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Safety and Efficacy of Miglustat in Chinese NPC Patients

Primary Purpose

Niemann-Pick Disease, Type C

Status
Completed
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Miglustat
Sponsored by
Actelion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Niemann-Pick Disease, Type C

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Peking University first Hospital
  • Xin Hua Hospital, Shanghai Jiao Tong University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Miglustat

Arm Description

Miglustat is administered three times a day as an oral capsule

Outcomes

Primary Outcome Measures

Observed change in HSEM (ms/deg)
Change in HSEM from baseline to week 52

Secondary Outcome Measures

Change in Pineda disability scale score
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.
Incidence of treatment-emergent AEs and SAEs
Treatment-emergent adverse events (AEs) up to 30 days after EOT

Full Information

First Posted
March 8, 2019
Last Updated
May 24, 2022
Sponsor
Actelion
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1. Study Identification

Unique Protocol Identification Number
NCT03910621
Brief Title
Safety and Efficacy of Miglustat in Chinese NPC Patients
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
April 2, 2020 (Actual)
Primary Completion Date
March 25, 2022 (Actual)
Study Completion Date
March 25, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Actelion

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Niemann-Pick Disease, Type C

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
17 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Miglustat
Arm Type
Experimental
Arm Description
Miglustat is administered three times a day as an oral capsule
Intervention Type
Drug
Intervention Name(s)
Miglustat
Intervention Description
capsule, oral use
Primary Outcome Measure Information:
Title
Observed change in HSEM (ms/deg)
Description
Change in HSEM from baseline to week 52
Time Frame
baseline to week 52
Secondary Outcome Measure Information:
Title
Change in Pineda disability scale score
Description
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.
Time Frame
baseline to week 52
Title
Incidence of treatment-emergent AEs and SAEs
Description
Treatment-emergent adverse events (AEs) up to 30 days after EOT
Time Frame
Baseline to 30 days after End of Treatment (Week 52)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yue Wu
Organizational Affiliation
Janssen China R&D
Official's Role
Study Director
Facility Information:
Facility Name
Peking University first Hospital
City
Beijing
ZIP/Postal Code
100034
Country
China
Facility Name
Xin Hua Hospital, Shanghai Jiao Tong University
City
Shanghai
ZIP/Postal Code
200092
Country
China

12. IPD Sharing Statement

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Safety and Efficacy of Miglustat in Chinese NPC Patients

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