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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
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 belowSelect...
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:Select...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ 6 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months
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 • NCT0152090917%
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%
Anaemia
2%
Constipation
2%
Impetigo
2%
Retinal vascular disorder
2%
Cellulitis
2%
Allergy to chemicals
2%
Furuncle
2%
Dermatitis allergic
2%
Dyspepsia
2%
Ear pain
2%
Excoriation
2%
Gingivitis
2%
Groin pain
2%
Osteoporosis
2%
Influenza like illness
2%
Paraesthesia
2%
Rash pruritic
2%
Soft tissue injury
2%
Pneumonia fungal
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
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
~970
Find a Location
Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,889 Previous Clinical Trials
47,834,172 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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am at least 2 years old.I cannot take medicine by mouth.I have a heart condition that could make participating in the study unsafe.You have a history of abusing alcohol or drugs.I have been diagnosed with Fanconi anemia through genetic testing or chromosome analysis.You have a known allergy to EPAG or any of its parts.I am not pregnant or nursing.I am a sexually active male and agree to use a condom during the study and for 30 days after.You are very sick and likely to pass away within the next 7 to 10 days.I haven't taken androgens like danazol or oxymetholone in the last 4 weeks.I have a genetic mutation in both copies of my BRCA2 gene.I am cancer-free with no expected recurrence within a year.I have low blood counts or need regular blood transfusions.I have had blood clots in the past.My test shows genetic changes linked to a worse outlook in my condition.You weigh more than 10 kilograms.I have either not responded to or chosen not to take androgen therapy.I am a woman able to have children and am not using strong birth control methods.I have severe health issues that prevent me from taking eltrombopag.My condition meets the WHO criteria for MDS or AML.I have been diagnosed with HIV.Your creatinine levels are more than 2.5 times the normal limit.Your liver enzyme levels are more than 5 times the normal limit.My direct bilirubin level is higher than 3.0mg/dL.I have an infection that isn't getting better with treatment.My liver is severely damaged and my albumin levels are low.
Research Study Groups:
This trial has the following groups:- Group 1: Group
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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