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Safety and Efficacy of Miglustat in Chinese NPC Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03910621
Recruitment Status : Not yet recruiting
First Posted : April 10, 2019
Last Update Posted : September 4, 2019
Information provided by (Responsible Party):

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
Niemann-Pick Disease, Type C Drug: Miglustat Phase 4

Detailed Description:
This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study.The study is conducted in Chinese subjects aged 4 years and older with Niemann Pick Type C disease (NPC). Approximately 19 subjects will be enrolled in this study. The study will be conducted at 2 sites in China. Patients will be treated with miglustat for 12 months, efficacy and safety outcomes will be measured

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Estimated Study Start Date : December 20, 2019
Estimated Primary Completion Date : July 1, 2021
Estimated Study Completion Date : February 1, 2022

Arm Intervention/treatment
Experimental: Miglustat
Miglustat is administered three times a day as an oral capsule
Drug: Miglustat
capsule, oral use

Primary Outcome Measures :
  1. Observed change in HSEM (ms/deg) [ Time Frame: baseline to week 52 ]
    Change in HSEM from baseline to week 52

Secondary Outcome Measures :
  1. Change in Pineda disability scale score [ Time Frame: baseline to week 52 ]
    Change in the modified Pineda disability scale score. The scale is assessing 4 key domains (manipulation, ambulation, language and swallowing). Individual scores of the domains are claculated ted into a composite score, 6 being the lowest and best score and 24 being the worst and highest score.

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

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

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Responsible Party: Actelion Identifier: NCT03910621     History of Changes
Other Study ID Numbers: AC-056C405
First Posted: April 10, 2019    Key Record Dates
Last Update Posted: September 4, 2019
Last Verified: September 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Histiocytosis, Non-Langerhans-Cell
Pick Disease of the Brain
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Frontotemporal Dementia
Frontotemporal Lobar Degeneration
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action