Trial Outcomes & Findings for A Study of Replagal in Treatment-naïve Adults With Fabry Disease (NCT NCT04840667)
NCT ID: NCT04840667
Last Updated: 2024-06-17
Results Overview
Renal function was planned to be assessed by estimated glomerular filtration rate (eGFR) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula. The eGFR was planned to be calculated by CKD-EPI formula: eGFR = 141 x min (Serum Creatinine \[Scr\]/κ,1)\^(α) x max(Scr/κ,1)\^(-1.209) x 0.993\^(Age) x 1.018 (if female) x 1.159 (if black) where: Scr was serum creatinine (mg/dL); κ was 0.7 for females and 0.9 for males; α was -0.329 for females and -0.411 for males; min indicated the minimum of Scr/κ or 1; max indicated the maximum of Scr /κ or 1. Change from baseline in renal function at Week 104 was planned to be reported.
TERMINATED
PHASE3
17 participants
Baseline, Week 104
2024-06-17
Participant Flow
A total of 17 participants were screened and signed informed consent but none of the participants received any treatment due to screen failures. The study was terminated by the sponsor due to enrolment challenges and no participants were treated, therefore no data were evaluated and collected to be reported in this study.
Participant milestones
| Measure |
REPLAGAL
Participants were to receive REPLAGAL 0.2 milligram per kilogram (mg/kg) body weight of intravenous (IV) infusion every other week (EOW) for 104 weeks.
|
|---|---|
|
Overall Study
STARTED
|
17
|
|
Overall Study
Treated
|
0
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
17
|
Reasons for withdrawal
| Measure |
REPLAGAL
Participants were to receive REPLAGAL 0.2 milligram per kilogram (mg/kg) body weight of intravenous (IV) infusion every other week (EOW) for 104 weeks.
|
|---|---|
|
Overall Study
Screen failures/ Failure to meet inclusion criteria
|
17
|
Baseline Characteristics
A Study of Replagal in Treatment-naïve Adults With Fabry Disease
Baseline characteristics by cohort
Baseline data not reported
PRIMARY outcome
Timeframe: Baseline, Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Renal function was planned to be assessed by estimated glomerular filtration rate (eGFR) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula. The eGFR was planned to be calculated by CKD-EPI formula: eGFR = 141 x min (Serum Creatinine \[Scr\]/κ,1)\^(α) x max(Scr/κ,1)\^(-1.209) x 0.993\^(Age) x 1.018 (if female) x 1.159 (if black) where: Scr was serum creatinine (mg/dL); κ was 0.7 for females and 0.9 for males; α was -0.329 for females and -0.411 for males; min indicated the minimum of Scr/κ or 1; max indicated the maximum of Scr /κ or 1. Change from baseline in renal function at Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Cardiac structure was planned to be assessed by left ventricular mass index (LVMI) using cardiac magnetic resonance imaging (cMRI). Change from baseline in cardiac structure at Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Annualized rate of change in eGFR up to Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Annualized rate of change in LVMI up to Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Change from baseline in eGFR up to Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Change from baseline in LVMI up to Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Proteinuria was to be measured based on protein/creatinine ratio (PCR). Change from baseline in proteinuria up to Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Change from baseline in cardiac fibrotic segments suggestive of cardiac fibrosis up to Week 104 was planned to be assessed by volume of fibrosis, measured by cMRI.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Change from baseline in interventricular septal end-diastolic thickness and posterior wall thickness in diastole up to Week 104 was planned to be measured by cMRI.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Change from baseline in lyso-Gb3 up to Week 104 was planned to be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From start of study drug administration up to follow-up visit (i.e., up to Week 106)Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 104Population: The study was terminated by the Sponsor due to enrolment challenges. No participants were treated in this study, therefore no data were evaluated and collected for this outcome measure.
Number of participants who will develop ADA to REPLAGAL was planned to be reported.
Outcome measures
Outcome data not reported
Adverse Events
REPLAGAL
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER