Trial · NCT05843916
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 15 of 15
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Lyso Gb3 Serum Levels | Baseline (after 5 week lead-in period on Fabrazyme®) and 26 weeks (after the switch to AGA BETA BS) | The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker. Endpoint: Mean serum Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by serum level of the marker Lyso-Gb3 at baseline. |
| Secondary | Change From Baseline in Lyso-Gb3 Serum Levels | Baseline (after 5 week lead-in period on Fabrazyme®) and 54 weeks (after the switch to AGA BETA BS) | To evaluate the difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year (54 weeks) of treatment in participants with Fabry disease previously stabilized with Fabrazyme®, by measuring disease biomarker. Endpoint: Mean serum Lyso-Gb3 marker ratio after 54 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 54 weeks (12 months) divided by serum level of the marker Lyso-Gb3 at baseline. |
| Secondary | Change From Baseline in Pain Severity as Assessed by Brief Pain Inventory-short Form Pain Severity Items Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the Brief Pain Inventory-short form (BPI). The Brief Pain Inventory (BPI) is a validated patient-reported outcome instrument assessing pain severity and pain-related interference with daily activities. Pain severity is assessed using four items rated on a 0-10 numeric rating scale (0 = no pain; 10 = pain as bad as imaginable), with the score calculated as the mean of items (range 0-10; higher scores indicate worse pain). Pain interference is assessed using seven items rated on a 0-10 numeric rating scale (0 = does not interfere; 10 = completely interferes), with the score calculated as the mean of items (range 0-10; higher scores indicate worse outcome). Endpoint: Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 and 54 weeks of treatment. |
| Secondary | Change From Baseline in Pain Interference as Assessed by BPI-short Form Pain Interference Items Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. The Brief Pain Inventory (BPI) is a validated patient-reported outcome instrument assessing pain severity and pain-related interference with daily activities. Pain severity is assessed using four items rated on a 0-10 numeric rating scale (0 = no pain; 10 = pain as bad as imaginable), with the score calculated as the mean of items (range 0-10; higher scores indicate worse pain). Pain interference is assessed using seven items rated on a 0-10 numeric rating scale (0 = does not interfere; 10 = completely interferes), with the score calculated as the mean of items (range 0-10; higher scores indicate worse outcome). Endpoint: Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 and 54 weeks of treatment. |
| Secondary | Change From Baseline in SF-36 Health Survey Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the Short Form-36 Health Survey (SF-36). The Short Form-36 Health Survey (SF-36) is a validated patient-reported outcome instrument consisting of 36 questions designed to assess health-related quality of life. The questionnaire generates scores across eight domains (physical functioning, role limitations due to physical health, pain, general health, energy/fatigue, social functioning, role limitations due to emotional problems, and emotional well being), each scored on a 0-100 scale. Domain scores are calculated according to standard scoring algorithms, with higher scores indicating better health status and quality of life. Endpoint: Change from baseline in SF-36 scores after 26 and 54 weeks of treatment. |
| Secondary | Mean Blood Urea Nitrogen (BUN) Levels at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Estimated Glomerular Filtration Rate (eGFR) at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Urine Albumin-to-Creatinine Ratio at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Electrolyte and Phosphate Levels at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Heart Rate From Electrocardiogram Assessments at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | Evaluation of Heart Rate based on the analysis of electrocardiogram exams performed at baseline, 26 weeks and 54 weeks. |
| Secondary | Mean PR, QRS, QT, QTcB, and QTcF Intervals at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | Evaluation of PR, QRS, QT , QTcB and QTcF intervals based on the analysis of electrocardiogram exams performed at baseline, 26 weeks and 54 weeks. |
| Secondary | Mean Left Ventricular Wall Thickness at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks. |
| Secondary | Mean Left Ventricular Mass Index at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks. |
| Secondary | Mean Left Ventricular Ejection Fraction at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks. |
| Secondary | Number of Participants With Positive Neutralizing Antibodies at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®),14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS). | Count of participants whose serum samples showed a neutralization percentage of agalsidase beta activity greater than 50%, the predefined threshold indicating a positive result for neutralizing antibodies. Neutralizing antibody activity was evaluated using blood samples collected at Baseline, Week 14, Week 26, and Week 54. Participants with neutralization values exceeding the 50% cutoff were classified as positive for neutralizing antibodies at each timepoint. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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