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Immunomodulator

Abatacept for Chronic Cytopenia in CTLA4 Haploinsufficiency

Phase 1 & 2
Waitlist Available
Led By Gulbu Uzel, M.D.
Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up to be measured at day 210
Awards & highlights

Study Summary

This trial is testing the safety and efficacy of abatacept as a treatment for CTLA4 deficiency, which can cause problems with the immune system. Eligible participants will receive either the study drug or a placebo for six months, and will be monitored for improvement.

Eligible Conditions
  • CTLA4 Haploinsufficiency
  • Chronic Cytopenia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~to be measured at day 210
This trial's timeline: 3 weeks for screening, Varies for treatment, and to be measured at day 210 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Clinical Efficacy of abatacept in normalizing cytopenias
assess safety and tolerability of abacept given at double doses
Secondary outcome measures
Clinical efficacy of abatacept in improving but not normalizing cytopenais
measure of time to cytopenia recurrence

Side effects data

From 2018 Phase 4 trial • 16 Patients • NCT02078882
13%
Nausea
6%
Right upper quadrant pain
6%
Upper respiratory infection
6%
Hilar Adenopathy
6%
Urinary Tract Infections
6%
Vomiting
6%
Elevated liver enzymes
6%
Urticarial Rash
6%
Chest Pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Abatacept 125 mg Weekly

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: abataceptExperimental Treatment1 Intervention
Adult and pediatric dosing will be based on weight per protocol
Group II: placeboPlacebo Group1 Intervention
will be given as the same IV volume as abatacept
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
abatacept
2006
Completed Phase 4
~1180

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,265 Previous Clinical Trials
5,484,052 Total Patients Enrolled
Gulbu Uzel, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
2 Previous Clinical Trials
5,021 Total Patients Enrolled

Media Library

Abatacept (Immunomodulator) Clinical Trial Eligibility Overview. Trial Name: NCT03733067 — Phase 1 & 2
CTLA4 Haploinsufficiency Research Study Groups: placebo, abatacept
CTLA4 Haploinsufficiency Clinical Trial 2023: Abatacept Highlights & Side Effects. Trial Name: NCT03733067 — Phase 1 & 2
Abatacept (Immunomodulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03733067 — Phase 1 & 2

Frequently Asked Questions

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

Is registration open for this investigation presently?

"The trial information available on clinicaltrials.gov confirms that the experiment is actively seeking participants; it was originally uploaded to the website on December 6th, 2022 and underwent its latest update by November 30th of the same year."

Answered by AI

How many participants have registered for this medical trial to date?

"Indeed, based on the information available on clinicaltrials.gov, this medical trial is actively enrolling patients. It was posted online in December 6th 2022 and last updated at the end of November 2022. The research team has set a goal to recruit 40 participants from one centre for their study."

Answered by AI

Are individuals aged over 30 eligible for inclusion in this clinical experiment?

"This medical trial seeks participants aged 8 or above and under 65."

Answered by AI

What criteria must potential participants meet in order to be eligible for this research?

"This trial is searching for 40 volunteers between 8 and 65 years of age who have ctla4 haploinsufficiency. They must also not have increased their medication dose, received abatacept within the last two months or blood transfusions in the last 30 days, nor started any new medications to treat cytopenia during that time frame. Additionally, they should require active treatment with steroids, immunomodulatory agents like mycophenolate mofetil and cyclosporine , tacrolimus mercaptopurine methotrexate sirolimus and high-dose intravenous immunoglobulin"

Answered by AI

What afflictions does abatacept frequently alleviate?

"Abatacept is a therapeutic option for those living with rheumatoid arthritis, as well as psoriatic arthirtis, polyarticular juvenile idiopathic arthritis (JIA), and the more serious variant of JIA."

Answered by AI

What precedent has been established with regards to abatacept's application in clinical studies?

"Currently, abatacept is the subject of 34 ongoing trials, 8 of which are in their third phase. Multiple locations across Toronto offer these studies, with a total count of 771 clinics hosting research on this drug."

Answered by AI
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~0 spots leftby Mar 2025