NCT02525172

Brief 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
8

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Aug 2015

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2015

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

August 10, 2015

Completed
7 days until next milestone

First Posted

Study publicly available on registry

August 17, 2015

Completed
4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2020

Completed
Last Updated

April 15, 2016

Status Verified

April 1, 2016

Enrollment Period

4.9 years

First QC Date

August 10, 2015

Last Update Submit

April 13, 2016

Conditions

Pompe Disease

Keywords

immune modulation therapyacid α-glucosidaseantibody

Outcome Measures

Primary Outcomes (1)

  • anti-recombinant human acid α-glucosidase (anti-rhGAA) antibody titers decrease

    6 months

Study Arms (1)

ITT

EXPERIMENTAL

immune modulation therapy

Drug: RituximabDrug: intravenous immune globulinDrug: BortezomibDrug: Methotrexate

Interventions

RituximabDRUG
ITT
intravenous immune globulinDRUG
ITT
BortezomibDRUG
ITT
MethotrexateDRUG
ITT

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Yin-Hsiu Chien

Taipei, 100, Taiwan

RECRUITING

Related Publications (1)

  • 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.

    PMID: 38715621DERIVED

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

RituximabImmunoglobulins, IntravenousBortezomibMethotrexate

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-DerivedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsImmunoglobulin GImmunoglobulin IsotypesBoronic AcidsAcids, NoncarboxylicAcidsInorganic ChemicalsBoron CompoundsOrganic ChemicalsPyrazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsAminopterinPterinsPteridinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Study Officials

  • Yin-Hsiu Chien

    National Taiwan University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yin-Hsiu Chien

CONTACT

+886223123456chienyh@ntu.edu.tw

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 10, 2015

First Posted

August 17, 2015

Study Start

August 1, 2015

Primary Completion

July 1, 2020

Study Completion

July 1, 2020

Last Updated

April 15, 2016

Record last verified: 2016-04

Locations

TW(1)