Phase 3
Completed N=35
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
Source: ClinicalTrials.gov NCT01124643 ↗Enrolled (actual)
35
Serious AEs
17.1%
Results posted
Aug 2014
Primary outcomePrimary: Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) — -0.75 g/m^2.7
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) |
-0.75 | — |
| PRIMARY Safety Evaluations |
3; 32; 2; 5; 6; 0 | — |
| SECONDARY Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise |
-0.7 | — |
| SECONDARY Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT) |
-11.2 | — |
| SECONDARY Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q) |
6.0 | — |
| SECONDARY Change From Baseline in New York Heart Association (NYHA) Functional Class |
0; 34; 1 | — |
| SECONDARY Change From Baseline in Plasma Gb3 |
-1.18 | — |
| SECONDARY Change From Baseline in eGFR |
-3.25 | — |
| SECONDARY Change From Baseline in Albumin/Creatinine (A/Cr) Ratio |
178.5 | — |
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT01124643). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.