Trial · NCT05144256
Registered outcomes27
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 27
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Achieving Transfusion Reduction Response (TRR) | Week 9 to Week 32 | TRR is defined as ≥33% reduction in total red blood cell (RBC) transfusion volume from Week 9 through Week 32 of the double-blind period, normalized by weight and actual study drug duration compared with the historical transfusion volume, standardized by weight, and to 24 weeks. |
| Secondary | Percentage of Participants With Transfusion-free Response | Week 9 to Week 32 | Transfusion-free response is defined as achievement of 0 transfusions administered from Week 9 through Week 32 of the double-blind period. |
| Secondary | Change in the Number of Transfusion Episodes | Week 9 to Week 32 | The change in the number of transfusion episodes from Week 9 through Week 32 of the double-blind period compared with the historical number of transfusion episodes standardized to 24 weeks. |
| Secondary | Percentage Change in Weight-normalized and Study Treatment Duration-normalized Total Transfusion Volume | Week 9 to Week 32 | The percentage change in weight-normalized and study treatment duration-normalized total transfusion volume from Week 9 through Week 32 of the double-blind period will be compared with the historical transfusion volume standardized by weight and to 24 weeks. |
| Secondary | Percentage of Participants With Normal Hemoglobin (Hb) Response | Week 9 to Week 32 | Normal Hb response is defined as achievement of Hb concentrations in the normal range at least once, 8 weeks or more after a transfusion during Week 9 through Week 32 of the double-blind period. |
| Secondary | Change From Baseline in Estradiol Concentration | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Estrone Concentration | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Total Testosterone Concentration | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Free Testosterone Concentration in Participants ≥7 Years of Age or Tanner Stage ≥2 (Whichever Occurs First) | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Sexual Maturity Rating with Tanner Stage | Baseline up to Week 298 | Tanner Stage 1 corresponds to the prepubertal form, with progression to Tanner Stage 5, the final adult form. |
| Secondary | Percentage Number of Female Participants With Development of Ovarian Cysts | Baseline up to Week 298 | |
| Secondary | Change From Baseline in the Size of Ovarian Cysts in Female Participants | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Height-for-age Z-score | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Weight-for-age Z-score | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Body Mass Index (BMI)-for-age Z-score | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Bone Mineral Density (BMD) Z-score | Baseline up to Week 298 | |
| Secondary | Change from Baseline in Serum Iron Concentration | Baseline up to Week 292 | |
| Secondary | Change from Baseline in Serum Ferritin Concentration | Baseline up to Week 292 | |
| Secondary | Change from Baseline in Total Iron-binding Capacity | Baseline up to Week 292 |
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.
For software
Read this list from software.
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GET https://conductscience.org/api/trials/v1/NCT05144256/registered