Trial Outcomes & Findings for Safety Study of Replagal® Therapy in Children With Fabry Disease (NCT NCT01363492)
NCT ID: NCT01363492
Last Updated: 2021-06-09
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
Baseline to week 55
Results posted on
2021-06-09
Participant Flow
Participant milestones
| Measure |
Replagal® (0.2 mg/kg)
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Overall Study
STARTED
|
14
|
|
Overall Study
COMPLETED
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety Study of Replagal® Therapy in Children With Fabry Disease
Baseline characteristics by cohort
| Measure |
Replagal (0.2 mg/kg)
n=14 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
|
|---|---|
|
Age, Continuous
|
12.16 Years
STANDARD_DEVIATION 2.992 • n=5 Participants
|
|
Age, Customized
<=18 years
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
UNITED STATES
|
14 Participants
n=5 Participants
|
|
Heart rate variability parameter SDNN
|
103.46 msec
STANDARD_DEVIATION 32.928 • n=5 Participants
|
|
Heart rate variability parameter rMSSD
|
75.92 msec
STANDARD_DEVIATION 45.747 • n=5 Participants
|
|
Heart rate variability parameter pNN50
|
32.79 msec
STANDARD_DEVIATION 19.997 • n=5 Participants
|
|
Left Ventricular Mass Index (LVMI)
|
35.37 (g/m^2.7)
STANDARD_DEVIATION 10.129 • n=5 Participants
|
|
Midwall Fractional Shortening (MFS)
|
18.63 (%)
STANDARD_DEVIATION 2.891 • n=5 Participants
|
|
Plasma Gb3
|
14.79 (nmol/mL)
STANDARD_DEVIATION 12.228 • n=5 Participants
|
|
Urine Gb3
|
1775.08 (nmol/g creatinine)
STANDARD_DEVIATION 3691.087 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=14 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Number of Serious Adverse Event (SAE)
|
0 events
|
PRIMARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=14 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Number of Treatment Emergent Adverse Event (TEAE)
|
166 events
|
PRIMARY outcome
Timeframe: Baseline to Week 55Reflects development of Anti-Agalsidase antibodies post baseline
Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=14 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Development of IgG Anti-Agalsidase Alfa Antibody
|
1 participants
|
PRIMARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=13 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Change From Baseline in Heart Rate Variability Parameter SDNN
|
10.46 msec
Standard Deviation 24.223
|
PRIMARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=13 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Change From Baseline in Heart Rate Variability Parameter rMSSD
|
1.46 msec
Standard Deviation 53.502
|
PRIMARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=13 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Change From Baseline in Heart Rate Variability Parameter pNN50
|
-4.13 msec
Standard Deviation 20.166
|
SECONDARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=14 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Change From Baseline in LVMI
|
0.16 (g/m^2.7)
Standard Deviation 6.059
|
SECONDARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=14 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Change From Baseline in MFS
|
-0.62 (%)
Standard Deviation 3.596
|
SECONDARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=14 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Change From Baseline in Plasma Gb3
|
-5.71 (nmol/mL)
Standard Deviation 8.799
|
SECONDARY outcome
Timeframe: Baseline to week 55Outcome measures
| Measure |
Replagal (0.2 mg/kg)
n=12 Participants
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)
|
|---|---|
|
Change From Baseline in Urine Gb3
|
-1403.25 (nmol/g creatinine)
Standard Deviation 3636.711
|
Adverse Events
Replagal® (0.2 mg/kg)
Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Replagal® (0.2 mg/kg)
n=14 participants at risk
0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Cardiac disorders
Sinus bradycardia
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Ear and labyrinth disorders
Ear pain
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Eye disorders
Hordeolum
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Eye disorders
Retinal vascular disorder
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Gastrointestinal disorders
Abdominal pain
|
7.1%
1/14 • Number of events 2 • Baseline to week 55
|
|
Gastrointestinal disorders
Abdominal pain upper
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Gastrointestinal disorders
Diarrhoea
|
28.6%
4/14 • Number of events 5 • Baseline to week 55
|
|
Gastrointestinal disorders
Dyspepsia
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Gastrointestinal disorders
Mouth ulceration
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Gastrointestinal disorders
Nausea
|
28.6%
4/14 • Number of events 12 • Baseline to week 55
|
|
Gastrointestinal disorders
Tooth malformation
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Gastrointestinal disorders
Vomiting
|
28.6%
4/14 • Number of events 12 • Baseline to week 55
|
|
General disorders
Chest discomfort
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
General disorders
Chills
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
General disorders
Fatigue
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
General disorders
Feeling cold
|
7.1%
1/14 • Number of events 2 • Baseline to week 55
|
|
General disorders
Malaise
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
General disorders
Non-cardiac chest pain
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
General disorders
Pain
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
General disorders
Pyrexia
|
42.9%
6/14 • Number of events 8 • Baseline to week 55
|
|
Immune system disorders
Seasonal allergy
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Infections and infestations
Alveolar osteitis
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Infections and infestations
Gastroenteritis
|
14.3%
2/14 • Number of events 4 • Baseline to week 55
|
|
Infections and infestations
Gastroenteritis viral
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Infections and infestations
Infectious mononucleosis
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Infections and infestations
Nasopharyngitis
|
21.4%
3/14 • Number of events 3 • Baseline to week 55
|
|
Infections and infestations
Otitis media
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Infections and infestations
Pharyngitis streptococcal
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Infections and infestations
Rhinitis
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Infections and infestations
Sinusitis
|
21.4%
3/14 • Number of events 4 • Baseline to week 55
|
|
Infections and infestations
Upper respiratory tract infection
|
28.6%
4/14 • Number of events 5 • Baseline to week 55
|
|
Injury, poisoning and procedural complications
Avulsion fracture
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Injury, poisoning and procedural complications
Contusion
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Injury, poisoning and procedural complications
Scratch
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Investigations
Body temperature increased
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Investigations
Heart rate increased
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Investigations
Liver palpable subcostal
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Metabolism and nutrition disorders
Hyperlipidaemia
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
28.6%
4/14 • Number of events 7 • Baseline to week 55
|
|
Nervous system disorders
Dizziness
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Nervous system disorders
Headache
|
28.6%
4/14 • Number of events 7 • Baseline to week 55
|
|
Nervous system disorders
Paraesthesia
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Psychiatric disorders
Restlessness
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Renal and urinary disorders
Proteinuria
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
28.6%
4/14 • Number of events 8 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
21.4%
3/14 • Number of events 11 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal erythema
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
14.3%
2/14 • Number of events 3 • Baseline to week 55
|
|
Respiratory, thoracic and mediastinal disorders
Throat tightness
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Acne
|
14.3%
2/14 • Number of events 2 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Erythema
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Pityriasis rosea
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
|
Vascular disorders
Flushing
|
7.1%
1/14 • Number of events 4 • Baseline to week 55
|
|
Vascular disorders
Hypotension
|
7.1%
1/14 • Number of events 1 • Baseline to week 55
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication
- Publication restrictions are in place
Restriction type: OTHER