Niemann-Pick Disease, Type CPhase 4NCT03910621
Safety and Efficacy of Miglustat in Chinese NPC Patients
A Phase 4 study testing Miglustat in people with Niemann-Pick Disease, Type C, run by Actelion at 2 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Niemann-Pick Disease, Type C
- What is being tested?
- Miglustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 2 sitesChina
- Study dates
- 2 Apr 2020to 25 Mar 2022
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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;
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
2 sites in 1 country.
China2 sites
Peking University first Hospital
Beijing, 100034
Xin Hua Hospital, Shanghai Jiao Tong University
Shanghai, 200092
About this study
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
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.
Read the 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
- Conditions
- Niemann-Pick Disease, Type C
- Being tested
- Miglustat
- Medical subject headings
- Niemann-Pick Disease, Type C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MiglustatDrug
capsule, oral use
| Study group | Role | What they receive |
|---|---|---|
| Miglustat | Experimental | Miglustat is administered three times a day as an oral capsule Receives: Drug: Miglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Observed change in HSEM (ms/deg)
Change in HSEM from baseline to week 52
Measured: baseline to week 52
Secondary outcomes (2)
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.
Measured: baseline to week 52
Incidence of treatment-emergent AEs and SAEs
Treatment-emergent adverse events (AEs) up to 30 days after EOT
Measured: Baseline to 30 days after End of Treatment (Week 52)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2019
- Study start2 Apr 2020
- Primary completion25 Mar 2022
- Study completion25 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Niemann-Pick Disease, Type CStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Actelion
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yue Wu, Study director, Janssen China R&D
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03910621
- Sponsor's protocol ID
- AC-056C405
- Status last verified
- Mar 2025
- First submitted
- 8 Mar 2019
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Zhang H, Xiong H, Wei C, Yi M, Che Y, Zhuo J, Li X. 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. Intractable Rare Dis Res. 2024 Nov 30;13(4):227-235. doi: 10.5582/irdr.2024.01056.
PubMed 39628618
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03910621
- Acronym / sponsor ID
- AC-056C405
- Conditions
- Niemann-Pick Disease, Type C
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- ActelionIndustry (pharma, biotech, devices)
- Study start
- 2 Apr 2020 (actual)
- Primary completion
- 25 Mar 2022 (actual)
- Study completion
- 25 Mar 2022 (actual)
- First posted
- 10 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Miglustat | Experimental | Drug: MiglustatMiglustat is administered three times a day as an oral capsule |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Miglustatcapsule, oral use | Drug | Miglustat |
Primary outcome
Observed change in HSEM (ms/deg)
Time frame: baseline to week 52
Change in HSEM from baseline to week 52
2 secondary outcomes
Change in Pineda disability scale score
Time frame: baseline to week 52
Incidence of treatment-emergent AEs and SAEs
Time frame: Baseline to 30 days after End of Treatment (Week 52)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2025.
- Derived from this study: PubMed 39628618Zhang H, Xiong H, Wei C, Yi M, Che Y, Zhuo J, Li X. 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. Intractable Rare Dis Res. 2024 Nov 30;13(4):227-235. doi: 10.5582/irdr.2024.01056.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yue Wu study director
Janssen China R&D
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Miglustat in people with Niemann-Pick Disease, Type C, run by Actelion at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Observed change in HSEM (ms/deg) (time frame: baseline to week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03910621, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2025.