Financiado en parte por Retrotope
Double blind trial of a deuterated form of linoleic acid (RT001) in Friedreich ataxia
Lynch DR, Mathews KD, Perlman S, Zesiewicz T, Subramony S, Omidvar O, Vogel AP, Krtolica A, Litterman N, van der Ploeg L, Heerinckx F, Milner P, Midei M
Journal of neurology · 13 citas
Etiquetas de revisión
Financiado por la industriaDatos neutrales que nuestra revisión registró sobre cómo se hizo este estudio. Describen el método, nunca si nos gusta el resultado.
Cómo se estudió
- Diseño
- Ensayo controlado aleatorizado (indexado por PubMed)
- Estudiado en
- Personas
- Resultado principal
- Marcadores de salud y funcionamiento
- Consumo medido con
- No se indica
Quién lo pagó
- Financiación
- Financiado por la industria
- Empresa
- Retrotope
Según 1 financiador declarado.
Publicación
- Publicado
- 2022-12-03 · J Neurol · vol. 270 · número 3 · págs. 1615–1623
- Editorial
- Springer Science+Business Media
- Citado
- 17 citas · más que el 75% de estudios similares · 1.1× el promedio de su campo
- Referencias
- 43 trabajos
- Acceso
- De pago
- Áreas de investigación
- Genetic Neurodegenerative Diseases · Metabolism and Genetic Disorders · Mitochondrial Function and Pathology
- Palabras clave
- Friedreich's ataxia, lipid peroxidation, oxidative damage, exercise capacity, VO2max, erythrocyte membrane, neurodegenerative disease treatment
- MeSH
- humans, friedreich ataxia, linoleic acids, linoleic acid, walking, double-blind method
13 autores
- David R. Lynch🇺🇸 · autor de correspondenciaChildren's Hospital of Philadelphia; University of Pennsylvania
- Katherine D. Mathews🇺🇸University of Iowa
- Susan Perlman🇺🇸University of California, Los Angeles
- Theresa A. Zesiewicz🇺🇸University of South Florida; James A. Haley Veterans' Hospital
- Sub H. Subramony🇺🇸University of Florida Health; University of Florida
- Omid Omidvar🇺🇸University of California, Los Angeles
- Adam P. Vogel🇦🇺The University of Melbourne
- Ana Krtolica🇺🇸Retrotope (United States)
- Nadia K. Litterman🇺🇸Retrotope (United States)
- Lex H.T. Van der Ploeg🇺🇸Retrotope (United States)
- Frederic Heerinckx🇺🇸Retrotope (United States)
- Peter Milner🇺🇸Retrotope (United States)
- Mark G. Midei🇺🇸Retrotope (United States)
Resumen
El texto original del estudio, en el idioma en que se publicó.
Objectives
Friedreich ataxia is (FRDA) an autosomal recessive neurodegenerative disorder associated with intrinsic oxidative damage, suggesting that decreasing lipid peroxidation (LPO) might ameliorate disease progression. The present study tested the ability of RT001, a deuterated form of linoleic acid (D2-LA), to alter disease severity in patients with FRDA in a double-blind placebo-controlled trial.
Methods
Sixty-five subjects were recruited across six sites and received either placebo or active drug for an 11-month study. Subjects were evaluated at 0, 4, 9, and 11 months, with the primary outcome measure being maximum oxygen consumption (MVO2) during cardiopulmonary exercise testing (CPET). A key secondary outcome measure was a composite statistical test using results from the timed 1-min walk (T1MW), peak workload, and MVO2.
Results
Forty-five subjects completed the protocol. RT001 was well tolerated, with no serious adverse events related to drug. Plasma and red blood cell (RBC) membrane levels of D2-LA and its primary metabolite deuterated arachidonic acid (D2-AA) achieved steady-state concentrations by 4 months. No significant changes in MVO2 were observed for RT001 compared to placebo. Similarly, no differences between the groups were found in secondary or exploratory outcome measures. Post hoc evaluations also suggested minimal effects of RT001 at the dosages used in this study.
Interpretations
The results of this study provide no evidence for a significant benefit of RT001 at the dosages tested in this Friedreich ataxia patient population.
Resumen vía Europe PMC. Los derechos de autor siguen siendo de los autores o la editorial.
Confianza de la comunidad
Cargando…
¿Cuánto confías en los hallazgos de este estudio?
Comentarios
¿Algo está mal aquí?
Señala este estudio si su información, etiquetas o financiación parecen incorrectas. Un editor revisa cada señalamiento.
Información educativa sobre investigación publicada. No es consejo médico ni una recomendación para empezar o dejar nada.