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Trial record 76 of 103 for:    Pompe Disease

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

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ClinicalTrials.gov Identifier: NCT01597596
Recruitment Status : Terminated (Study was stopped due to approved label expansion of alglucosidase alfa.)
First Posted : May 14, 2012
Results First Posted : January 18, 2016
Last Update Posted : January 18, 2016
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Pompe Disease (Infantile-Onset)
Glycogen Storage Disease Type II (GSD II)
Glycogenosis 2
Acid Maltase Deficiency
Intervention Biological: alglucosidase alfa
Enrollment 4
Recruitment Details The study was conducted between 21 August 2012 and 1 December 2014.
Pre-assignment Details A total of 5 participants were screened and 4 participants were treated.
Arm/Group Title Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Hide Arm/Group Description Alglucosidase alfa (4000 L material) 20 mg/kg intravenous (IV) infusion every other week (QOW) for 52 weeks. Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Period Title: Overall Study
Started 1 3
Completed 0 2
Not Completed 1 1
Reason Not Completed
Study terminated by sponsor             1             0
Physician Decision             0             1
Arm/Group Title Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants) Total
Hide Arm/Group Description Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks. Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks. Total of all reporting groups
Overall Number of Baseline Participants 1 3 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 1 participants 3 participants 4 participants
0.3 0.5  (0.28) 0.4  (0.14)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 3 participants 4 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
1
 100.0%
3
 100.0%
4
 100.0%
1.Primary Outcome
Title Change From Baseline in Cardiac Function at Week 52
Hide Description Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population defined as all participants who receive at least 1 infusion of alglucosidase alfa. For this endpoint no participants were analyzed in 'Algucosidase Alfa 4000 L material’ arm at Baseline and Week 52. One participant from ‘Algucosidase Alfa 160 L Material’ arm was discontinued from study at Week 31 due to physician’s decision
Arm/Group Title Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Hide Arm/Group Description:
Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Overall Number of Participants Analyzed 0 2
Mean (Standard Deviation)
Unit of Measure: Z-score
-5.06  (1.103)
2.Secondary Outcome
Title Percentage of Participants With Estimated Probability of Survival
Hide Description [Not Specified]
Time Frame Up to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population.
Arm/Group Title Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Hide Arm/Group Description:
Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Overall Number of Participants Analyzed 1 3
Measure Type: Number
Unit of Measure: percentage of participants
100 100
3.Secondary Outcome
Title Number of Participants With Invasive Ventilator-Free Survival
Hide Description Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported.
Time Frame Up to Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population.
Arm/Group Title Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Hide Arm/Group Description:
Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Overall Number of Participants Analyzed 1 3
Measure Type: Number
Unit of Measure: participants
1 2
4.Secondary Outcome
Title Change From Baseline in Motor Development Status at Week 52
Hide Description Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item was 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). The score for each dimension was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis population. For this endpoint no participants were analyzed in 'Algucosidase Alfa 4000 L material’ arm at Baseline and Week 52. One participant from ‘Algucosidase Alfa 160 L Material’ arm was discontinued from study at Week 31 due to physician’s decision.
Arm/Group Title Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Hide Arm/Group Description:
Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
Overall Number of Participants Analyzed 0 2
Mean (Standard Deviation)
Unit of Measure: percentage of maximum total score
48.65  (17.183)
Time Frame All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (Week 52) regardless of seriousness or relationship to investigational product.
Adverse Event Reporting Description Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the ‘on treatment period' (the period from the first infusion date to the date that the last data were collected). Analysis was carried out on full analysis population.
 
Arm/Group Title Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Hide Arm/Group Description Alglucosidase alfa (4000 L material) 20 mg/kg IV infusion QOW for 52 weeks. Alglucosidase alfa (160 L material) 20 mg/kg IV infusion QOW for 52 weeks.
All-Cause Mortality
Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Affected / at Risk (%) Affected / at Risk (%)
Total   0/1 (0.00%)   3/3 (100.00%) 
Cardiac disorders     
Cardiac failure  1  0/1 (0.00%)  1/3 (33.33%) 
General disorders     
Pyrexia  1  0/1 (0.00%)  1/3 (33.33%) 
Infections and infestations     
Adenovirus infection  1  0/1 (0.00%)  1/3 (33.33%) 
Lobar pneumonia  1  0/1 (0.00%)  1/3 (33.33%) 
Otitis media acute  1  0/1 (0.00%)  1/3 (33.33%) 
Respiratory syncytial virus infection  1  0/1 (0.00%)  1/3 (33.33%) 
Upper respiratory tract infection  1  0/1 (0.00%)  1/3 (33.33%) 
Nervous system disorders     
Hypotonia  1  0/1 (0.00%)  1/3 (33.33%) 
Syncope  1  0/1 (0.00%)  1/3 (33.33%) 
Respiratory, thoracic and mediastinal disorders     
Pulmonary oedema  1  0/1 (0.00%)  1/3 (33.33%) 
Respiratory failure  1  0/1 (0.00%)  1/3 (33.33%) 
Skin and subcutaneous tissue disorders     
Urticaria  1  0/1 (0.00%)  1/3 (33.33%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Alglucosidase Alfa 4000 L Material (Non-US Participants) Alglucosidase Alfa 160 L Material (US Participants)
Affected / at Risk (%) Affected / at Risk (%)
Total   1/1 (100.00%)   3/3 (100.00%) 
Blood and lymphatic system disorders     
Anaemia  1  0/1 (0.00%)  1/3 (33.33%) 
Cardiac disorders     
Cardiac failure  1  0/1 (0.00%)  2/3 (66.67%) 
Cardiac failure congestive  1  0/1 (0.00%)  1/3 (33.33%) 
Cardiomyopathy  1  0/1 (0.00%)  1/3 (33.33%) 
Nodal rhythm  1  0/1 (0.00%)  1/3 (33.33%) 
Ear and labyrinth disorders     
Middle ear effusion  1  0/1 (0.00%)  1/3 (33.33%) 
Gastrointestinal disorders     
Abdominal pain  1  1/1 (100.00%)  0/3 (0.00%) 
Diarrhoea  1  0/1 (0.00%)  1/3 (33.33%) 
Vomiting  1  0/1 (0.00%)  1/3 (33.33%) 
General disorders     
Device occlusion  1  0/1 (0.00%)  1/3 (33.33%) 
Infusion site erythema  1  0/1 (0.00%)  1/3 (33.33%) 
Pyrexia  1  1/1 (100.00%)  3/3 (100.00%) 
Infections and infestations     
Bronchitis  1  0/1 (0.00%)  1/3 (33.33%) 
Croup infectious  1  0/1 (0.00%)  1/3 (33.33%) 
Eye infection  1  0/1 (0.00%)  1/3 (33.33%) 
Fungal infection  1  0/1 (0.00%)  1/3 (33.33%) 
Nasopharyngitis  1  0/1 (0.00%)  1/3 (33.33%) 
Pneumonia  1  0/1 (0.00%)  1/3 (33.33%) 
Urinary tract infection  1  0/1 (0.00%)  1/3 (33.33%) 
Injury, poisoning and procedural complications     
Arthropod bite  1  0/1 (0.00%)  1/3 (33.33%) 
Investigations     
Oxygen saturation decreased  1  0/1 (0.00%)  1/3 (33.33%) 
Musculoskeletal and connective tissue disorders     
Muscle contracture  1  0/1 (0.00%)  1/3 (33.33%) 
Nervous system disorders     
Nystagmus  1  0/1 (0.00%)  1/3 (33.33%) 
Respiratory, thoracic and mediastinal disorders     
Aspiration  1  0/1 (0.00%)  1/3 (33.33%) 
Asthma  1  0/1 (0.00%)  1/3 (33.33%) 
Bronchial secretion retention  1  1/1 (100.00%)  0/3 (0.00%) 
Cough  1  0/1 (0.00%)  1/3 (33.33%) 
Hypoxia  1  0/1 (0.00%)  1/3 (33.33%) 
Nasal congestion  1  0/1 (0.00%)  1/3 (33.33%) 
Pleural effusion  1  0/1 (0.00%)  1/3 (33.33%) 
Respiratory disorder  1  0/1 (0.00%)  1/3 (33.33%) 
Rhinorrhoea  1  0/1 (0.00%)  1/3 (33.33%) 
Skin and subcutaneous tissue disorders     
Dermatitis contact  1  0/1 (0.00%)  1/3 (33.33%) 
Dermatitis diaper  1  0/1 (0.00%)  1/3 (33.33%) 
Rash erythematous  1  0/1 (0.00%)  1/3 (33.33%) 
Rash pruritic  1  0/1 (0.00%)  1/3 (33.33%) 
Urticaria  1  0/1 (0.00%)  1/3 (33.33%) 
Vascular disorders     
Hypotension  1  0/1 (0.00%)  1/3 (33.33%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17.1
Certain Agreements
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
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01597596     History of Changes
Other Study ID Numbers: AGLU07510
2011-005595-42 ( EudraCT Number )
First Submitted: May 10, 2012
First Posted: May 14, 2012
Results First Submitted: September 25, 2015
Results First Posted: January 18, 2016
Last Update Posted: January 18, 2016