Pompe DiseasePhase 4NCT02525172
Immune Modulation Therapy for Pompe Disease
A Phase 4 study testing Rituximab, intravenous immune globulin and Bortezomib in people with Pompe Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
The purpose of this study is to assess anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers after treatment with immune modulation therapy in patients of Pompe disease. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Pompe Disease
- What is being tested?
- Rituximab, intravenous immune globulin and Bortezomib
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteTaiwan
- Study dates
- Aug 2015to Jul 2020
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The patient (and/or patient's legal guardian if patient is < 18years) must provide written informed consent prior to any study-related procedures that are performed;
- The patient must have a confirmed diagnosis of Pompe disease defined as a documented acid α-glucosidase (GAA) enzyme deficiency from blood samples or 2 GAA gene mutations;
- The patient (and/or legal guardian) must have ability to comply with clinical protocol;
- Regimen A only: The patient is receiving enzyme replacement therapy, exhibits clinical decline, and has persistent high anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme;
- Regimen B only: The patient is cross-reactive immune material (CRIM) -negative AND have not received Myozyme infusion prior to enrollment
Who cannot join
- The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;
- The patient is at risk of reactivation of tuberculosis or has regular contact with individuals who are being actively treated for tuberculosis;
- The patient has used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment;
- The patient is pregnant or lactating;
- The patient has had or is required to have any live vaccination within one month prior to enrollment.
Trial locations and contacts
1 site in 1 country.
Taiwan1 site, 1 recruiting
- Recruiting
Yin-Hsiu Chien
Taipei, 100
Yin-Hsiu Chien, +886223123456, chienyh@ntu.edu.tw
Yin-Hsiu Chien
About this study
Official title: Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients
The purpose of this study is to assess anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers after treatment with immune modulation therapy in patients of Pompe disease.
- Conditions
- Pompe Disease
- Being tested
- Rituximab, intravenous immune globulin, Bortezomib, Methotrexate
- Keywords
- immune modulation therapy, acid α-glucosidase, antibody
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RituximabDrug
intravenous immune globulinDrug
BortezomibDrug
MethotrexateDrug
| Study group | Role | What they receive |
|---|---|---|
| ITT | Experimental | immune modulation therapy Receives: Drug: Rituximab; Drug: intravenous immune globulin; Drug: Bortezomib; Drug: Methotrexate |
What is being measured
The primary outcome is the main question the study is designed to answer.
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2015 (estimated)
- Study startAug 2015
- Primary completionJul 2020 (estimated)
- Study completionJul 2020 (estimated)
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 Pompe DiseaseStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe sponsor's other registered studies.
- 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
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yin-Hsiu Chien, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02525172
- Sponsor's protocol ID
- 201504036MIPB
- Status last verified
- Apr 2016
- First submitted
- 10 Aug 2015
- Last updated
- 15 Apr 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Chen HA, Hsu RH, Fang CY, Desai AK, Lee NC, Hwu WL, Tsai FJ, Kishnani PS, Chien YH. Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy. Front Immunol. 2024 Apr 23;15:1336599. doi: 10.3389/fimmu.2024.1336599. eCollection 2024.
PubMed 38715621
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients
- ClinicalTrials.gov ID
- NCT02525172
- Acronym / sponsor ID
- 201504036MIPB
- Conditions
- Pompe Disease
- Keywords
- immune modulation therapy, acid α-glucosidase, antibody
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- Aug 2015
- Primary completion
- Jul 2020 (estimated)
- Study completion
- Jul 2020 (estimated)
- First posted
- 17 Aug 2015 (estimated)
- 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.
The purpose of this study is to assess anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers after treatment with immune modulation therapy in patients of Pompe disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ITT | Experimental | Drug: Rituximab, Drug: intravenous immune globulin, Drug: Bortezomib, Drug: Methotrexateimmune modulation therapy |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- The patient (and/or patient's legal guardian if patient is < 18years) must provide written informed consent prior to any study-related procedures that are performed;
- The patient must have a confirmed diagnosis of Pompe disease defined as a documented acid α-glucosidase (GAA) enzyme deficiency from blood samples or 2 GAA gene mutations;
- The patient (and/or legal guardian) must have ability to comply with clinical protocol;
- Regimen A only: The patient is receiving enzyme replacement therapy, exhibits clinical decline, and has persistent high anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of Myozyme;
- Regimen B only: The patient is cross-reactive immune material (CRIM) -negative AND have not received Myozyme infusion prior to enrollment
Key exclusion criteria (5 listed)
- The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;
- The patient is at risk of reactivation of tuberculosis or has regular contact with individuals who are being actively treated for tuberculosis;
- The patient has used any investigational product (other than alglucosidase alfa) within 30 days prior to study enrollment;
- The patient is pregnant or lactating;
- The patient has had or is required to have any live vaccination within one month prior to enrollment.
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 |
|---|---|---|
| Rituximab | Drug | ITT |
| intravenous immune globulin | Drug | ITT |
| Bortezomib | Drug | ITT |
| Methotrexate | Drug | ITT |
Primary outcome
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2016.
- Derived from this study: PubMed 38715621Chen HA, Hsu RH, Fang CY, Desai AK, Lee NC, Hwu WL, Tsai FJ, Kishnani PS, Chien YH. Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy. Front Immunol. 2024 Apr 23;15:1336599. doi: 10.3389/fimmu.2024.1336599. eCollection 2024.
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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.
Yin-Hsiu Chien principal investigator
National Taiwan University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Rituximab, intravenous immune globulin and Bortezomib in people with Pompe Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
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 5 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 15 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yin-Hsiu Chien (+886223123456, chienyh@ntu.edu.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease (time frame: 6 months). 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 NCT02525172, 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 15 Apr 2016; AzTrials last refreshed its copy on 28 Oct 2026.