Trial Outcomes & Findings for Dosing Study of Replagal in Patients With Fabry Disease (NCT NCT00068107)

Last Updated: 2015-03-31

Results Overview

The rate of decline in renal function, as measured by estimation of glomerular filtration rate at baseline when participants were receiving agalsidase alfa (Relagal) every 2 weeks and when participants were receiving weekly infusion of Relagal.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

Relagal was administered every 2 weeks for 2-4 years pre-study, Relagal was administred weekly during the study (approx. 4.5-10 years)

Results posted on

2015-03-31

Participant Flow

13 participants were recruited; 1 participant was excluded because of stable kidney function; 12 started treatment

Participant milestones

Participant milestones
Measure	All Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month. Patients will receive a dose of 0.2 mg/kg of body weight every week.
Overall Study STARTED	12
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	All Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month. Patients will receive a dose of 0.2 mg/kg of body weight every week.
Overall Study end-stage renal disease	6
Overall Study Death	2
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Dosing Study of Replagal in Patients With Fabry Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=13 Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Age, Continuous	41 years STANDARD_DEVIATION 9 • n=99 Participants
Sex: Female, Male Female	0 Participants n=99 Participants
Sex: Female, Male Male	13 Participants n=99 Participants
Region of Enrollment United States	13 participants n=99 Participants

PRIMARY outcome

Timeframe: Relagal was administered every 2 weeks for 2-4 years pre-study, Relagal was administred weekly during the study (approx. 4.5-10 years)

Outcome measures

Outcome measures
Measure	All Participants n=12 Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.	All Participants at Last Observation
Estimated Glomerular Filtration Rate (eGFR) eGFR on infusions every 2 wks	-0.66 ml/min/month Standard Deviation 0.24	—
Estimated Glomerular Filtration Rate (eGFR) eGFR on infusions weekly	-0.32 ml/min/month Standard Deviation 0.34	—

SECONDARY outcome

Timeframe: Baseline and last observation (up to 10 years)

Outcome measures

Outcome measures
Measure	All Participants n=11 Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.	All Participants at Last Observation n=11 Participants
Globotriaosylceramide (Gb(3)) in Plasma participant 1	2.35 nanomole/mL	4.10 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 9	4.25 nanomole/mL	1.91 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 10	2.59 nanomole/mL	3.41 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 11	6.62 nanomole/mL	5.62 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 2	4.62 nanomole/mL	5.58 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 3	2.43 nanomole/mL	2.62 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 5	3.17 nanomole/mL	3.60 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 6	6.78 nanomole/mL	5.04 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 7	3.32 nanomole/mL	3.21 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 8	3.69 nanomole/mL	4.51 nanomole/mL
Globotriaosylceramide (Gb(3)) in Plasma participant 12	6.56 nanomole/mL	6.63 nanomole/mL

SECONDARY outcome

Timeframe: Baseline and last observation (up to 10 years)

Outcome measures

Outcome measures
Measure	All Participants n=11 Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.	All Participants at Last Observation n=11 Participants
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 1	49 nanomole/(gram of Creatinine)	1888 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 2	126 nanomole/(gram of Creatinine)	89 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 3	248 nanomole/(gram of Creatinine)	82 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 5	136 nanomole/(gram of Creatinine)	162 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 6	3026 nanomole/(gram of Creatinine)	652 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 7	3128 nanomole/(gram of Creatinine)	1682 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 8	1393 nanomole/(gram of Creatinine)	450 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 9	137 nanomole/(gram of Creatinine)	370 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 10	1457 nanomole/(gram of Creatinine)	488 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 11	2342 nanomole/(gram of Creatinine)	213 nanomole/(gram of Creatinine)
Globotriaosylceramide (Gb(3)) in Urine Sediment participant 12	624 nanomole/(gram of Creatinine)	169 nanomole/(gram of Creatinine)

SECONDARY outcome

Timeframe: Baseline and last observation (up to 10 years)

Quantitative sudomotor axon reflex test (QSART) is a measure of sweat function

Outcome measures

Outcome measures
Measure	All Participants n=12 Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.	All Participants at Last Observation
Number of Participants With a Change in Quantitative Sudomotor Axon Reflex Test	0 participants	—

SECONDARY outcome

Timeframe: pre-study was 2-4 years, during study sensory testing measured for approx. 4.5-5 years

Population: Number of participants with a sufficient number of data points to estimate slope

For quantitative sensory testing, the outcome variable (detection threshold score) was analyzed for all combinations of location (foot, hand, and thigh) and test (cold, vibration, and warm). Possible threshold scores may range from 1-25. Score values of "\>25" were set to 25. Higher scores indicate higher sensory detection threshold. Therefore, lower score is better. Time, measured in years, was centered at the date in which patients switched treatment regimen. All available measurements were used. A linear mixed model analysis was used to test for differences in the linear association between time and detection threshold score pre-and-post ERT regiment change while accounting for the correlation among observations from the same individual. Specifically, the model contained a subject specific random intercept with year as a fixed effect and knot at time of the treatment change.

Outcome measures

Outcome measures
Measure	All Participants n=10 Participants All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.	All Participants at Last Observation
Quantitative Sensory Testing Vibration Sensory testing on Foot (pre-study)	0.3353 units on a scale/year Standard Error 0.1004	—
Quantitative Sensory Testing Vibration on Foot (difference pre- to post-)	-0.3912 units on a scale/year Standard Error 0.2041	—
Quantitative Sensory Testing Cold Sensory testing on Foot (pre-study)	-0.2108 units on a scale/year Standard Error 0.1366	—
Quantitative Sensory Testing Cold Sensory on Foot (difference pre- to post-)	0.3863 units on a scale/year Standard Error 0.2784	—
Quantitative Sensory Testing Warm Sensory testing on Foot (pre-study)	-0.08536 units on a scale/year Standard Error 0.09264	—
Quantitative Sensory Testing Warm on Foot (difference pre- to post-)	0.4169 units on a scale/year Standard Error 0.1887	—
Quantitative Sensory Testing Cold Sensory testing on Hand (pre-study)	-0.1026 units on a scale/year Standard Error 0.1413	—
Quantitative Sensory Testing Cold on Hand (difference pre- to post-)	0.5141 units on a scale/year Standard Error 0.2874	—
Quantitative Sensory Testing Vibration Sensory testing on Hand (pre-study)	0.2279 units on a scale/year Standard Error 0.07427	—
Quantitative Sensory Testing Vibration on Hand (difference pre- to post-)	-0.1966 units on a scale/year Standard Error 0.1503	—
Quantitative Sensory Testing Warm Sensory testing on Hand (pre-study)	-0.2138 units on a scale/year Standard Error 0.1179	—
Quantitative Sensory Testing Warm on Hand (difference pre- to post-)	1.1781 units on a scale/year Standard Error 0.2401	—
Quantitative Sensory Testing Cold Sensory testing on Thigh (pre-study)	0.2502 units on a scale/year Standard Error 0.1437	—
Quantitative Sensory Testing Cold on Thigh (difference pre- to post-)	-0.2991 units on a scale/year Standard Error 0.2884	—
Quantitative Sensory Testing Warm Sensory testing on Thigh (pre-study)	-0.549 units on a scale/year Standard Error 0.1124	—
Quantitative Sensory Testing Warm on Thigh (difference pre- to post-)	0.8255 units on a scale/year Standard Error 0.2258	—

SECONDARY outcome

Timeframe: 10 years

Population: Doppler skin blood flow was not collected in this study because it was judged to not be useful early in the study.

Outcome measures

Outcome data not reported

Adverse Events

All Participants

Serious events: 10 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Participants n=13 participants at risk All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.
Cardiac disorders myocardial infarction	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Sharp chest pain and right leg pain	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders syncopal episode	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders nausea and vomiting	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders pain crisis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders elective mitral valve annuloplasty	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders shortness of breath	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders increased intensity of angina	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders aortic stenosis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders ventricular tachycardia	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Infections and infestations bacterial sepsis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders recurrent ventricular arrhythmia	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Infections and infestations hepatitis C infection	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders aortic valve replacement	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders dizziness, tachycardia and chest pain	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders severe renal decline	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders clogged port-a-cath replacement and pacemaker adjustment	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders placement of a pacemaker due to a bradycardic episode	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders acute occipital ischemic stroke	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders death	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders catheter-induced peritonitis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Infections and infestations sepsis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders hypotension	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders advanced renal insufficiency	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.

Other adverse events

Other adverse events
Measure	All Participants n=13 participants at risk All participants were followed on a prior protocol and found to have a mean slope on Replagal (agalsidase alfa) infused every 2 weeks of -0.66 +- 0.24 ml/min/month.
Immune system disorders Allergies	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Blood and lymphatic system disorders Anemia	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Metabolism and nutrition disorders Decreased appetite	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders Arrythmia	46.2% 6/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Impaired balance	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Skin and subcutaneous tissue disorders Blisters	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Skin and subcutaneous tissue disorders Burn	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Cancer - skin	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders Cardiac palpitations	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Gastrointestinal disorders Cholethisiasis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Infections and infestations Cold symptoms	38.5% 5/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Creatinine worsened	30.8% 4/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Psychiatric disorders Depression	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Gastrointestinal disorders Diarrhea	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Dialysis	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Diaphoresis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Dizziness	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Dreams (abnormal)	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Skin and subcutaneous tissue disorders Ecchymosis - varied	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Vascular disorders Edema	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Infections and infestations Eosinophils - increased	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Electrolytes - elevated	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Skin and subcutaneous tissue disorders Erythema	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Fatigue	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Blood and lymphatic system disorders Ferritin - decreased	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Blood and lymphatic system disorders Ferritin - increased	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Fever	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Musculoskeletal and connective tissue disorders Fracture	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Gastrointestinal disorders GI symptoms	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Glucose - elevated	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Hair loss	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Headache	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Ear and labyrinth disorders Hearing Loss	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Cardiac disorders Heart Block	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Vascular disorders Hypertension	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Vascular disorders Hypotension	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Endocrine disorders Hypothyroid	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Infections and infestations Infection	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Injury, poisoning and procedural complications Injury - trauma	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Blood and lymphatic system disorders Iron - decreased	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Skin and subcutaneous tissue disorders Itching	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Kidney nodules/cysts	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Malaise	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Memory - decreased	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Mental status altered	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Microalbuminuria - worsened	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Numbness	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Musculoskeletal and connective tissue disorders Orthopedic	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - back	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - chest (no specific angina)	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - finger	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - GI	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - knee	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - leg (spastic)	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - neck area	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - pelvic area	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Pain - shoulder	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Respiratory, thoracic and mediastinal disorders Pneumonia	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Blood and lymphatic system disorders Potassium - elevated	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Proteinuria - increased	30.8% 4/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Prostatic hypertrophy	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Prostatitis	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders PSA - elevated	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Skin and subcutaneous tissue disorders Rash	30.8% 4/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Renal failure	23.1% 3/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Restless Leg Syndrome	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Respiratory, thoracic and mediastinal disorders Shortness of Breath	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Infections and infestations Sinus Infection	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Blood and lymphatic system disorders Sodium - elevated	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Speech - impaired	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Gastrointestinal disorders Stool leakage	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Stroke - acute ischemic	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Throat - dry	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Transplant - kidney	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Nervous system disorders Tremors	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Endocrine disorders TSH - elevated	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Renal and urinary disorders Urine - bloody s/p prostate biopsy	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Virus - post transplant	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Eye disorders Vision - hazy	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
Gastrointestinal disorders Vomiting	7.7% 1/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.
General disorders Weight loss - unexpected	15.4% 2/13 All SAEs were adjudicated to be disease related and not related to the agalsidase alfa infusions.

Additional Information

Dr. Raphael Schiffmann

Baylor Research Institute

Phone: 214-820-4533

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place