Trial · NCT07517133
Registered outcomes29
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 29
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Achieved a Hemoglobin (Hb) Response | Baseline, Week 12 through Week 24 | Hb response is defined as a ≥1.0 grams per deciliter (g/dL) increase in average Hb concentration from Week 12 through Week 24 compared with baseline. |
| Secondary | Change From Baseline in Average Hb Concentration From Week 12 Through Week 24 | Baseline, Week 12 through Week 24 | |
| Secondary | Percentage of Participants Who Achieved a Hb 1.5+ Response | Baseline, Week 12 through Week 24 | Hb 1.5+ response is defined as a ≥1.5 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with Baseline. |
| Secondary | Change From Baseline in Indirect Bilirubin at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Haptoglobin at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Reticulocytes at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Erythropoietin at Week 24 | Baseline, Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Estradiol Through Week 24 | Baseline, up to Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Estrone Through Week 24 | Baseline, up to Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Total Testosterone Through Week 24 | Baseline, through Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Free Testosterone Through Week 24 | Baseline, through Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Luteinizing Hormone Through Week 24 | Baseline, through Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Sexual Maturity Rating With Tanner Stage Through Week 24 | Baseline, through Week 24 | |
| Secondary | Cohort 1: Percentage of Female Participants With Newly Developed Ovarian Cysts Through Week 24 | Baseline, through Week 24 | |
| Secondary | Change From Baseline Over Time in Height-for-age Z-Score, Weight-for-age Z-Score, and Body Mass Index (BMI)-for-age Z-Score Through Week 24 | Baseline, through Week 24 | |
| Secondary | Change from Baseline in Bone Mineral Density (BMD) Through Week 24 | Baseline, through Week 24 | |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From first dose of the study drug to the end of DB period (through Week 24) | |
| Secondary | Change from Baseline Through Week 24 in Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale | Baseline, through Week 24 | The PedsQL Multidimensional Fatigue Scale measures dimensions of fatigue over a 7-day recall period. It includes 3 subscales: general fatigue, sleep/rest fatigue, and cognitive fatigue. Items are reverse-scored and linearly transformed to a 0-100 scale so that higher PedsQL Multidimensional Fatigue Scale scores indicate better health-related quality of life (HRQOL) (fewer symptoms of fatigue). |
| Secondary | Change from Baseline Through Week 24 in PedsQL 4.0 Generic Core Scale (GCS) | Baseline, through Week 24 | HRQOL will be assessed using the PedsQL Multidimensional 4.0 Generic Core Scales (for participants 2 years or older). The PedsQL 4.0 Generic Core Scales are multidimensional, well-validated measures of quality of life in childhood. They each consist of 4 subscales: physical health, emotional functioning, social functioning, and school functioning are reverse-scored and linearly transformed to a 0-100 scale, with higher scores indicating better HRQOL. |
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
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