Safety and Efficacy of Miglustat in Chinese NPC Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 6 June 2022
• Main ID: NCT03910621
• Date of registration: 08/03/2019
• Prospective Registration: Yes
• Primary sponsor: Actelion
• Public title: Safety and Efficacy of Miglustat in Chinese NPC Patients
• Scientific title: A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese Subjects
• Date of first enrolment: April 2, 2020
• Target sample size: 17
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03910621
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

China

Contacts

• Name: Yue Wu
• Affiliation: Janssen China R&D

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with confirmed 2 pathogenic mutations in either Niemann Pick Type C Gene 1 or
2 (NPC 1 or NPC 2) or 1 pathogenic mutation in either NPC1 or NPC2 plus a positive
biomarker (oxysterol or lysosphingolipids or bile acids) plus high clinical suspicion

- Signed informed consent prior to any study-mandated procedure.

- For subjects who are younger than 18 years consent must be sought of at least one
legal guardian who shall sign the informed consent form and indicate the relationship
between him/her and the subject.

- Subjects who are 18 years or older must sign the consent. If the subject cannot make
an independent decision to participate in the study, consent must be sought of the
legal agents who shall sign the informed consent form and indicate the relationship
between him/her and the subject.

- Male and female subjects aged 4 years and older.

- Subjects who can performed the tests for the horizontal and vertical saccadic eye
movements;

- Subjects who are able to swallow the study drug;

- Women of childbearing potential are only eligible if the following applies:

- Negative urine pregnancy test at screening.

- Agreement to undertake monthly urine pregnancy tests during the study and up to at
least 30 days after study treatment discontinuation.

- Agreement to use one of the methods of birth control / follow the contraception scheme
from screening up to at least 30 days after study treatment discontinuation.

- A fertile male (physiologically capable of fathering a child according to
investigator's judgment) is eligible only if he agrees to use a condom during the
treatment period and for an additional 12 weeks after treatment discontinuation.

Exclusion Criteria:

Subjects must not fulfill any of the following exclusion criteria. It is not permitted to
waive any of the criteria for any subject:

- Subjects suffering from clinically significant diarrhea (>3 liquid stools per day for
>7 days) without definable cause within 3 months before enrollment.

- Known hypersensitivity to the investigational treatment or drugs of the same class, or
any of their excipients.

- Subjects who suffer from renal insufficiency with a creatinine clearance rate (CCR) of
< 30ml/min per 1.73m2.

- Pregnant, planning to be become pregnant or lactating females, not using reliable
birth control male adult subjects.

- Previous exposure to investigational treatment for more than 12 months before study
start.

- Planned or current treatment with another investigational treatment up to 3 months
prior to randomization. Symptomatic therapies are allowed (such as curcumin).

- Any known factor or disease that might interfere with treatment compliance, study
conduct, or interpretation of the results, such as drug or alcohol dependence or
psychiatric disease, end stage disease including wheelchair bound patients, bedridden
patients etc.

- Subjects who are judged unqualified for the clinical trial by the investigator.

- Subjects who suffer lysosomal storage diseases, enzyme deficiency or neurological
diseases other than NPC.

- Subjects who suffer variant filipin staining without confirmatory genetic diagnosis of
NPC.

- Subjects with uncontrolled epilepsy.

- Subjects with complete ophthalmoplegia.

- Known concomitant life-threatening disease with a life expectancy < 12 months.



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Niemann-Pick Disease, Type C

Intervention(s)

Drug: Miglustat

Primary Outcome(s)

Observed change in HSEM (ms/deg) [Time Frame: baseline to week 52]

Secondary Outcome(s)

Incidence of treatment-emergent AEs and SAEs [Time Frame: Baseline to 30 days after End of Treatment (Week 52)]

Change in Pineda disability scale score [Time Frame: baseline to week 52]

Secondary ID(s)

AC-056C405

Source(s) of Monetary Support

Please refer to primary and secondary sponsors