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Immunosuppressant

Eltrombopag for Aplastic Anemia

Phase 2
Waitlist Available
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
- Patients must agree to treatment with hATG + CsA concurrent with eltrombopag.
- Patients must not have been previously treated with IST, and must meet all criteria as described in Table 5-1.
Must not have
- Any cytogenetic abnormalities by karyotyping or FISH.
- Other known underlying inherited marrow failure syndrome (such as but not limited to Dyskeratosis Congenita, Congenital Amegakaryocytic Thrombocytopenia, or Shwachman-Diamond Syndrome).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 12, week 26, week 52, week 78, and then annually up to 3 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is a phase II, open label study of eltrombopag given orally to pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia. The study will have four periods: Screening, Treatment, Follow-up, and Long-term Follow-up.

Who is the study for?
This trial is for pediatric patients aged 1 to <18 with severe aplastic anemia (SAA) or recurrent aplastic anemia who haven't had a transplant option. They must not have certain bone marrow failure syndromes, prior immunosuppressive therapy, or active infections. Participants will receive eltrombopag alongside standard immunosuppressive treatments.
What is being tested?
The study tests how the body processes the drug eltrombopag when given with hATG and CsA in children with SAA or recurrent AA. It's an open-label, multi-center trial where doses of eltrombopag are increased within each patient to understand its pharmacokinetics.
What are the potential side effects?
Eltrombopag may cause liver issues, headaches, nausea, diarrhea, coughing, and increase the risk of blood clots. Immunosuppressants like hATG and CsA can lead to weakened immunity resulting in infections, as well as potential kidney damage and high blood pressure.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I agree to be treated with hATG, CsA, and eltrombopag together.
Select...
I have never been treated with IST.
Select...
I was previously diagnosed with severe aplastic anemia.
Select...
I have been diagnosed with severe aplastic anemia.
Select...
I am not a candidate for or have refused a stem cell transplant.
Select...
I can do most activities but may need help, regardless of my age.
Select...
I am between 1 and 17 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My tests show genetic abnormalities in my cells.
Select...
I have a known genetic condition affecting my bone marrow.
Select...
I have been diagnosed with Fanconi anemia.
Select...
I have been diagnosed with myelodysplastic syndrome.
Select...
I have taken eltrombopag or similar drugs for 2 months without improvement.
Select...
I have symptoms of PNH or more than half of my blood cells are PNH clones.
Select...
I have an infection that isn't getting better with treatment.
Select...
I have been diagnosed with cancer.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 12, week 26, week 52, week 78, and then annually up to 3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 12, week 26, week 52, week 78, and then annually up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Eltrombopag PK parameter: AUCtau
Eltrombopag PK parameter: Cmax
Eltrombopag PK parameter: Ctrough
Secondary study objectives
Acceptability and palatability for both tablets and powder for oral suspension
Alternate Overall response (aOR)
Bone marrow cellularity
+12 more

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%
Dyspepsia
2%
Retinal vascular disorder
2%
Constipation
2%
Excoriation
2%
Paraesthesia
2%
Soft tissue injury
2%
Cellulitis
2%
Anaemia
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

3Treatment groups
Experimental Treatment
Group I: Cohort BExperimental Treatment3 Interventions
previously untreated SAA, hATG (ATGAM®), CsA and eltrombopag begin on Day 1 and all patients will be treated with the same regimen
Group II: Cohort A (option 2)Experimental Treatment2 Interventions
CsA and eltrombopag begin on Day 1.
Group III: Cohort A (Option 1)Experimental Treatment3 Interventions
Regimen 1: hATG (ATGAM®), CsA and eltrombopag begin on Day 1.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eltrombopag
2013
Completed Phase 4
~960
hATG
2015
Completed Phase 3
~210
CsA
2016
Completed Phase 4
~370

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,916 Previous Clinical Trials
4,253,802 Total Patients Enrolled
4 Trials studying Aplastic Anemia
55 Patients Enrolled for Aplastic Anemia

Media Library

CsA (Immunosuppressant) Clinical Trial Eligibility Overview. Trial Name: NCT03025698 — Phase 2
Aplastic Anemia Research Study Groups: Cohort A (Option 1), Cohort B, Cohort A (option 2)
Aplastic Anemia Clinical Trial 2023: CsA Highlights & Side Effects. Trial Name: NCT03025698 — Phase 2
CsA (Immunosuppressant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03025698 — Phase 2
~6 spots leftby Dec 2025
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