Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

NCT01124643 | Completed Phase 3 Results

• 2 other identifiers: HGT-REP-0602009-015985-75
• Study Type: interventional
• Target: 35
• Locations: 7 countries
• Sites: 9

Brief Summary

This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).

Conditions

Fabry Disease

Keywords

Agalsidase alfa; Alpha-Galactosidase; Replagal; Enzyme Replacement Therapy

Outcome Measures

Primary Outcomes (2)

• Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)

  Baseline to 12 months

• Safety Evaluations

  Baseline to 12 months

Secondary Outcomes (7)

• Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise

  Baseline to 12 months

• Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)

  Baseline to 12 months

• Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)

  Baseline to 12 months

• Change From Baseline in New York Heart Association (NYHA) Functional Class

  Baseline to 12 months

• Change From Baseline in Plasma Gb3

  Baseline to 12 months

Study Arms (1)

Replagal 0.2 mg/kg EOW

EXPERIMENTAL

Intravenous, 0.2mg/kg EOW

Biological: Replagal

Interventions

Replagal BIOLOGICAL

0.2 mg/kg administered intravenously \[IV\] every other week

Also known as: algalsidase alfa, alpha-Galactosidase, DRX005B

Replagal 0.2 mg/kg EOW

Eligibility Criteria

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

You may qualify if:

• Complete all study requirements and assessments for Study TKT028 less than 30 days (+/- 7 days) prior to the first dose in this extension protocol
• Voluntarily signed an Institutional Review Board/Independent Ethics Committee- approved informed consent form after all relevant aspects of the study have been explained and discussed.
• Has received and tolerated at least 80% of the total planned Replagal infusions in Study TKT028
• Female participants of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have a negative pregnancy test at the time of study entry and as required throughout participation in study

You may not qualify if:

• Has received treatment with any investigational drug (other than Replagal) or device within 30 days prior to study entry.
• Is unable to comply with the protocol, (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator
• Has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C(HCV) antibody, or human immunodeficiency virus (HIV) antibody
• Is pregnant or lactating
• Is morbidly obese, defined as body mass index (BMI) \>39 kg/m2
• Has any safety or medical issues, as assessed by the Investigator, that contraindicate participation in the study (eg, has experienced an adverse reaction to treatment with Replagal or has a known hypersensitivity to any of the components of Replagal

Sponsors & Collaborators

• Shire: lead

Study Sites (9)

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

O&O Alpan LLC

Fairfax, Virginia, 22030, United States

Location

The Royal Melbourne Hospital

Parkville, 3050, Australia

Location

1st School of Medicine Charles University

Prague, Czechia

Location

Turku University Central Hospital

Turku, FI-20520, Finland

Location

Szpital Uniwersytecki w Krakowie

Krakow, 31-066, Poland

Location

Instytut Kardiologii, I Klinika Choroby Wiencowej

Warsaw, Poland

Location

General Hospital Slovenj Gradec

Slovenj Gradec, 2380, Slovenia

Location

Salford Royal NHS Foundation Trust

Salford, England, M6 8HD, United Kingdom

Location

Related Publications (1)

• Golan L, Goker-Alpan O, Holida M, Kantola I, Klopotowski M, Kuusisto J, Linhart A, Musial J, Nicholls K, Gonzalez-Rodriguez D, Sharma R, Vujkovac B, Chang P, Wijatyk A. Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease. Drug Des Devel Ther. 2015 Jul 8;9:3435-44. doi: 10.2147/DDDT.S80928. eCollection 2015.

  PMID: 26185417 RESULT

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase alfa; alpha-Galactosidase

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

Intervention Hierarchy (Ancestors)

Galactosidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

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 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 23, 2010
• First Posted: May 17, 2010
• Study Start: April 13, 2010
• Primary Completion: July 8, 2013
• Study Completion: July 8, 2013
• Last Updated: June 8, 2021
• Results First Posted: August 22, 2014

Record last verified: 2021-05

Locations

AU (1); CZ (1); PL (2); SL (1); GB (1); FI (1); US (2)