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HGT-REP-082
Completed

This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.

Clinicaltrials.gov
#NCT01304277

About this clinical trial

This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.

CA
Interventional Phase 2 clinical trial.

At a glance

What medical conditions were being studied?

Fabry Disease

What was the clinical trial testing?

agalsidase alfa

How many participants were enrolled?

17

When was the clinical trial conducted?

Nov 2011 - Dec 2012

How long was participation in the clinical trial?

This study is complete and no longer enrolling patients.

Key requirements

Sexes

Male

Age

18 to 65 Years

Healthy volunteers?

No

Inclusion Criteria:

1. The patient must be diagnosed with Fabry disease using the following criteria: The
patient is a hemizygous male with Fabry disease as confirmed by a deficiency of
α-galactosidase A activity measured in serum, leukocytes, or fibroblasts or has a
confirmed mutation of the α-galactosidase A gene.

2. Patient is male and between 18 and 65 years of age, inclusive.

3. Patient must be willing to remain in the clinic as required by the study and comply
with the procedures and evaluations of the study.

4. At the time of confirmation of study eligibility visit, patients must have received
at least 26 weeks of treatment with RB Replagal at a dose of 0.2 mg/kg administered
IV EOW.

5. Patient provides informed consent.

Patients who are naive to ERT:

1. Treatment naive patients must have a pretreatment plasma Gb3 level above the normal
range (if value is available).

Exclusion Criteria:

1. Patient is unable to be venipunctured and/or tolerate venous access.

2. Patient has tested positive for anti-agalsidase alfa antibodies either at screening
or confirmation of eligibility visit.

3. Patient had pre-ERT plasma Gb3 levels within the normal range (if value is
available).

4. Patient is participating in any other Shire HGT investigational study.

5. Patient is currently on dialysis, is expected to begin dialysis during the study,
has received a kidney transplant, or is on the renal transplant waiting list.

6. Patient is unable to comply with the protocol (eg, clinical relevant medical
condition making implementation of the protocol difficult, unstable social
situation, or otherwise unlikely to complete the study) or is, in the opinion of the
Investigator, otherwise unsuited for the study.

7. The patient is enrolled in another clinical study that involves clinical
investigations or use of any investigational product (drug or device), except for
the Canadian Fabry Disease Initiative, within 6 months prior to receiving the first
dose of AF Replagal in this study or at any time during the study.

8. The patient has previously received AF Replagal prior to study entry.

Study results

Locations