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Immunosuppressant

Eltrombopag for Aplastic Anemia

Q: Is Eltrombopag a viable therapeutic option with minimal associated risks?

The safety of eltrombopag was rated a 2, as this Phase 2 trial has demonstrated some data suggesting its security but there is yet to be any research that demonstrates efficacy.

Q: How many individuals are actively participating in the experiment?

Currently, this clinical trial is not enrolling new patients. The study was initially posted on September 30th 2017 and last edited November 21st 2022. If you're looking for a different medical study, there are currently 243 trials related to aplastic anemia and 93 studies involving Eltrombopag actively searching for candidates.

Q: Is there an opportunity to volunteer for this scientific research?

The eligibility requirements for this medical trial mandate that potential participants have aplastic anemia and are between one year old to eighteen. This research is seeking 51 individuals in total.

Q: Are elderly individuals eligible to be participants in this experiment?

Per the guidelines for this research, only individuals between 1 year and 18 years of age can be considered for enrollment.

Q: In what kinds of conditions is Eltrombopag typically prescribed?

Eltrombopag has been used to manage the symptoms of transplantation, excessive tearing, and <a href="https://www.withpower.com/clinical-trials/lupus">lupus</a> nephritis.

Q: Is there still scope to join this research project?

Clinicaltrials.gov data reveals that recruitment for this medical trial, first posted on September 30th 2017, has concluded. However, there are still 336 other studies requiring volunteers at the present moment.

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.

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

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 12, week 26, week 52, week 78, and then annually up to 3 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Eltrombopag PK parameter: AUCtau

Eltrombopag PK parameter: Cmax

Eltrombopag PK parameter: Ctrough

Secondary outcome measures

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

Pyrexia

Aspartate Aminotransferase increased

Decreased appetite

Alanine Aminotransferase increased

Vitamin D deficiency

Abdominal pain upper

Oropharyngeal pain

Rash

Toothache

Diarrhoea

Activated partial thromboplastin time prolonged

Blood alkaline Phosphatase increased

Blood creatinine increased

Bronchitis

Contusion

Gingival bleeding

Mouth haemorrhage

Nausea

Rhinorrhoea

Vomiting

Cellulitis

Soft tissue injury

Constipation

Groin pain

Dyspepsia

Excoriation

Gingivitis

Furuncle

Retinal vascular disorder

Pneumonia fungal

Osteoporosis

Ear pain

Allergy to chemicals

Paraesthesia

Rash pruritic

Impetigo

Anaemia

Dermatitis allergic

Influenza like illness

Lip haemorrhage

Menorrhagia

Viral pharyngitis

Pneumonia

Influenza

Joint injury

Lice infestation

Motion sickness

Pharyngitis

Platelet count increased

Somnolence

Subcutaneous abscess

Tongue haemorrhage

Tonsillar hypertrophy

Meningitis aseptic

Alanine aminotransferase abnormal

Aspartate aminotransferase abnormal

Gastritis

Asthenia

Back pain

Bronchospasm

Bulimia nervosa

Non-cardiac chest pain

Pain

100%

80%

60%

40%

20%

Study treatment Arm

Part 1: Placebo

Part 1: Eltrombopag

Part 2: Eltrombopag

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

CsA

2016

Completed Phase 4

~370

hATG

2015

Completed Phase 3

~210

Eltrombopag

2013

Completed Phase 4

~970

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor

2,855 Previous Clinical Trials

4,197,299 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is Eltrombopag a viable therapeutic option with minimal associated risks?

"The safety of eltrombopag was rated a 2, as this Phase 2 trial has demonstrated some data suggesting its security but there is yet to be any research that demonstrates efficacy."

Answered by AI

How many individuals are actively participating in the experiment?

"Currently, this clinical trial is not enrolling new patients. The study was initially posted on September 30th 2017 and last edited November 21st 2022. If you're looking for a different medical study, there are currently 243 trials related to aplastic anemia and 93 studies involving Eltrombopag actively searching for candidates."

Answered by AI

Is there an opportunity to volunteer for this scientific research?

"The eligibility requirements for this medical trial mandate that potential participants have aplastic anemia and are between one year old to eighteen. This research is seeking 51 individuals in total."

Answered by AI

Are elderly individuals eligible to be participants in this experiment?

"Per the guidelines for this research, only individuals between 1 year and 18 years of age can be considered for enrollment."

Answered by AI

In what kinds of conditions is Eltrombopag typically prescribed?

"Eltrombopag has been used to manage the symptoms of transplantation, excessive tearing, and lupus nephritis."

Answered by AI

Is there still scope to join this research project?

"Clinicaltrials.gov data reveals that recruitment for this medical trial, first posted on September 30th 2017, has concluded. However, there are still 336 other studies requiring volunteers at the present moment."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

2017. "A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03025698.

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