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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01124643
• Date of registration: 23/04/2010
• Prospective Registration: No
• Primary sponsor: Shire
• Public title: Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
• Scientific 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
• Date of first enrolment: April 13, 2010
• Target sample size: 35
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01124643
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Czech Republic, Czechia, Finland, Paraguay, Poland, Slovenia, United Kingdom, United States

Contacts

• Name: Study Director
• Affiliation: Shire

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Biological: Replagal

Primary Outcome(s)

Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) [Time Frame: Baseline to 12 months]

Safety Evaluations [Time Frame: Baseline to 12 months]

Secondary Outcome(s)

Change From Baseline in eGFR [Time Frame: Baseline to 12 months]

Change From Baseline in New York Heart Association (NYHA) Functional Class [Time Frame: Baseline to 12 months]

Change From Baseline in Plasma Gb3 [Time Frame: Baseline to 12 months]

Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q) [Time Frame: Baseline to 12 months]

Change From Baseline in Albumin/Creatinine (A/Cr) Ratio [Time Frame: Baseline to 12 months]

Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT) [Time Frame: Baseline to 12 months]

Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise [Time Frame: Baseline to 12 months]

Secondary ID(s)

HGT-REP-060

2009-015985-75

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 22/08/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01124643