Treatment With N-Acetyl-L-Leucine Beneficial for Niemann-Pick Disease

Greater mean change from baseline seen in total score on the Scale for the Assessment and Rating of Ataxia with NALL versus placebo

By Elana Gotkine HealthDay Reporter

THURSDAY, Feb. 1, 2024 (HealthDay News) — Treatment with N-acetyl-L-leucine (NALL) for 12 weeks yields better neurological status than placebo among patients with genetically confirmed Niemann-Pick disease type C, according to a study published in the Feb. 1 issue of the New England Journal of Medicine.

Tatiana Bremova-Ertl, M.D., Ph.D., from the University Hospital Bern in Switzerland, and colleagues randomly assigned 60 patients age 4 years or older with genetically confirmed Niemann-Pick disease type C to receive 12 weeks of NALL followed by 12 weeks of placebo or to receive 12 weeks of placebo followed by 12 weeks of NALL in a 1:1 ratio.

The researchers found that the mean change from baseline in the Scale for the Assessment and Rating of Ataxia total score was −1.97 ± 2.43 and −0.60 ± 2.39 points after 12 weeks of receiving NALL and placebo, respectively (least-squares mean difference, −1.28 points). For the secondary end points, the results were generally supportive of the findings in the primary analysis; however, these were not adjusted for multiple comparisons. A similar incidence of adverse events was seen with NALL and placebo; there were no treatment-related serious adverse events reported.

“In this phase 3 trial involving patients with Niemann-Pick disease type C, treatment with NALL reduced neurologic signs and symptoms as compared with placebo over 12 weeks,” the authors write.

The study was funded by IntraBio.

Abstract/Full Text (subscription or payment may be required)

Editorial (subscription or payment may be required)

Copyright © 2024 HealthDay. All rights reserved.