ArticleMolecular genetics and metabolism reports2026
Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy.
Article in Molecular genetics and metabolism reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02612129 (Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C), which is not on this map. Not yet cited in PubMed.
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Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C
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7 authors.
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Abstract
Background: Arimoclomol has been approved in the US for the treatment of Niemann-Pick disease type C (NPC) in patients aged ≥2 years, in combination with miglustat. This multicenter, open-label substudy of the phase 2/3 NPC-002 trial (NCT02612129) evaluated the safety, pharmacokinetics (PK) and impact on clinical status outcomes of arimoclomol in infants with NPC 6-<24 months of age. Methods: Infants with NPC aged 6-<24 months received arimoclomol in addition to their standard of care management for up to 36 months. The dosing regimen used for patients <24 months differed from the regimen recommended in the FDA label. The primary endpoint was safety and tolerability of arimoclomol; secondary endpoints were changes in clinical status (physical examination and Bayley III developmental scores), biomarkers, and PK. Results: Five patients (three females, two males; aged 14-23 months at screening) were enrolled; four remained in the study >12 months; arimoclomol exposure ranged from 72 to 1109 days. All patients received concomitant miglustat. Across 108 reported adverse events (AEs), most were considered mild or moderate in severity and non-serious. A total of 15 serious AEs were reported for two patients. Two AEs in one patient (elevated alanine/aspartate aminotransferases) were considered probably related to arimoclomol and resolved within 51 days; the patient was withdrawn from the substudy. No clinically significant changes were observed in hematology, kidney ultrasound imaging, or vital signs. Mean arimoclomol exposure over the first 8 h post-dose (1378.3-2988 h∙μg/L) was comparable to levels in NPC patients aged 2-19 years. Changes in Bayley III scores and biomarkers varied between individuals. Conclusion: Arimoclomol was well tolerated in infants initiating treatment before 2 years of age, with no new safety signals. PK profiles support the dosing regimen used. These findings suggest that early initiation of arimoclomol could be considered for the 6-24-month population. Further investigation in larger cohorts is warranted to elucidate the impact of arimoclomol in NPC patients under 2 years of age.
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