Trial record 1 of 1 for:
aglu03807
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00701129 |
Recruitment Status :
Completed
First Posted : June 19, 2008
Results First Posted : May 13, 2014
Last Update Posted : May 13, 2014
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Sponsor:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Conditions |
Pompe Disease Glycogen Storage Disease Type II |
Interventions |
Biological: Alglucosidase Alfa Drug: Methotrexate Drug: Rituximab |
Enrollment | 4 |
Participant Flow
Recruitment Details | The study was conducted at 2 centers in the United States of America between October 01, 2009 and March 27, 2013. |
Pre-assignment Details |
Arm/Group Title | Alglucosidase Alfa |
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Alglucosidase alfa (Myozyme®) 20 milligrams per kilogram (mg/kg) intravenous (IV) infusion every other week (qow) (or optionally 20 mg/kg IV infusion every week [qw]) beginning from Day 0 to a minimum of 18 months or if the patient was less than (<) 6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 milligrams per square meter (mg/m^2) (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information. |
Period Title: Overall Study | |
Started | 4 |
Full Analysis Set (FAS) | 4 [1] |
Completed | 2 |
Not Completed | 2 |
Reason Not Completed | |
Death | 2 |
[1]
All enrolled patients who signed informed consent, received at least 1 dose of alglucosidase alfa.
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Baseline Characteristics
Arm/Group Title | Alglucosidase Alfa | |
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Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week [qw]) beginning from Day 0 to a minimum of 18 months or if the patient was <6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information. | |
Overall Number of Baseline Participants | 4 | |
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Safety population included all patients who received at least one dose of alglucosidase alfa.
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Age, Customized
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 4 participants |
Less Than or Equal to (=<) 6 Months | 2 | |
Greater Than (>) 6 Months | 2 | |
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 4 participants | |
Female |
3 75.0%
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Male |
1 25.0%
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Race/Ethnicity, Customized
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 4 participants |
Race: Black | 2 | |
Race: White | 1 | |
Race: White, Black | 1 | |
Ethnicity: Hispanic | 2 | |
Ethnicity: Non Hispanic | 2 | |
Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G
[1] Measure Type: Number Unit of measure: Participants |
Number Analyzed | 4 participants |
Negative | 4 | |
Positive | 0 | |
[1]
Measure Description: As all patients were treatment naïve, it was expected that no patient would be Anti-rhGAA immunoglobulin G (IgG) antibody positive at baseline.
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Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index
[1] Measure Type: Number Unit of measure: Participants |
Number Analyzed | 4 participants |
LVM Z-score: Normal | 0 | |
LVM Z-score: Abnormal | 4 | |
LVM index: Normal | 0 | |
LVM index: Abnormal | 4 | |
[1]
Measure Description: LVM Z-Score and LVM Index were assessed by echocardiography (ECHO). LVM Z-Score: an indicator of degree of standard deviations from mean in a normal distribution. Negative values indicate a smaller LVM than mean and values higher than 0 indicate a larger LVM than mean. Normal range is -2 to 2; values <-2 or >2 indicate abnormal score. LVM index: an index value derived by normalizing LVM by body surface area. LVM index provides evidence of cardiomyopathy. LVM index values <65 gram per square meter (g/m^2) were considered as normal and LVM index values >=65 g/m^2 were considered as abnormal.
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Number of Patients With Ventilator Use
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 4 participants |
Yes | 3 | |
No | 1 | |
Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88)
[1] Median (Full Range) Unit of measure: Percentage of maximum total score |
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Number Analyzed | 4 participants | |
5.10
(0.39 to 7.49)
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[1]
Measure Description: GMFM-88:an 88-item measure to detect gross motor function; consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item is scored on a 4-point Likert scale(0=cannot do; 1=initiates [<10% of the task]; 2=partially completes [10% to <100% of the task]; 3=task completion). Score for each dimension is expressed as a percentage of maximum score for that dimension. Total score=sum of percentage scores for each dimension divided by number of dimensions. Total score range: 0% to 100%, where higher scores indicate better motor functions.
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Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS)
[1] Median (Full Range) Unit of measure: Months |
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Number Analyzed | 4 participants | |
0
(0 to 1.37)
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[1]
Measure Description: AIMS:58-item in 4 subscales: prone; supine; sitting; and standing. Each item is scored as observed/not observed. Item in observed range create a motor window. Subscale scores are calculated by giving child 1 point for observed items within motor window in addition to being given 1 point for all less mature items before motor window. AIMS total score=sum of scores for 58 items, range: 0-58,higher score=more mature motor development. Score was then compared with age-equivalent peers from normative sample and equivalence level age (in months) is reported. Here, number of patients analyzed = 3.
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Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
[1] Median (Full Range) Unit of measure: Units on a scale |
Number Analyzed | 4 participants |
Functional Skills: Mobility Score (n=4) |
4.5
(0 to 18.4)
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Functional Skills: Self-Care Score (n=4) |
14.4
(4.9 to 16.1)
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Functional Skills: Social Function Score (n=1) |
10.5
(10.5 to 10.5)
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Caregiver Assistance: Mobility Score (n=2) |
0
(0 to 0)
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Caregiver Assistance: Self-Care Score (n=2) |
0
(0 to 0)
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Caregiver Assistance: Social Function Score (n=2) |
0
(0 to 0)
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[1]
Measure Description: It consists of all items of original PEDI (197 functional skill items in 3 domains:self-care;mobility;social function) and additional items in functional skills,mobility,self-care domains to reflect functional skills for children with Pompe. Each domain consists of 2 subdomains: functional skill performance, caregiver assistance scale. Norm-based scoring was developed for additional items, and scoring for PEDI have been adjusted to reflect additional normative data collected for Pompe PEDI. Total score range for each domain (mean of subdomains) and subdomain=0-100;higher score=high capability.
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Outcome Measures
Adverse Events
Limitations and Caveats
Due to the small number of patients assessed in this study the results must be interpreted with caution.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
Results Point of Contact
Name/Title: | Trial Transparency Team |
Organization: | Sanofi |
EMail: | Contact-us@sanofi.com |
Responsible Party: | Sanofi ( Genzyme, a Sanofi Company ) |
ClinicalTrials.gov Identifier: | NCT00701129 |
Other Study ID Numbers: |
AGLU03807 MSC12862 ( Other Identifier: Sanofi ) |
First Submitted: | June 17, 2008 |
First Posted: | June 19, 2008 |
Results First Submitted: | February 24, 2014 |
Results First Posted: | May 13, 2014 |
Last Update Posted: | May 13, 2014 |