Trial · NCT07338344
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 20
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 | Percentage of participants with a ≥50% reduction in RBC transfusion burden compared to baseline (RBC transfusion burden during the 12 weeks before randomization) and a reduction of at least 2 units for 12 consecutive weeks between Weeks 13 and 24 after randomization |
| Secondary | Percentage of Participants Who Achieved ≥ 50% And a Reduction of ≥ 2 RBC units 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 ≥50% reduction in RBC transfusion burden relative to baseline, with a reduction of ≥ 2 RBC units, for 12 consecutive weeks between 37 and 48 weeks after randomization. |
| Secondary | Percentage Of Participants Who Achieved ≥ 33% And a Reduction of ≥ 2 RBC units 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 ≥33% reduction in RBC transfusion burden relative to baseline, with a reduction of ≥ 2 RBC units, for 12 consecutive weeks between 37 and 48 weeks after randomization. |
| Secondary | Percentage Of Participants Who Achieved ≥ 50% Reduction From Baseline in Transfusion Burden - weeks 1-12, 13-24, 25-36, 37-48 | Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48 | Percentage Of participants who achieved a ≥50% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) for any consecutive 12 weeks (weeks 1-12, 13-24, 25-36, 37-48). |
| Secondary | Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - weeks 1-12, 13-24, 25-36, 37-48 | Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48 | Percentage Of participants who achieved a ≥33% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) for any consecutive 12 weeks (weeks 1-12, 13-24, 25-36, 37-48). |
| Secondary | Percentage Of Participants Who Achieved ≥ 50% Reduction From Baseline in Transfusion Burden - weeks 1-24, 25-48 | Baseline: Day -83 to Day 1; Treatment: Weeks1-24, 25-48 | Percentage Of participants with a ≥50% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) during any 24 consecutive weeks (weeks 1-24, 25-48); |
| Secondary | Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - weeks 1-24, 25-48 | Baseline: Day -83 to Day 1; Treatment: Weeks1-24, 25-48 | Percentage Of participants with a ≥33% reduction in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) during any 24 consecutive weeks (weeks 1-24, 25-48); |
| Secondary | Maximum percentage change in transfusion burden from baseline over 12 consecutive weeks | Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48 | Percentage Of participants change in RBC transfusion burden relative to baseline (RBC transfusion burden during the 12 weeks prior to randomization) during any 12 consecutive weeks (weeks 1-12, 13-24, 25-36, 37-48); |
| Secondary | Percentage of Participants Who Maintained Red Blood Cell (RBC) Transfusion Independence For ≥8 Weeks | weeks 1-8, 9-16, 17-24, 25-32, 33-40, 41-48 | Percentage of Participants who became transfusion-independent during any 8 consecutive weeks (weeks 1-8, 9-16, 17-24, 25-32, 33-40, 41-48); |
| Secondary | Percentage of Participants Who Maintained Red Blood Cell (RBC) Transfusion Independence For ≥12 Weeks | weeks 1-12, 13-24, 25-36, and 37-48 | Percentage of Participants who became transfusion-independent during any 12 consecutive weeks (weeks 1-12, 13-24, 25-36, and 37-48); |
| Secondary | Maximum percentage change from baseline in the number of transfusion events over 12 consecutive weeks | Baseline: Day -83 to Day 1; Treatment: Weeks1-12, 13-24, 25-36, 37-48 | Maximum percentage change from baseline in the number of transfusion events (RBC transfusion burden during the 12 weeks prior to randomization) during any 12 consecutive weeks (weeks 1-12, 13-24, 25-36, and 37-48); |
| Secondary | Concentration Change From Baseline to End of Treatment in serum ferritin | Baseline (prior to first dose of study drug) and Treatment (weeks 12,24,36,48) | Concentration change from baseline in serum ferritin at weeks 12, 24, 36, and 48 after randomization; |
| Secondary | Change From Baseline to End of Treatment in Iron deposition in organs | Baseline (prior to first dose of study drug) and Treatment (weeks 24,48) | Change from baseline in myocardial iron and liver iron concentrations at weeks 24 and 48 after randomization; |
| Secondary | Change From Baseline to End of Treatment in the average daily dose of iron chelation therapy | Baseline (prior to first dose of study drug) and Treatment (weeks 1-48) | Change from baseline in the average daily dose of iron chelation therapy (ICT) during the treatment period (up to 48 weeks); |
| Secondary | Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL | Baseline (prior to first dose of study drug) and Treatment (weeks 24,48) | 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 | Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by SF-36 | Baseline (prior to first dose of study drug) and Treatment (weeks 24,48) | 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. |
| Other | Percent Change From Baseline to End of Treatment in Fetal hemoglobin | Baseline (prior to first dose of study drug) and Treatment (weeks 12,24,36,48) | Percent changes from baseline in fetal hemoglobin (HbF) at weeks 12, 24, 36, and 48 after randomization |
| Other | Change From Baseline to End of Treatment in γ-Globin Gene | Baseline (prior to first dose of study drug) and Treatment (weeks 12,24,36,48) | Change from baseline to end of treatment in γ-globin geneγ-globin gene expression at weeks 12, 24, 36, and 48 after randomizatio |
| Other | Concentration Change From Baseline to End of Treatment in Hemolysis Indices | Baseline (prior to first dose of study drug) and Treatment (weeks 12,24,36,48) | Changes in concentration values from baseline to end of treatment in hemolysis indices the changes in hemolysis indicators (total bilirubin (TIBL), indirect bilirubin (IBIL), lactate dehydrogenase (LDH), and reticulocytes) relative to baseline at weeks 12, 24, 36, and 48 after randomization |
| Other | Safety - Incidence of Adverse Events (AEs) | Weeks1--48 | An AE is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A serious AE is any AE occurring at any dose that - Results in death - Is life-threatening - Requires or prolongs existing inpatient hospitalization - Results in persistent or significant disability/incapacity - Is a congenital anomaly/birth defect - Constitutes an important medical event. The Investigator assessed the relationship of each AE to study drug and graded the severity according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 4.03): - Grade 1 = Mild - Grade 2 = Moderate (some limitation in activity; no/minimal medical intervention) - Grade 3 = Severe (limitation in activity; medical intervention required) - Grade 4 = Life-threatening - Grade 5 = Death. |
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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