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Completed

NCT Number: NCT01124643

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

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

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Royal Melbourne Hospital, Parkville, Australia

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About this study

HGT-REP-060 was designed to provide participants who participated in TKT028 an additional 52 weeks of treatment with Replagal at the standard dose of 0.2 milligram per kilogram (mg/kg) every other week (EOW) to assess the effect of continued Replagal therapy on long-term safety and clinical outcomes for this participant population.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

Replagal

Biological

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

Other names: algalsidase alfa, alpha-Galactosidase, DRX005B

Primary outcomes

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

    Time frame: Baseline to 12 months

  2. Safety Evaluations

    Time frame: Baseline to 12 months

Secondary outcomes

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

    Time frame: Baseline to 12 months

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

    Time frame: Baseline to 12 months

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

    Time frame: Baseline to 12 months

    The MLHF-Q contains 21 questions with answers ranging from 0 (no) to 5 (very much). The final score ( 0 to 105) is the sum of the points for the 21 questions. A higher score indicates a worse quality of life.

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

    Time frame: Baseline to 12 months

    Class I: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea. Class II: Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea. Class III: Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV: Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased.

  5. Change From Baseline in Plasma Gb3

    Time frame: Baseline to 12 months

  6. Change From Baseline in eGFR

    Time frame: Baseline to 12 months

  7. Change From Baseline in Albumin/Creatinine (A/Cr) Ratio

    Time frame: Baseline to 12 months

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Official study title

An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease

Important dates

Study start
2010
Primary completion
2013
Study completion
2013
First posted
May 17, 2010
Registry last updated
Jun 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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