Trial Outcomes & Findings for Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa (NCT NCT01288027)
NCT ID: NCT01288027
Last Updated: 2014-12-19
Results Overview
Tissue glycogen content was measured by quadriceps biopsies as 'percent area of tissue occupied by glycogen'.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
16 participants
Primary outcome timeframe
Baseline, Week 26
Results posted on
2014-12-19
Participant Flow
The study was conducted at 11 centers between July 06, 2011 and December 19, 2013.
Participant milestones
| Measure |
Alglucosidase Alfa
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
Full Analysis Set (FAS)
|
16
|
|
Overall Study
COMPLETED
|
16
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
Baseline characteristics by cohort
| Measure |
Alglucosidase Alfa
n=16 Participants
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Age, Continuous
|
51.6 years
STANDARD_DEVIATION 13.69 • n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 26Population: FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here, n = number of participants with both Baseline and Week 26 assessment of tissue glycogen content.
Tissue glycogen content was measured by quadriceps biopsies as 'percent area of tissue occupied by glycogen'.
Outcome measures
| Measure |
Alglucosidase Alfa
n=16 Participants
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26
Baseline (n=14)
|
5.3 percent area occupied by glycogen
Standard Deviation 4.59
|
|
Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26
Change at Week 26 (n=13)
|
-1.6 percent area occupied by glycogen
Standard Deviation 4.11
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 26Population: No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 26Population: No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 26Population: FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here, Number of participants analyzed= participants with both Baseline and Week 26 assessment of muscle involvement, n= number of participants with both Baseline and Week 26 assessment of muscle involvement for specified category.
Muscle involvement was assessed by T1-weighted magnetic resonance imaging (MRI). T1-weighted MRI data was analyzed using the Mercuri scoring in both legs (Total score = 1-4; where 1=Normal appearance, 2=Mild involvement, 3=Moderate involvement, and 4=Severe involvement). For each participants, the average for each the upper (thigh) and lower leg was computed for Mercuri grading.
Outcome measures
| Measure |
Alglucosidase Alfa
n=14 Participants
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Percent Change From Baseline in Muscle Involvement Using Mercuri Scoring at Week 26
Mercuri Scoring - Lower Leg (n=14)
|
0.0 percent change
Standard Deviation 0.0
|
|
Percent Change From Baseline in Muscle Involvement Using Mercuri Scoring at Week 26
Mercuri Scoring - Upper Leg (n=13)
|
2.6 percent change
Standard Deviation 9.24
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here, number of participants analyzed = number of participants with both Baseline and Week 26 assessment of degree of fatty infiltration.
Degree of Fatty Infiltration was assessed by 3-point 3D Dixon acquisition using skeletal muscle MRI in a subset of participants.
Outcome measures
| Measure |
Alglucosidase Alfa
n=3 Participants
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Percent Change From Baseline in Degree of Fatty Infiltration Using 3-Point 3-Dimensional (3D) Dixon at Week 26
|
2.0 percent change
Standard Deviation 12.83
|
SECONDARY outcome
Timeframe: Baseline, Week 26Population: FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here Number of participants analyzed = number of participants with both baseline and Week 26 assessment of disease activity.
Disease activity (inflammation and/or water content within muscles) was quantitatively assessed by T2 MRI values in a subset of participants. A T2 MRI value of greater than (\>) 39 millisecond (ms) was defined as abnormal. T2 estimation normally requires an additional acquisition for computing the B1 spatial deviation however, can still be estimated if this acquisition is missing.
Outcome measures
| Measure |
Alglucosidase Alfa
n=6 Participants
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Percent Change From Baseline in Disease Activity Using T2 Magnetic Resonance Imaging (MRI) at Week 26
T2 with B1
|
8.1 percent change
Standard Deviation 14.19
|
|
Percent Change From Baseline in Disease Activity Using T2 Magnetic Resonance Imaging (MRI) at Week 26
T2 without B1
|
7.2 percent change
Standard Deviation 13.58
|
Adverse Events
Alglucosidase Alfa
Serious events: 1 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Alglucosidase Alfa
n=16 participants at risk
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Eye disorders
Retinal vascular thrombosis
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
Other adverse events
| Measure |
Alglucosidase Alfa
n=16 participants at risk
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
|
|---|---|
|
Cardiac disorders
Bradycardia
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Ear and labyrinth disorders
Deafness
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Ear and labyrinth disorders
Tympanic membrane perforation
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Eye disorders
Ocular hyperaemia
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Gastrointestinal disorders
Nausea
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Gastrointestinal disorders
Vomiting
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
General disorders
Adverse drug reaction
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
General disorders
Fatigue
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
General disorders
Feeling cold
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
General disorders
Influenza like illness
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
General disorders
Injection site extravasation
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
General disorders
Malaise
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Infections and infestations
Bronchitis
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Infections and infestations
Ear infection
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Infections and infestations
Nasopharyngitis
|
31.2%
5/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Infections and infestations
Rhinitis
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Infections and infestations
Subcutaneous abscess
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Infections and infestations
Upper respiratory tract infection
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
18.8%
3/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Investigations
Heart rate increased
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Nervous system disorders
Dizziness
|
18.8%
3/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Nervous system disorders
Headache
|
25.0%
4/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Nervous system disorders
Muscle contractions involuntary
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Nervous system disorders
Paraesthesia
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Nervous system disorders
Sciatica
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Psychiatric disorders
Insomnia
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Reproductive system and breast disorders
Epididymitis
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal oedema
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Skin and subcutaneous tissue disorders
Rash
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Vascular disorders
Flushing
|
12.5%
2/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Vascular disorders
Hypotension
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Vascular disorders
Peripheral coldness
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
|
Vascular disorders
Thrombosis
|
6.2%
1/16 • From signature of the informed consent form up to the final visit (Week 26)
In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER