INCLUSION CRITERIA
- Age greater than or equal to 6 months
- Partial lipodystrophy (either genetic or acquired)
- Previously or currently treated with metreleptin under NIH study 02-DK-0022 and/or NIH study 13-DK-0057.
- Documented metabolic benefit from prior or current metreleptin treatment, defined as one or more of the following:
- TG reduction greater than or equal to 30% OR
- HbA1c reduction greater than or equal to 1% OR
- Decrease in insulin requirements greater than or equal to 40% OR
- Decrease in episodes of pancreatitis OR
- Improvement in steatohepatitis OR
- Withdrawal of metreleptin led to marked worsening of metabolic parameters
EXCLUSION CRITERIA
- Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs.
- Known HIV infection or HIV-associated lipodystrophy.
- Psychiatric disorder impeding competence or compliance.
- Any medical condition or medication that will increase risk to the subject.
- Current alcohol or substance abuse.
- Subjects who have a known hypersensitivity to E. coli derived proteins.
Tratamiento, grupos y participación
El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.
- Asignación
- No aplica
- Enmascaramiento
- Sin enmascaramiento
Leptin therapy
leptin administered via SC injections BID
Drug: Metreleptin
Metreleptin DRUG
A leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with partial lipodystrophy.
Qué evalúan los investigadores
Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.
Medidas principales
Serum hemoglobin A1C
Plazo: every 6-12 months
improvement in lab value
Serum triglycerides
Plazo: every 6-12 months
improvement in lab value
Revisa las tablas completas y sus limitaciones en el registro oficial.
Abrir resultados oficiales ↗Centros de investigación (1)
El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.
1 centros
National Institutes of Health Clinical CenterBethesda, Maryland, United StatesEstado desconocido
Equipo y fechas
- Patrocinador principal
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Inicio del estudio
- 14 oct 2014 Real
- Finalización primaria
- 31 jul 2027 Estimado
- Finalización del estudio
- 31 jul 2027 Estimado
- Primera publicación
- 13 oct 2014 Estimado
- Última actualización
- 9 nov 2026 Real
Contactos oficiales del estudio
No hay un contacto central indicado. Revisa los centros y el registro oficial.
Título oficial del estudio
Compassionate Use of Metreleptin in Previously-Treated Patients With Partial Lipodystrophy
Consulta a Wellness Clinical Research
Tu mensaje va a WCR, incluso si otra organización realiza este estudio. WCR puede orientarte, pero podría no realizar este estudio, tener acceso a su equipo ni poder inscribirte.
Fuente: API v2 de ClinicalTrials.gov. Patrocinadores e investigadores envían la información. La NLM no aprueba la seguridad ni la ciencia de todos los estudios registrados. Aviso del registro.
Consultado: 12 nov 2026 · Última actualización del registro: 9 nov 2026