Immune Modulation Therapy for Pompe Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-08-31

Study Completion Date

2020-07-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Treatment of ITP With Rituximab and / or Accutane

NCT02757196

Immune Thrombocytopenia

UNKNOWN PHASE2

Low-dose Rituximab Regimens in Chinese Adult Patients With Immune Thrombocytopenia

NCT01719692

Immune Thrombocytopenic Purpura

COMPLETED NA

Phase II Study of Rituximab in Patients With Immune Thrombocytopenic Purpura

NCT00005652

Purpura, Thrombocytopenic, Idiopathic

COMPLETED PHASE2

Efficacy of Immunomudulatory Therapy With All-trans Retinoid Acid for Adults With Chronic Immune Thrombocytopenia

NCT01668615

Immune Thrombocytopenic Purpura

COMPLETED

A Single-center Clinical Trial of Bortezomib in Management of Immune Thrombocytopenia (ITP)

NCT03013114

Immune Thrombocytopenia

UNKNOWN PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Pompe Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

ITT

immune modulation therapy

Group Type EXPERIMENTAL

Rituximab

Intervention Type DRUG

intravenous immune globulin

Intervention Type DRUG

Bortezomib

Intervention Type DRUG

Methotrexate

Intervention Type DRUG

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Rituximab

Intervention Type DRUG

intravenous immune globulin

Intervention Type DRUG

Bortezomib

Intervention Type DRUG

Methotrexate

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No