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Immunosuppressant
Eltrombopag + Immunosuppression for Severe Aplastic Anemia
Phase 1 & 2
Recruiting
Led By Neal S 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
Age greater than or equal to 2 years old
Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent (excluding lymphocytes)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 months to 5 years
Awards & highlights
Study Summary
This trial is testing the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia.
Who is the study for?
This trial is for individuals aged 2 years and older with severe aplastic anemia who haven't been treated yet. They must have specific low blood cell counts, weigh over 12 kg, and not have certain liver issues or infections. People with prior immunosuppressive therapy, pregnancy, or severe concurrent diseases can't participate.Check my eligibility
What is being tested?
The trial tests the addition of a drug called Eltrombopag to standard treatment (horse-ATG and cyclosporine) for severe aplastic anemia. Participants are divided into groups receiving Eltrombopag at different times and durations alongside their standard care.See study design
What are the potential side effects?
Eltrombopag may cause liver problems, headaches, nausea, diarrhea, coughing up blood or bleeding more easily than normal. Horse-ATG can lead to allergic reactions and cyclosporine might result in kidney damage or high blood pressure.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am at least 2 years old.
Select...
My bone marrow is very underactive.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3 months to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 months to 5 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Rate of Complete Hematologic Response
Secondary outcome measures
Rate of Response at 3 and 12 Months Then Yearly; Rate of Relapse; Rate of Clonal Evolution to PNH, MDS and AML; Rate of Survival; Rate of Response for Relapse Subjects That Re-start Treatment and Effects of CsA Dose Starting at Month 6 to Month 24.
Trial Design
4Treatment groups
Experimental Treatment
Group I: Extrension CohortExperimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18 months, and receive eltrombopag day 1 to month 6
Group II: Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6Experimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6
Group III: Cohort 2: hATG, CsA, EPAG Day 14 to Month 3Experimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3
Group IV: Cohort 1: hATG, CsA, EPAG Day 14 to Month 6Experimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6
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Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,837 Previous Clinical Trials
47,851,387 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
NovartisIndustry Sponsor
1,611 Previous Clinical Trials
2,720,828 Total Patients Enrolled
2 Trials studying Aplastic Anemia
89 Patients Enrolled for Aplastic Anemia
Neal S Young, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
18 Previous Clinical Trials
2,797 Total Patients Enrolled
1 Trials studying Aplastic Anemia
27 Patients Enrolled for Aplastic Anemia
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My liver is too weak for certain medications due to low albumin levels.I do not have severe health issues that would stop me from tolerating the treatment.I am at least 2 years old.I have been diagnosed with Fanconi anemia.My tests show a genetic disorder related to blood cell production, but I might have very low white blood cells.I have previously received immunosuppressive therapy.My infection is not getting better despite treatment.I am not currently on chemotherapy or drugs affecting blood for my cancer.I understand the study's purpose and can give informed consent.My bone marrow is very underactive.
Research Study Groups:
This trial has the following groups:- Group 1: Extrension Cohort
- Group 2: Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6
- Group 3: Cohort 2: hATG, CsA, EPAG Day 14 to Month 3
- Group 4: Cohort 1: hATG, CsA, EPAG Day 14 to Month 6
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.
Who meets the criteria to partake in this medical experiment?
"This investigation is accepting 207 individuals, aged two to ninety-five who have aplastic anemia. In order to join the trial, patients must weigh more than 12 kilograms and be at least 2 years old."
Answered by AI
Is this medicinal experiment recruiting participants?
"Affirmative, the information posted on clinicaltrials.gov shows that this medical study is still recruiting participants as of April 5th 2022. The trial was initially published on July 2nd 2012 and seeks to enrol 207 patients from a single site."
Answered by AI
What medical issue does the treatment regimen Cohort 1: hATG, CsA and EPAG Day 14 to Month 6 address?
"Cohort 1: hATG, CsA and EPAG administered between day 14 to month 6 is typically employed as a therapy for transplantation. It has also been known to help with conditions such as excessive tearing, lupus nephritis and bulla."
Answered by AI
Does this trial allow for participation from geriatric individuals?
"This research project outlines an age range of 2 to 95 for participants; thus, any person within that window can be accepted."
Answered by AI
How many individuals are joining this trial's roster?
"Yes, the information available online indicates that this clinical trial is in need of participants. It was initially advertised on July 2nd 2012 and underwent its latest update on April 5th 2022. This study seeks 207 individuals from a single location for further evaluation."
Answered by AI
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