Trial · NCT02604433
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 28
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | Erythroid Response was defined as red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Week 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48 | Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Weeks 37 - 48 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Weeks 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Week 37 to Week 48 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | Baseline was defined as the total number of Red Blood Cells (RBC) units transfused during the 12-week interval on or prior to Dose 1 Day 1. This is compared to the total number of RBC units transfused during the 12-week interval from treatment weeks 13-24. |
| Secondary | Mean Change From Baseline In Liver Iron Concentration (LIC) At Week 48 | Baseline: Week -12 to Day -1; Treatment: Week 48 | Baseline was defined as the last value on or before the first dose of study drug was administered; if multiple values were present for the same date, the average of these values was used. If a participant had 1 postbaseline assessment, it was used as the Week 48 value. If a participant had multiple postbaseline assessments, the last one was used as the Week 48 value. The value of LIC was collected by magnetic resonance imaging. Participants with a LIC value \> 43 mg/g were not included in the analysis. |
| Secondary | Mean Change From Baseline In Mean Daily Dose Of Iron Chelation Therapies (ICT) At Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | Three different types of Iron Chelation Therapy (ICT) were analyzed: 1. Deferasirox 2. Deferiprone 3. Deferoxamine Mesilate/Deferoxamine The baseline mean daily dose was calculated using the ICT dosage during the 12 weeks prior to first study drug administration and the postbaseline mean daily dose was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or the last 12 weeks of the study treatment for early discontinued participants. |
| Secondary | Mean Change From Baseline In Mean Serum Ferritin At Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | For each participant, the baseline mean serum ferritin level was calculated during the 12 weeks prior to first study drug administration. The postbaseline mean serum ferritin level was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or last 12 weeks of study treatment, if discontinued early. The change was calculated as the difference of post baseline mean serum ferritin level and baseline mean serum ferritin level. |
| Secondary | Mean Change From Baseline In Total Hip And Lumbar Spine Bone Mineral Density (BMD) At Week 48 | Baseline: Day 1; Treatment: Week 48 | For BMD, the lumbar spine and total hip were measured at baseline and 48 weeks by dual energy x-ray absorptiometry (DXA). Baseline was defined as the last value on or before the first dose of study drug is administered; if multiple values are present for the same date, the average of these values was used. If during the 48 week double-blinded treatment period, a participant has only one assessment, it is counted as 'Week 48' visit; if a participant has multiple assessments, the last one is used as 'Week 48' visit. The analysis was done on the population that had at least 2 measurements. |
| Secondary | Mean Change From Baseline In Myocardial Iron At Week 48 | Baseline: Day 1; Treatment: Week 48 | Myocardial Iron levels were measured by Magnetic Resonance Imaging (MRI), using MRI parameter T2\* (Unit: ms). T2\* values correlates with heart failure (HF) risk (e.g. T2\*\<6ms: high HF risk). |
| Secondary | Mean Change From Baseline in the Transfusion-dependent Quality of Life (TranQol) Questionnaire At Week 24 | Baseline: 4 weeks prior to Day 1; Treatment: Week 24 | The TranQol is a self-administered quality of life tool developed for beta-thalassemia patients. The adult self-report version used in this study, includes 36 questions assessed on a 5-point response, that are grouped into 5 domains (Physical Health, Emotional Health, Sexual Health, Family Functioning, School/Career Functioning). Scores are calculated according to specific scoring algorithms developed by the authors. Both individual domains score and the total score range from 0 (worst) to 100 (best). Total Score and Physical Health domain score are reported. Positive change from baseline values indicate improvement. |
| Secondary | Mean Change From Baseline in the 36-item Short Form Health Survey (SF-36) Questionnaire At Weeks 24 | Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24 | The SF-36 (version 2) is a generic, self-administered instrument consisting of 8 multi-item scales that assess 8 health domains. The raw score for each health domain is transformed into a 0 (worst) to 100 (best) domain score. The 0-100 scale score for each health domain is further converted to normbased scores using a T-score transformation, with a mean of 50 and a standard deviation (SD) of 10. Higher norm-based T-scores indicate better heath/QoL. The domains/summaries reported are: 1. Physical Functioning (Range of possible T-scores is 19.26 - 57.54) 2. General Health (Range of possible T-scores is 18.95 - 66.50) 3. Physical Component summary (PCS) (Range of possible T-scores is 5.02 - 79.78). Positive change from baseline values indicate improvement. |
| Secondary | Number of Participants Who Utilized Healthcare Resources During Study | From informed consent signing (up to 12 weeks before start of treatment) to end of treatment (up to approximately 227 weeks) | Number of participants who had any of the following types of Healthcare Resource Utilization (HRU): - a doctor office visit (non-study scheduled) - an emergency department visit - a hospitalization |
| Secondary | Number of Days Spent in Higher Care Hospital Units | From informed consent signing (up to 12 weeks before start of treatment) to end of treatment (up to approximately 227 weeks) | Types of hospitals units considered to be 'higher care' are: - Intensive Care Unit - Coronary Care Unit |
| Secondary | Percentage Of Participants Who Were Transfusion Independent For ≥ 8 Weeks During Treatment | From first dose through 3 weeks post last dose (up to approximately 218 weeks) | Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, i.e, Days 1 to 56, Days 2 to 57 and so on. |
| Secondary | Duration of Reduction in Transfusion Burden | From first dose to end of study treatment (up to approximately 215 weeks) | Responders were defined as subjects who achieved ≥ 33% reduction or ≥ 50% reduction in Red Blood Cells Transfusion (RBC-T) burden from baseline with a reduction of at least 2 RBC units during any rolling 12-week interval. The duration of reduction is calculated as Last Day of Response - First day of response +1 |
| Secondary | Longest Duration of Transfusion Independence | From first dose through 3 weeks post last dose (up to approximately 218 weeks) | Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, ie, Days 1 to 56, Days 2 to 57 and so on. Longest duration of transfusion independence was estimated based on Kaplan-Meier model. |
| Secondary | Time to Erythroid Response | From first dose to 48 weeks following first dose | Time to erythroid response was defined as the time from first dose of the study drug to first erythroid response. This is reported for participants with a ≥ 33% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval., as well as participants with a ≥ 50% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval. |
| Secondary | Post-Baseline Transfusion Event Frequency | From first dose through 3 weeks post last dose (up to approximately 218 weeks) | The number of transfusion events after start of study treatment were evaluated. For the definition of transfusion events, if multiple transfusions happen on the same date, they are counted as one event; if multiple transfusions happen on two consecutive dates, they are counted as one event; if multiple transfusions happen on three consecutive dates, they are counted as two events. Results are presented in 24-week intervals, up to 96 weeks after start of study treatment |
| Secondary | Pharmacokinetic (PK) Parameters: Bayesian Estimate of Apparent Clearance (CL/F) | Blood serum samples taken pre-dose and on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337 |
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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