NCT03910621 is a Phase 4 clinical trial of Miglustat in Niemann-Pick Disease, Type C, sponsored by Actelion.

Who is sponsoring NCT03910621?

NCT03910621 is sponsored by Actelion.

What drugs are being tested in NCT03910621?

Interventions in NCT03910621: Miglustat.

What condition does NCT03910621 study?

NCT03910621 studies Niemann-Pick Disease, Type C.

Is NCT03910621 still recruiting?

NCT03910621 is currently completed. Last updated 30 March 2025.

Last reviewed 2025-03-30 · How we verify

NCT03910621

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety and Efficacy of Miglustat in Chinese NPC Patients

Completed Phase 4 Last updated 30 March 2025

What this trial tests

Phase 4 trial testing Miglustat in Niemann-Pick Disease, Type C in 17 participants. Completed in 25 March 2022.

Timeline

2 April 2020

Primary endpoint
25 March 2022

25 March 2022

Quick facts

Lead sponsor	Actelion
Phase	Phase 4
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	17
Start date	2 April 2020
Primary completion	25 March 2022
Estimated completion	25 March 2022
Sites	2 locations across China

Drugs / interventions tested

Miglustat (MIGLUSTAT) — full drug profile →

Conditions studied

Niemann-Pick Disease, Type C — all drugs for Niemann-Pick Disease, Type C →

Sponsor

Actelion — full company profile →

Who can join

4 and older, any sex, with Niemann-Pick Disease, Type C. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study. Approximately 19 subjects with Niemann Pick Type C disease (NPC) will be enrolled in this study. The study will be conducted at 2 sites in China.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

Evaluation of the safety and efficacy of miglustat for the treatment of Chinese patients with Niemann-Pick disease type C: A prospective, open-label, single-arm, phase IV trial.
Zhang H, Xiong H, Wei C, Yi M, et al · · 2024 · cited 2× · PMID 39628618 · DOI 10.5582/irdr.2024.01056

Verify or expand the search:

Other trials of Miglustat

Trials testing the same drug.

NCT06121011 — A Global Prospective Observational Registry of Patients With Pompe Disease · recruiting
NCT03822013 — Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) · Phase 3 · terminated
NCT03729362 — A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease · Phase 3 · completed
NCT00319046 — Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 G · Phase 3 · completed

Other recruiting trials for Niemann-Pick Disease, Type C

Currently open trials in the same condition.

NCT05588167 — Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C · recruiting
NCT00344331 — Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C · recruiting

Other Actelion trials

Trials by the same sponsor.

NCT05731492 — A Study of Macitentan in Children Below 2 Years of Age · Phase 1 · withdrawn
NCT05433675 — A Study of Two Macitentan Formulations in Healthy Adult Participants · Phase 1 · completed
NCT05427162 — A Study of Single and Multiple Doses of Different Formulations of a Prostacyclin Receptor Agonist · Phase 1 · completed
NCT05392530 — A Study of Two Different Test Formulations Compared to the Reference Formulation of Macitentan in Healthy Adult Particip · Phase 1 · completed
NCT05179876 — A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no O · Phase 3 · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03910621 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Actelion
Last refreshed: 30 March 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03910621.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing