Immune Tolerance Induction Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00701701
• Date of registration: 17/06/2008
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: Immune Tolerance Induction Study
• Scientific title: An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients With Pompe Disease Who Have Previously Received Myozyme
• Date of first enrolment: December 14, 2008
• Target sample size: 4
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00701701
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Canada, Israel, United States

Contacts

• Name: Medical Monitor
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion criteria

Inclusion Criteria:

- The participant (and/or participant's legal guardian if participant was < 18 years)
provided written informed consent prior to any study-related procedures that were
performed.

- The participants had a confirmed diagnosis of Pompe disease defined as a documented
acid a-glucosidase (GAA) enzyme deficiency from any tissue source or 2 GAA gene
mutations.

- The participant (and/or legal guardian) had ability to comply with clinical protocol.

- If the participant was CRIM-positive, he/she had received at least 6 consecutive
months of Myozyme® infusions (20 mg/kg qow).

- If the participant was CRIM-negative, he/she had received at least 1 Myozyme® infusion
prior to enrollment.

- Regimen A only: The participants exhibits clinical decline; The participant had
persistent high anti-recombinant human acid a-glucosidase (anti-rhGAA) antibody titers
and/or tested positive for antibodies that inhibit enzymatic activity and/or uptake of
Myozyme®;

- Regimen B only: The participant was CRIM-negative AND The participant did not exhibit
clinical decline; OR all of the following: The participant was CRIM-negative AND The
participant exhibited clinical decline AND The participant did not exhibit high
anti-rhGAA antibody titers and had not tested positive for antibodies that inhibit
enzymatic activity and/or uptake of Myozyme®.

Exclusion Criteria:

- The participant had a clinical condition unrelated to Pompe disease that would
interfere with program assessments.

- The participant was at risk of reactivation or was a carrier of Hepatitis B or
Hepatitis C.

- The participant was at risk of reactivation or had positive serology suggestive of
active infection for cytomegalovirus, Herpes simplex, JC virus, Parvovirus or Epstein
Barr virus.

- The participant was at risk of reactivation of tuberculosis or had regular contact
with individuals who were being actively treated for tuberculosis.

- The participant had low serum albumin.

- The participant had a major congenital abnormality.

- The participant had used any investigational product (other than alglucosidase alfa)
within 30 days prior to study enrollment.

- The participant was pregnant or lactating.

- The participant has had or was required to have any live vaccination within one month
prior to enrollment.



Age minimum: 1 Month
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Glycogen Storage Disease Type II (GSD-II)

Glycogenesis 2 Acid Maltase Deficiency

Pompe Disease

Intervention(s)

Biological: Myozyme® (alglucosidase alfa)

Primary Outcome(s)

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [Time Frame: From Baseline up to 18 months]

Secondary ID(s)

AGLU03707

MSC12817

2015-000583-34

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/10/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00701701