- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02525172
Immune Modulation Therapy for Pompe Disease
April 13, 2016 updated by: National Taiwan University Hospital
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.
Study Overview
Status
Unknown
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Anticipated)
8
Phase
- Phase 4
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Yin-Hsiu Chien
- Phone Number: +886223123456
- Email: chienyh@ntu.edu.tw
Study Locations
-
-
-
Taipei, Taiwan, 100
- Recruiting
- Yin-Hsiu Chien
-
Contact:
- Yin-Hsiu Chien
- Phone Number: +886223123456
- Email: chienyh@ntu.edu.tw
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: ITT
immune modulation therapy
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease
Time Frame: 6 months
|
6 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
August 1, 2015
Primary Completion (Anticipated)
July 1, 2020
Study Completion (Anticipated)
July 1, 2020
Study Registration Dates
First Submitted
August 10, 2015
First Submitted That Met QC Criteria
August 14, 2015
First Posted (Estimate)
August 17, 2015
Study Record Updates
Last Update Posted (Estimate)
April 15, 2016
Last Update Submitted That Met QC Criteria
April 13, 2016
Last Verified
April 1, 2016
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Lysosomal Storage Diseases, Nervous System
- Glycogen Storage Disease
- Glycogen Storage Disease Type II
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Nucleic Acid Synthesis Inhibitors
- Enzyme Inhibitors
- Antirheumatic Agents
- Antimetabolites, Antineoplastic
- Antimetabolites
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Antineoplastic Agents, Immunological
- Dermatologic Agents
- Reproductive Control Agents
- Abortifacient Agents, Nonsteroidal
- Abortifacient Agents
- Folic Acid Antagonists
- Antibodies
- Immunoglobulins
- Immunoglobulins, Intravenous
- Rituximab
- Bortezomib
- Methotrexate
- gamma-Globulins
- Rho(D) Immune Globulin
Other Study ID Numbers
- 201504036MIPB
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Pompe Disease
-
University of Erlangen-Nürnberg Medical SchoolRecruitingPompe Disease (Late-onset) | Pompe Disease | Pompe's Disease Juvenile Onset | Pompe Disease Infantile-OnsetGermany
-
Centre for Analytical Biochemistry and Biomedical...University of GiessenRecruiting
-
GeneCradle IncRecruitingPompe Disease Infantile-OnsetChina
-
Seventh Medical Center of PLA General HospitalGeneCradle Therapeutics, IncRecruiting
-
Fundació Institut de Recerca de l'Hospital de la...Genzyme, a Sanofi CompanyUnknownPompe Disease | Late Onset Pompe DiseaseSpain
-
Astellas Gene TherapiesRecruitingPompe Disease (Late-onset)United States, Taiwan, France, Spain
-
Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
-
Duke UniversityCompleted
-
Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
-
BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
Clinical Trials on Methotrexate
-
University Hospital, MontpellierPfizer; Hôpital CochinCompletedRheumatoid ArthritisFrance, Monaco
-
Nicolaus Copernicus UniversityCompleted
-
Amneal Pharmaceuticals, LLCAccutest Research Laboratories (I) Pvt. Ltd.Unknown
-
Hee Young JuNot yet recruitingLymphoblastic Leukemia in Children
-
PfizerCompletedRheumatoid ArthritisUnited States, Mexico, Argentina, Chile, Croatia, Czech Republic, Hungary, Poland, Puerto Rico
-
PfizerCompletedRhematoid ArthritisSpain, United Kingdom, United States, Korea, Republic of, Poland, Israel, Australia, Taiwan, Thailand, South Africa, Bulgaria, Estonia, Latvia, Philippines, Canada, Romania, Russian Federation, Turkey, Mexico, Bosnia and Herzegovina and more
-
Cairo UniversityCompleted
-
Chugai Pharma TaiwanCompletedRheumatoid Arthritis (RA)Taiwan
-
CHA UniversityCompleted
-
Czech Lymphoma Study GroupUnknownDiffuse Large B-cell LymphomaCzech Republic