Trial · NCT03206086
Eltrombopag for Fanconi Anemia
NCT03206086, Phase 2, Active not recruiting, National Heart, Lung, and Blood Institute (NHLBI)
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Eltrombopag for Fanconi Anemia. Trials, record NCT03206086. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03206086
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Eltrombopag for Fanconi Anemia
- NCT number
- NCT03206086
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 25 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Fanconi Anemia
- Interventions
- Eltrombopag
- First posted
- 2017-07-02
- Last updated
- 2026-09-16
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Drug Responders | 6 months (+/- 14 day window) | Defined as one or more of the following: Peripheral Blood Response: * Platelet count increases of at least 20,000/uL above baseline or stable platelet counts with transfusion independence for those participants that were transfusion dependent prior to treatment for a minimum of 8 consecutive weeks prior to response assessment; * An increase in hemoglobin by \> 1.5g/dL or a reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks; * At least a 100% increase in ANC in participants with a pretreatment absolute neutrophil count (ANC) of \<0.5 x 10\^9/L, or an ANC increase \>0.5 x 10\^9/L Bone Marrow Response: * \>= 2-fold increase in normal marrow CD34+ cells by CD34 immunohistochemistry or flow cytometry, and/or * \>= 2-fold increase in normal marrow cellularity as measured by standard stains (H\&E) of bone marrow biopsy/aspirate sections. |
| Primary | Toxicity Profile: Number of Participants With Eltrombopag-Related Adverse Events During the First 6 Months, Overall and by Event Term and Maximum CTCAE Grade | Up to 6 months (+/- 14 day window) | The table reports the number of participants with at least one related adverse event overall and, for each event term, the number by maximum grade (Grade 1-2 or Grade 3 or higher). Each participant was counted once in the overall row and once per event term at the highest grade experienced; event-term categories are not mutually exclusive. Adverse events were assessed by the investigator as possibly, probably, or definitely related to eltrombopag, and were graded using Common Terminology Criteria for Adverse Events (CTCAE) version 4.0: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe or medically significant), Grade 4 (life-threatening), and Grade 5 (death related to adverse event). |
| Secondary | Number of Participants With a Protocol-Defined Hematologic Response at Month 3 | Month 3 (+/- 14 day window) | Participants with an evaluable Month 3 assessment were responders if they met at least one criterion: 1. platelet response: platelet count increased by ≥20,000/µL from baseline, or, if platelet-transfusion dependent before treatment, stable counts with transfusion independence for ≥8 consecutive weeks before assessment; 2. erythroid response: hemoglobin increased by \>1.5 g/dL, or packed red blood cell transfusion units decreased by ≥50% during the 8 weeks before assessment versus the 8 weeks before treatment; 3. neutrophil response: for pretreatment absolute neutrophil count \<0.5 × 10\^9/L, absolute neutrophil count increased by ≥100% or by \>0.5 × 10\^9/L; or 4. bone marrow response: ≥2-fold increase from baseline in normal marrow CD34-positive cells by immunohistochemistry or flow cytometry and/or ≥2-fold increase in marrow cellularity by hematoxylin and eosin staining. |
| Secondary | Number of Participants Who Relapsed During the Extension Phase | From entry into the extension phase at Month 6 through up to 3 years of extension-phase follow-up, corresponding to up to Month 42 after initiation of eltrombopag | Among participants who met the protocol-defined response criteria at Month 6 and entered the extension phase, the number who subsequently lost a previously achieved hematologic response. Relapse was assessed using serial peripheral blood counts, transfusion requirements, and bone marrow evaluations obtained during protocol follow-up. Each participant was counted once at the first documented relapse. |
| Secondary | Number of Participants With Clonal Evolution During Study Follow-up | From baseline through the end of study follow-up, up to 42 months after initiation of eltrombopag | Number of participants who developed at least one new cytogenetic abnormality and/or new morphologic evidence of progression to myelodysplastic syndrome or acute myeloid leukemia after baseline. Clonal evolution was assessed using serial bone marrow morphology and chromosomal analysis by standard cytogenetic techniques. A myelodysplastic syndrome fluorescence in situ hybridization panel could be performed when clinically indicated and at the principal investigator's discretion. Each participant was counted once in the overall measure. |
| Secondary | Number of Participants With Adverse Events During the Extension Phase | From entry into the extension phase at Month 6 through up to 3 years of extension-phase treatment and follow-up, corresponding to up to Month 42 after initiation of eltrombopag | Among participants who entered the extension phase, the number who experienced one or more hematologic or nonhematologic adverse events during extended eltrombopag therapy and follow-up. Adverse events were graded using Common Terminology Criteria for Adverse Events version 4.0 and summarized by event term, maximum grade, seriousness, investigator-assessed relationship to eltrombopag, and action taken with study treatment. Each participant was counted once per adverse-event term at the maximum reported grade. |
| Secondary | Change From Baseline in Health-Related Quality-of-Life Scores at Months 3 and 6 | Baseline, Month 3 (+/- 14 day window), and Month 6 (+/- 14 day window) | Within-participant change from baseline in health-related quality-of-life scores at Months 3 and 6. Age-appropriate adult, pediatric, or parent-proxy questionnaires were used, as applicable. The protocol-specified measures included: * Patient-Reported Outcomes Measurement Information System Global Health. * Patient-Reported Outcomes Measurement Information System Sleep Disturbance. * Patient-Reported Outcomes Measurement Information System Applied Cognition-Abilities. * Patient-Reported Outcomes Measurement Information System Anxiety. * Patient-Reported Outcomes Measurement Information System Depression. * Functional Assessment of Cancer Therapy measures addressing anemia, thrombocytopenia, and neutropenia. Changes from baseline were summarized separately for each instrument or domain and evaluated overall and in relation to hematologic treatment response. |
| Secondary | Number of Participants With Fanconi Anemia-Associated Organ-System Findings at Baseline, Month 3, and Month 6 | Baseline, Month 3 (+/- 14 day window), and Month 6 (+/- 14 day window) | Number of participants with clinically identified findings involving organ systems commonly affected in Fanconi anemia at baseline, Month 3, and Month 6. Prespecified categories included skin lesions, endocrine dysfunction, and new head and neck, oropharyngeal, gastrointestinal, anogenital, or skin cancers. Findings at Months 3 and 6 were compared with baseline to identify newly diagnosed, worsened, improved, or unchanged abnormalities. Participants were counted once within each applicable category at each assessment. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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 trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03206086
GET https://conductscience.org/api/trials/v1/NCT03206086/registered