Trial · NCT04469465
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 19 of 19
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Hgb at Week 12 | Baseline, Week 12 | Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM. |
| Secondary | Percentage of Participants With Hgb Increase of ≥2 Grams/Deciliter (g/dL) (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12 | Week 12 | The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 12 and remaining transfusion free during the 12-Week TP1. Participants who withdrew from the study early during the 12-Week TP1 or had missing Hgb value at Week 12 were considered as not achieving the criterion. |
| Secondary | Percentage of Participants With Transfusion Avoidance Through Week 12 | Week 12 | Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 12. Participants who discontinued study treatment early before Week 12 were considered as not achieving transfusion avoidance. |
| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12 | Baseline, Week 12 | The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue and better health-related quality of life. LS mean and SE were produced using MMRM. |
| Secondary | Change From Baseline in Absolute Reticulocyte Count at Week 12 | Baseline, Week 12 | LS mean and SE were produced using MMRM. |
| Secondary | Change in the Number of Red Blood Cell (RBC) Units Transfused From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment | 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment | |
| Secondary | Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment | 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment | |
| Secondary | Percentage of Participants With Transfusion Avoidance Through Week 24 | 24 weeks | Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 24. Participants who discontinued study treatment early before Week 24 were considered as not achieving transfusion avoidance. |
| Secondary | Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment | 12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment | LS mean and SE were produced using analysis of covariance (ANCOVA). |
| Secondary | Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment | 12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks) | LS mean and SE were produced using ANCOVA. |
| Secondary | Change From Baseline FACIT Fatigue Scores at Week 24 | Baseline, Week 24 | The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue and better health-related quality of life. LS mean and SE were produced using MMRM. |
| Secondary | Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan | Week 12 to Week 24 | The criterion was defined as Hgb stabilization avoidance of a \>1 g/dL (\>10 g/L) decrease in Hgb level at Week 24 from Week 12. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb stabilization regardless of the actual value observed at Week 24. |
| Secondary | Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24 | Week 24 | The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 24 and remaining transfusion free during the 12-Week TP2. Participants who withdrew from the study early during the 12-Week TP2 or had missing Hgb value at Week 24 were considered as not achieving the criterion. |
| Secondary | Change From Baseline in Total and Direct Bilirubin at Week 12 | Baseline, Week 12 | Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM. |
| Secondary | Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12 | Baseline, Week 12 | The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM. |
| Secondary | Change From Baseline in Complement Component 3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12 | Baseline, Week 12 | Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM. |
| Secondary | Change From Baseline in Lactate Dehydrogenase at Week 12 | Baseline, Week 12 | Baseline was defined as the average of all available assessments prior to the first dose of study intervention. LS mean and SE were produced using MMRM. |
| Secondary | Percentage of Participants With Hgb Normalization at Week 12 | Week 12 | Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 12 were considered as not meeting Hgb normalization regardless of actual value observed at Week 12. |
| Secondary | Percentage of Participants With Hgb Normalization at Week 24 | Week 24 | Hgb normalization was defined as Hgb value above LLN reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb normalization regardless of actual value observed at Week 24. |
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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