Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03206086 · ClinicalTrials.gov registry record · Phase 2
Eltrombopag for People With Fanconi Anemia
A Phase 2 study of Fanconi Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Active
- Registry status
- Phase 2
- Development phase
- 25
- Enrollment target
- 1
- Study location
NCT03206086: Active Phase 2 study of Fanconi Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT03206086 is a Phase 2 study of Fanconi Anemia that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 25 participants, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (93% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03206086, a Phase 2 study of Fanconi Anemia, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 25 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new drug will help people with this disease. Objective: To find out if a new drug, eltrombopag, is effective in people with Fanconi anemia. To know how long the drug needs to be given to improve blood counts. Eligibility: People at least 6 years old with Fanconi anemia with reduced blood cell counts. Design: Participants will be screened with blood and urine tests. They will repeat this before starting to take the study drug. Participants will take eltrombopag pills by mouth once a day for 24 weeks. They will be monitored closely for side effects. Participants will have blood tests every 2 weeks while on eltrombopag. Participants will visit NIH 3 months and 6 months after starting eltrombopag. At these visits, participants will: Answer questions about their medical history, how they are feeling, and their quality of life Have a physical exam Have blood and urine tests Have a bone marrow sample taken by needle from the hip. The area will be numbed. If participants blood cell counts improve, they might join the extended access part of the study. They will continue taking eltrombopag for 3 years and sign a different consent. After 24 weeks of treatment, if there is no improvement in blood cell counts, participants will stop taking eltrombopag. They will return for an optional follow-up visit that repeats the study visits....
Primary Outcome
Peripheral blood platelet count increases to 20,000/microliter above baseline at six months or stable platelet counts with transfusion independence, 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, or an ANC increase \>0.5 x 109/L; and \>= 2-fold increase in normal marr
Conditions Studied
Interventions
- DRUG Eltrombopag
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2018-11-02 |
| Est. Completion | 2028-08-01 |
| Phase | Phase 2 |
What the registry record for NCT03206086 still lists
NCT03206086 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (93% lower).
The record links to 1 condition, with Fanconi Anemia appearing as the primary indexed condition, and to 1 intervention - of which Eltrombopag is the first listed.
NCT03206086 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT03206086 about?
NCT03206086 is a clinical study titled "Eltrombopag for People With Fanconi Anemia". Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new ...
What is the current status of trial NCT03206086?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 25 participants. The study started on 2018-11-02. Estimated completion is 2028-08-01.
What conditions does trial NCT03206086 study?
This clinical trial studies the following conditions: Fanconi Anemia.
What interventions are being tested in trial NCT03206086?
The interventions under investigation include: Eltrombopag (DRUG).
Who is sponsoring clinical trial NCT03206086?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03206086 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Fanconi Anemia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03206086's enrollment target sits among peer trials
25 10th of 15 higher than 6 of 15 other Fanconi Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Fanconi Anemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
RECRUITING · Phase 2
-
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
RECRUITING · Phase 2
-
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
RECRUITING · Phase 2
-
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
ACTIVE NOT RECRUITING · Phase 2
-
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
COMPLETED · Phase 2
-
Hematopoietic Stem Cell Transplant for Fanconi Anemia
COMPLETED · Phase 2
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01166854 · 25 participants
Characterization of Familial Myopathy and Paget Disease of Bone
- NCT01913106 · 25 participants · Phase 1
HSV-tk + Valacyclovir Therapy in Combination With Brachytherapy for Recurrent Prostate Cancer
- NCT02138669 · 25 participants · NA
Intacs Prescription Inserts for Keratoconus Patients
- NCT02174549 · 25 participants · Phase 1
Dose-defining Study of Tirapazamine Combined With Embolization in Liver Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI