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Thrombopoietin Receptor Agonist

Eltrombopag for Fanconi Anemia

Phase 2
Recruiting
Led By Andre Larochelle, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 2 years old
Confirmed diagnosis of Fanconi anemia confirmed by a biallelic mutation in a known FANC gene and/or by positive chromosome breakage analysis in lymphocytes and/or skin fibroblasts (for mosaicism)
Must not have
Inability to take oral medication
History or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the study
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug, eltrombopag, to see if it is effective in people with Fanconi anemia. People with the disease often have reduced blood cell counts and may need transfusions. The trial will monitor participants closely for side effects.

Who is the study for?
This trial is for people aged 2 or older with Fanconi anemia who have low blood cell counts and weigh over 10kg. They should not have had recent cancer, thromboembolic events, HIV, certain genetic mutations, severe illnesses that could affect the drug's tolerance, or be on other investigational drugs.
What is being tested?
The study tests eltrombopag effectiveness in improving blood counts for those with Fanconi anemia. Participants take eltrombopag daily for 24 weeks and are monitored through regular visits and blood tests. If successful, they may continue treatment up to three years.
What are the potential side effects?
While not explicitly listed in the provided information, common side effects of eltrombopag can include headaches, nausea, diarrhea, fatigue, coughing and potential liver issues which will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 2 years old.
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I have been diagnosed with Fanconi anemia through genetic testing or chromosome analysis.
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I have low blood counts or need regular blood transfusions.
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I have either not responded to or chosen not to take androgen therapy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I cannot take medicine by mouth.
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I have a heart condition that could make participating in the study unsafe.
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I am not pregnant or nursing.
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I am a sexually active male and agree to use a condom during the study and for 30 days after.
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I have a genetic mutation in both copies of my BRCA2 gene.
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I am cancer-free with no expected recurrence within a year.
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I have had blood clots in the past.
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My test shows genetic changes linked to a worse outlook in my condition.
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I am a woman able to have children and am not using strong birth control methods.
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My condition meets the WHO criteria for MDS or AML.
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I have been diagnosed with HIV.
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My direct bilirubin level is higher than 3.0mg/dL.
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I have an infection that isn't getting better with treatment.
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My liver is severely damaged and my albumin levels are low.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Proportion of drug responders
Toxicity profile

Side effects data

From 2014 Phase 3 trial • 92 Patients • NCT01520909
17%
Nasopharyngitis
16%
Rhinitis
13%
Epistaxis
11%
Upper respiratory tract infection
11%
Cough
10%
Headache
10%
Abdominal pain
6%
Pyrexia
6%
Aspartate Aminotransferase increased
5%
Alanine Aminotransferase increased
5%
Decreased appetite
5%
Vitamin D deficiency
5%
Abdominal pain upper
5%
Oropharyngeal pain
5%
Rash
5%
Toothache
5%
Diarrhoea
3%
Activated partial thromboplastin time prolonged
3%
Blood alkaline Phosphatase increased
3%
Blood creatinine increased
3%
Bronchitis
3%
Contusion
3%
Gingival bleeding
3%
Mouth haemorrhage
3%
Nausea
3%
Rhinorrhoea
3%
Vomiting
2%
Pneumonia fungal
2%
Impetigo
2%
Furuncle
2%
Retinal vascular disorder
2%
Constipation
2%
Excoriation
2%
Paraesthesia
2%
Soft tissue injury
2%
Cellulitis
2%
Anaemia
2%
Dyspepsia
2%
Allergy to chemicals
2%
Ear pain
2%
Rash pruritic
2%
Dermatitis allergic
2%
Gingivitis
2%
Groin pain
2%
Osteoporosis
2%
Influenza like illness
2%
Lip haemorrhage
2%
Menorrhagia
2%
Viral pharyngitis
2%
Pneumonia
2%
Influenza
2%
Joint injury
2%
Lice infestation
2%
Motion sickness
2%
Pharyngitis
2%
Platelet count increased
2%
Somnolence
2%
Subcutaneous abscess
2%
Tongue haemorrhage
2%
Tonsillar hypertrophy
2%
Meningitis aseptic
2%
Alanine aminotransferase abnormal
2%
Aspartate aminotransferase abnormal
2%
Gastritis
2%
Asthenia
2%
Back pain
2%
Bronchospasm
2%
Bulimia nervosa
2%
Non-cardiac chest pain
2%
Pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 1: Placebo
Part 1: Eltrombopag
Part 2: Eltrombopag

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: GroupExperimental Treatment1 Intervention
Eltrombopag
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eltrombopag
2013
Completed Phase 4
~960

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,943 Previous Clinical Trials
47,797,908 Total Patients Enrolled
10 Trials studying Fanconi Anemia
302 Patients Enrolled for Fanconi Anemia
Andre Larochelle, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
1 Previous Clinical Trials
500 Total Patients Enrolled

Media Library

Eltrombopag (Thrombopoietin Receptor Agonist) Clinical Trial Eligibility Overview. Trial Name: NCT03206086 — Phase 2
Fanconi Anemia Research Study Groups: Group
Fanconi Anemia Clinical Trial 2023: Eltrombopag Highlights & Side Effects. Trial Name: NCT03206086 — Phase 2
Eltrombopag (Thrombopoietin Receptor Agonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03206086 — Phase 2
~6 spots leftby May 2026
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