Phase 3 N=44 Randomized Treatment

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

Fabry Disease

Bottom Line

View on ClinicalTrials.gov: NCT00864851 ↗

Enrolled (actual)

Serious AEs

34.1%

Results posted

Jun 2014

Primary outcome: Primary: Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI) — 3.2; 0.5; -10.3 g/m^2.7

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Replagal (Biological)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Shire
Primary completion: Jun 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI)	3.2; 0.5; -10.3	—
SECONDARY Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise	-2.0; -0.3; 2.2	—
SECONDARY Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT)	-10.4; 37.9; 24.7	—
SECONDARY Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score	-3.1; 2.1; -8.6	—
SECONDARY Change From Baseline to Month 12 in New York Heart Association (NYHA) Functional Class	2; 2; 2; 15; 16; 3	—
SECONDARY Change From Baseline to Month 12 in Plasma Globotriaosylceramide (GB3)	-1.046; -2.132; -2.076	—
SECONDARY Change From Baseline to Month 12 in Estimated Glomerular Filtration Rate (eGFR)	-1.2; -3.3; -1.7	—
SECONDARY Change From Baseline to Month 12 in Urinary Albumin/Creatinine (A/Cr) Ratio	83.9; -54.1; -54.2	—
SECONDARY Safety Evaluation	1; 2; 0; 3; 19; 17	—

Summary

The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.

Eligibility Criteria

Inclusion Criteria

>18 years-old;
Male: Fabry disease confirmed by deficiency of alfa galactosidase A activity OR Female:Fabry disease confirmed by a mutation of the alfa galactosidase A gene;
ERT-naïve;
LVM/h > 50g/m2.7 for males and >47 g/m2.7 for females;
Negative pregnancy test at enrollment and contraception use required throughout study for female patients;
Signed informed consent;

Exclusion Criteria

Class IV heart failure;
Clinically significant hypertension;
Hemodynamically significant valvular stenosis or regurgitation;
Morbid obesity;
Known autosomal dominant sarcoplasmic contractile protein gene mutation;
Treatment with any investigational drug or device within the 30 days;
Unable to comply with the protocol as determined by the Investigator;
Positive for hepatitis B, hepatitis C or HIV

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00864851). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.