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

NCT01304277 | Completed Phase 2 Results

• 1 other identifier: HGT-REP-082
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 5

Brief Summary

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

Conditions

Fabry Disease

Keywords

Replagal

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to Week 16 (EOS) in Urine Gb3 Levels

  Baseline to EOS

Secondary Outcomes (6)

• Change From Baseline to Week 16 (EOS) in Plasma Gb3 Levels

  Baseline to EOS

• Dose-normalized Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Sample (AUClast/Dose)

  Week 0 to Week 14

• Dose-normalized AUC Extrapolated to Infinity (AUC∞/Dose)

  Week 0 to Week 14

• Dose-normalized Maximum Serum Concentration (Cmax/Dose)

  Week 0 to Week 14

• To Assess Safety and Tolerability by Anti-agalsidase Alfa Antibody Status (in Serum) at End of Study

  EOS

Study Arms (1)

Replagal® (0.2 mg/kg, IV, EOW)

EXPERIMENTAL

Screening period of approximately 14 days during which all patients received 1 infusion of 0.2 mg/kg Replagal RB (Week 0) Treatment period of 14 weeks during which all patients received 7 infusions of 0.2 mg/kg Replagal AF

Biological: agalsidase alfa

Interventions

agalsidase alfa BIOLOGICAL

Also known as: Replagal

Replagal® (0.2 mg/kg, IV, EOW)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Patient is male and between 18 and 65 years of age, inclusive.
• Patient must be willing to remain in the clinic as required by the study and comply with the procedures and evaluations of the study.
• 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.
• Patient provides informed consent.
• Patients who are naive to ERT:
• \. Treatment naive patients must have a pretreatment plasma Gb3 level above the normal range (if value is available).

You may not qualify if:

• Patient is unable to be venipunctured and/or tolerate venous access.
• Patient has tested positive for anti-agalsidase alfa antibodies either at screening or confirmation of eligibility visit.
• Patient had pre-ERT plasma Gb3 levels within the normal range (if value is available).
• Patient is participating in any other Shire HGT investigational study.
• 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.
• 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.
• 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.
• The patient has previously received AF Replagal prior to study entry.

Sponsors & Collaborators

• Shire: lead

Study Sites (5)

University of Alberta Hospital

Edmonton, Alberta, T6G 2H7, Canada

Location

Queen Elizabeth II Health Sciences Centre

Halifax, Nova Scotia, B3H 1V8, Canada

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

INC Research

Toronto, Ontario, M5V 2T3, Canada

Location

Hopital du Sacre-Coeur de Montreal

Montreal, Quebec, H4J 1C5, Canada

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase alfa

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 15, 2011
• First Posted: February 25, 2011
• Study Start: November 17, 2011
• Primary Completion: December 28, 2012
• Study Completion: December 28, 2012
• Last Updated: July 19, 2021
• Results First Posted: April 25, 2014

Record last verified: 2021-07

Locations

CA (5)