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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00483379




Registration number
NCT00483379
Ethics application status
Date submitted
6/06/2007
Date registered
7/06/2007
Date last updated
7/03/2014

Titles & IDs
Public title
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Scientific title
An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen
Secondary ID [1] 0 0
AGLU03306
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pompe Disease 0 0
Glycogen Storage Disease Type II (GSD-II) 0 0
Glycogenesis 2 Acid Maltase Deficiency 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - alglucosidase alfa

Experimental: alglucosidase alfa 20 mg/kg every week - Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.

Experimental: alglucosidase alfa 40 mg/kg every other week - Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.


Treatment: Other: alglucosidase alfa
intravenous infusion
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment
Timepoint [1] 0 0
Baseline, Week 52
Primary outcome [2] 0 0
Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Motor Function Decline on Standard Treatment
Timepoint [2] 0 0
Baseline, Week 52
Primary outcome [3] 0 0
Summary of Participants Reporting Treatment-Emergent Adverse Events During the Treatment Period
Timepoint [3] 0 0
Day 1 up to Week 52
Secondary outcome [1] 0 0
Baseline Values for Left Ventricular Mass (LVM) Z-Scores
Timepoint [1] 0 0
Day 0
Secondary outcome [2] 0 0
Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Baseline Values for Left Ventricular Mass Index (LVMI)
Timepoint [3] 0 0
Day 0
Secondary outcome [4] 0 0
Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52
Timepoint [4] 0 0
Baseline, Week 52
Secondary outcome [5] 0 0
Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)
Timepoint [5] 0 0
Baseline, approximately Week 52
Secondary outcome [6] 0 0
Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52
Timepoint [6] 0 0
Baseline, Week 52
Secondary outcome [7] 0 0
Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results
Timepoint [7] 0 0
Day 0
Secondary outcome [8] 0 0
Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52
Timepoint [8] 0 0
Baseline, Week 52
Secondary outcome [9] 0 0
Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
Timepoint [9] 0 0
Day 0
Secondary outcome [10] 0 0
Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52
Timepoint [10] 0 0
Baseline, Week 52
Secondary outcome [11] 0 0
Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)
Timepoint [11] 0 0
Day 0
Secondary outcome [12] 0 0
Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52
Timepoint [12] 0 0
Baseline, Week 52

Eligibility
Key inclusion criteria
* The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures;
* The patient must have a clinical diagnosis of Pompe disease as defined by documented GAA deficiency in skin fibroblasts or blood;
* The patient must have been compliant with the standard dosing regimen of alglucosidase alfa (20 mg/kg every other week) for a minimum of 6 months immediately prior to study entry
* The patient must have clinical decline or sub-optimal improvement in at least one of the following parameters as compared to their condition prior to the beginning alglucosidase alfa treatment:

1. Cardiac: Left Ventricular Mass (LVM) Z-score =6 or LVM index =150 g/m2 after a minimum of 6 months of regular treatment with alglucosidase alfa; OR
2. Respiratory: New development of respiratory failure requiring the use of ventilatory assistance (invasive or non-invasive) after a minimum of 6 months of regular treatment with alglucosidase alfa. Ventilatory assistance must have been required for at least 4 weeks prior to study enrollment; OR
3. Motor Skills:
* For patients = 2 years of age at study entry, failure to acquire at least 2 new gross motor milestones after a minimum of 6 months of regular treatment with alglucosidase alfa; OR
* For patients > 2 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through loss of functional use of the upper extremities after a minimum of 6 months of regular treatment with alglucosidase alfa, OR
* For patients > 8 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through longitudinal assessments of manual muscle testing after a minimum of 6 months of regular treatment with alglucosidase alfa, OR
* For patients previously ambulatory, progression to use of an assistive device for ambulation due to worsening of proximal lower extremity muscle weakness after a minimum of 6 months of regular treatment with alglucosidase alfa.
Minimum age
6 Months
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* For patients < 18 years of age, negative Cross-Reactive Immunologic Material (CRIM) assay result (added in protocol amendment #2);
* Any medical condition which, in the opinion of the Investigator, could interfere with treatment or evaluation of safety and/or efficacy of alglucosidase alfa;
* The patient is not currently receiving alglucosidase alfa;
* The patient has major congenital abnormality;
* The patient has used any investigational product (other than alglucosidase alfa in those regions where the product is not commercially available) within 30 days prior to study enrollment;
* The patient is pregnant or lactating.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 4
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/05/2007
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/07/2010
Sample size
Target
Accrual to date
Final
13
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Parkville Victoria
Recruitment postcode(s) [1] 0 0
- Parkville Victoria
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
Canada
State/province [10] 0 0
Alberta

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Genzyme, a Sanofi Company
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT00483379