NCT01363492

Safety Study of Replagal® Therapy in Children With Fabry Disease

Completed Phase 2 Results posted Last updated 9 June 2021

What this trial tests

Phase 2 trial testing Replagal (agalsidase alfa) in Fabry Disease in 15 participants. Completed in 17 April 2013.

Timeline

12 May 2011

Primary endpoint
17 April 2013

17 April 2013

Quick facts

Lead sponsor	Shire
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	15
Start date	12 May 2011
Primary completion	17 April 2013
Estimated completion	17 April 2013
Sites	5 locations across United States

Drugs / interventions tested

• Replagal (agalsidase alfa) — full drug profile →

Conditions studied

• Fabry Disease — all drugs for Fabry Disease →

Sponsor

Shire — full company profile →

Who can join

Adults 7 to 17, any sex, with Fabry Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Serious Adverse Event (SAE) Primary · Baseline to week 55

Group	Value	95% CI
Replagal (0.2 mg/kg)	0

Number of Treatment Emergent Adverse Event (TEAE) Primary · Baseline to week 55

Group	Value	95% CI
Replagal (0.2 mg/kg)	166

Development of IgG Anti-Agalsidase Alfa Antibody Primary · Baseline to Week 55

Reflects development of Anti-Agalsidase antibodies post baseline

Group	Value	95% CI
Replagal (0.2 mg/kg)	1

Change From Baseline in LVMI Secondary · Baseline to week 55

Group	Value	95% CI
Replagal (0.2 mg/kg)	0.16	± 6.059

Change From Baseline in MFS Secondary · Baseline to week 55

Group	Value	95% CI
Replagal (0.2 mg/kg)	-0.62	± 3.596

Change From Baseline in Plasma Gb3 Secondary · Baseline to week 55

Group	Value	95% CI
Replagal (0.2 mg/kg)	-5.71	± 8.799

Change From Baseline in Urine Gb3 Secondary · Baseline to week 55

Group	Value	95% CI
Replagal (0.2 mg/kg)	-1403.25	± 3636.711

Change From Baseline in Heart Rate Variability Parameter SDNN Primary · Baseline to week 55

Group	Value	95% CI
Replagal 0.2 mg/kg	10.46	± 24.223

Change From Baseline in Heart Rate Variability Parameter rMSSD Primary · Baseline to week 55

Group	Value	95% CI
Replagal 0.2 mg/kg	1.46	± 53.502

Change From Baseline in Heart Rate Variability Parameter pNN50 Primary · Baseline to week 55

Group	Value	95% CI
Replagal 0.2 mg/kg	-4.13	± 20.166

Adverse events — posted to ClinicalTrials.gov

Time frame: Baseline to week 55. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Replagal® (0.2 mg/kg)

Serious: 0/14 (0%)

Deaths: —

Other adverse events (71 terms — click to expand)

Reaction	System	Replagal® (0.2 mg/kg)
Pyrexia	General disorders	—
Diarrhoea	Gastrointestinal disorders	—
Nausea	Gastrointestinal disorders	—
Vomiting	Gastrointestinal disorders	—
Upper respiratory tract infection	Infections and infestations	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—
Headache	Nervous system disorders	—
Cough	Respiratory, thoracic and mediastinal disorders	—
Nasopharyngitis	Infections and infestations	—
Sinusitis	Infections and infestations	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—
Ear pain	Ear and labyrinth disorders	—
Abdominal pain upper	Gastrointestinal disorders	—
Chest discomfort	General disorders	—
Chills	General disorders	—
Pain	General disorders	—
Gastroenteritis	Infections and infestations	—
Gastroenteritis viral	Infections and infestations	—
Pharyngitis streptococcal	Infections and infestations	—
Rhinitis	Infections and infestations	—
Arthralgia	Musculoskeletal and connective tissue disorders	—
Dizziness	Nervous system disorders	—
Paraesthesia	Nervous system disorders	—
Proteinuria	Renal and urinary disorders	—
Pharyngeal erythema	Respiratory, thoracic and mediastinal disorders	—
Throat irritation	Respiratory, thoracic and mediastinal disorders	—
Acne	Skin and subcutaneous tissue disorders	—
Anaemia	Blood and lymphatic system disorders	—
Iron deficiency anaemia	Blood and lymphatic system disorders	—
Sinus bradycardia	Cardiac disorders	—
Hordeolum	Eye disorders	—
Retinal vascular disorder	Eye disorders	—
Abdominal pain	Gastrointestinal disorders	—
Dyspepsia	Gastrointestinal disorders	—
Mouth ulceration	Gastrointestinal disorders	—
Tooth malformation	Gastrointestinal disorders	—
Fatigue	General disorders	—
Feeling cold	General disorders	—
Malaise	General disorders	—
Non-cardiac chest pain	General disorders	—

Data from ClinicalTrials.gov NCT01363492 adverse events section.

Sponsor's own description

The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Patient and observer reported outcome measures to evaluate health-related quality of life in inherited metabolic diseases: a scoping review.
   Pascoal C, Brasil S, Francisco R, Marques-da-Silva D, et al · · 2018 · cited 24× · PMID 30486833 · DOI 10.1186/s13023-018-0953-9
2. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy.
   Goker-Alpan O, Longo N, McDonald M, Shankar SP, et al · · 2016 · cited 17× · PMID 27307708 · DOI 10.2147/dddt.s102761