Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Summary

A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC). The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the participant's current prescribed best routine clinical care; participant's routine clinical care may, or may not, include miglustat. The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including participants aged 6 to \<24 months at study enrolment.

Timeline

Start

2016-06-14

Primary completion

2018-06-25

Completion

2024-10-31

Outcome

Met primary endpoint

paper A statistically significant treatment difference in favour of arimoclomol of -1.40 (95% confidence interval: -2.76, -0.03; P = .046) was observed reads as met primary endpoint PMID 34418116 ↗

release “Interim data analysis of four-year, open-label extension from Phase 2/3 clinical trial suggests arimoclomol may reduce long-term progression of Niemann-Pick Disease Type C (NPC)” reads as unknown zevra.com ↗

release “In the pivotal Phase 3 trial, arimoclomol halted disease progression compared to placebo over the twelve month duration of the trial” reads as met primary endpoint zevra.com ↗

release “In the post hoc efficacy analysis of the randomized, placebo-controlled NPC002 trial, arimoclomol combined with miglustat demonstrated a statistically significant slowing of disease progression” reads as met primary endpoint zevra.com ↗

Publications

• Background Patterson MC, Lloyd-Price L, Guldberg C, Doll H, Burbridge C, Chladek M, iDali C, Mengel E, Symonds T. Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. Orphanet J Rare Dis. 2021 Feb 12;16(1):79. doi: 10.1186/s13023-021-01719-2.
• Results Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Gronborg S, Harmatz P, Heron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Andreasen AK, Geist MA, Havnsoe Torp Petersen N, Ingemann L, Hansen T, Blaettler T, Kirkegaard T, I Dali C. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Arimoclomol	Small molecule	31 mg	Oral
Subject	Arimoclomol	Small molecule	124 mg	Oral

Indications