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Thrombopoietin Receptor Agonist
Eltrombopag for Diamond-Blackfan Anemia
Phase 1 & 2
Waitlist Available
Led By David J Young, 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
Weight greater than or equal to 12 kilograms
Clinically-significant anemia as defined as either: hemoglobin less than 9.0 g/dL red cell transfusion of at least 2 units PRBC for adults or 30 cc/kg for children (whichever is less) in the eight weeks prior to study enrollment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 3 years
Awards & highlights
Study Summary
This trial is testing the safety and efficacy of eltrombopag in people with Diamond-Blackfan anemia (DBA) who have not responded to steroids or could not take them.
Who is the study for?
This trial is for people aged 2 and older with Diamond-Blackfan anemia who haven't responded to steroids or can't take them. They must weigh at least 12 kg, live in the US or be able to stay there during the trial, and not have certain severe health conditions or be pregnant. People taking certain drugs or with a history of blood clots (other than catheter-related) are excluded.Check my eligibility
What is being tested?
The study tests Eltrombopag's ability to increase red blood cells in patients with refractory Diamond-Blackfan anemia. Participants will take daily pills for 24 weeks, have regular blood tests, and undergo various assessments including MRI scans and bone marrow biopsies. If successful, treatment may continue up to three years.See study design
What are the potential side effects?
Eltrombopag might cause liver issues, heart problems if predisposed (QT prolongation), potential bleeding risks due to increased platelets count especially if it exceeds normal levels (>400k/microliter), as well as possible interactions leading to altered drug metabolism.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I weigh at least 12 kilograms.
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I have severe anemia or needed a blood transfusion recently.
Select...
I am at least 2 years old.
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My condition worsened after or didn't respond to steroids.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of Adverse Events
Participants That Responded to Eltrombopag
Time (Weeks) to Response
Secondary outcome measures
Health-related Quality-of-life
Iron Overload
Long-term Safety of Drug
+4 moreSide 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%
Decreased appetite
5%
Alanine Aminotransferase increased
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%
Soft tissue injury
2%
Constipation
2%
Groin pain
2%
Furuncle
2%
Cellulitis
2%
Retinal vascular disorder
2%
Dyspepsia
2%
Osteoporosis
2%
Pneumonia fungal
2%
Ear pain
2%
Allergy to chemicals
2%
Paraesthesia
2%
Gingivitis
2%
Excoriation
2%
Rash pruritic
2%
Impetigo
2%
Anaemia
2%
Dermatitis allergic
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
Trial Design
1Treatment groups
Experimental Treatment
Group I: Refractory Diamond-Blackfan Anemia in EltrombopagExperimental Treatment1 Intervention
Participants with Refractory Diamond-Blackfan Anemia will be administered Eltrombopag. Participants 12 years of age an above will receive the adult dose of 150 mg by mouth daily. Participants between ages of 6 and 11 years old will start at 75 mg by mouth daily, and children 2 and 5 years of age will start at 2.5 mg/kg, not to exceed 75 mg by mouth daily.
To adjust for the higher expected exposure in participants of East Asian and South East Asian ancestry, the starting dose for East Asian and South East Asian participants 12 years of age and above will be 75 mg by mouth once daily. For East Asian and South East Asian participants between 6 and 11 years of age, the starting dose will be 37.5 mg once daily by mouth, and for children between 2 and 5, the starting dose will be 1.25 mg/kg by mouth.
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
3,837 Previous Clinical Trials
47,851,612 Total Patients Enrolled
David J Young, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
1 Previous Clinical Trials
30 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have an infection that isn't getting better with treatment.You are not expected to live for more than 3 months due to any reason.My liver is severely damaged and my albumin levels are low.I do not have severe health issues that would stop me from tolerating the treatment.I was diagnosed with DBA after age 3 and have a confirmed gene mutation.You are currently using alcohol or drugs excessively.My kidney function is severely reduced.My condition meets the WHO criteria for MDS or AML.I am not on medications that affect eltrombopag's action.I have had blood clots not related to catheter use.I am using or willing to use effective birth control methods if I can become pregnant.I do not have significant heart problems or recent serious heart issues.You are allergic to eltrombopag or any of its ingredients.I weigh at least 12 kilograms.I haven't had chemotherapy, specific cancer drugs, or radiation in the last 6 months.I cannot take the study medication.I am at risk of my cancer returning within the next year.I have severe anemia or needed a blood transfusion recently.I am not pregnant or nursing.My bilirubin level is higher than 2.0 mg/dL.I can understand the study details and can give informed consent, or I have someone who can do it on my behalf.I was diagnosed with Diamond-Blackfan anemia before I turned three.I am at least 2 years old.I haven't taken androgens or corticosteroids in the last 4 weeks.My condition worsened after or didn't respond to steroids.
Research Study Groups:
This trial has the following groups:- Group 1: Refractory Diamond-Blackfan Anemia in Eltrombopag
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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What are the goals that this clinical trial is hoping to achieve?
"The primary objective of this 6-month study is to assess the toxicity of the experimental medication. Secondary goals include observing neurodevelopment in pediatric patients, measuring rates of relapse and survival, and Platelet count as measured by serial CBC assessments."
Answered by AI
How large is the pool of individuals being monitored in this experiment?
"The study has completed recruitment as of October 5th, 2022. The listing was first posted on December 1st, 2020. If you are interested in similar trials, there are currently 235 studies for anemia, diamond-blackfan and 25 trials for Eltrombopag actively admitting participants."
Answered by AI
Is it still possible to sign up for this research project?
"Unfortunately, this particular trial is no longer enrolling patients. Although the last update was on October 5th, 2022, the study has not been recruiting since December 1st 2020. However, there are 260 other trials that are still open for enrollment."
Answered by AI
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