Safety and Efficacy of Abatacept for Treating Chronic Cytopenia in Cytotoxic T-Lymphocyte Antigen… (NCT03733067) | Clinical Trial Compass
WithdrawnPhase 1/2
Safety and Efficacy of Abatacept for Treating Chronic Cytopenia in Cytotoxic T-Lymphocyte Antigen 4 (CTLA4) Haploinsufficiency
Stopped: No enrollment
United States0Started 2023-11-30
Plain-language summary
Background:
CTLA4 stands for cytotoxic T-lymphocyte antigen-4. It is a protein the body makes naturally to check its immune system from attacking itself. Some people don t produce enough CTLA4 protein, causing problems due to overactive immune system such as big spleens, repeated lung infections, breathing problems, stomach and intestine symptoms as well as inflamed brain and nerve problems. Many have problems with their bone marrow causing low numbers of blood cells like platelets, red blood cells or white blood cells, which is called cytopenia. Researchers want to see if the drug abatacept can treat cytopenias by replacing the missing protein CTLA4.
Objective:
To see if abatacept is safe and helps treat cytopenias caused by CTLA4 deficiency.
Eligibility:
People ages 8-65 years who have CTLA4 deficiency with cytopenia
Design:
Participants will be screened with medical history, medication review, physical exam and blood and urine tests. They will continue their current medications and may start taking antibiotics daily. Participants will receive either abatacept or placebo through a vein for 6 months. The study team will not know if you are receiving the study drug or the placebo
Women who can become pregnant must agree to use birth control measures.
Men who get someone pregnant during the study will be asked to collect information and have the partner contact the study team.
Participants will undergo the following procedures before starting the study and at the completion:
* radiology scans of body and brain
* heart and lung function tests
* Bone marrow examination by a needle inserted into the hip bone to remove a small amount of tissue to study.
* Participants may have a small camera on a long, thin tool passed down the throat into the stomach and small intestine for evaluation of their gut.
* Questionnaires about their disease, symptoms and quality of life
Over 6 months, participants will have regular study visits and get 8 doses of the study drug or a placebo by intravenous injection. They will repeat some of the same tests done earlier at the end of the study at assess response.
About 1 month after the last study drug visit, participants will have a final study visit.
Some participants may join a treatment extension for the study drug abatacept with no placebo. They will sign a separate consent form for this.
Who can participate
Age range
8 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age 8-65 years.
. Documented CTLA4 mutation (requires documentation of confirmed mutation via Sanger sequencing at a laboratory approved by the Clinical Laboratory Improvement Amendments \[CLIA\]).
. At least one of the following established hematologic abnormalities during the past 6 months (including results from outside CLIA-certified laboratories) prior to screening:
. The above mentioned hematologic abnormalities should require active treatment with steroids, immunomodulatory agents (e.g., mycophenolate mofetil, cyclosporine, tacrolimus, mercaptopurine, methotrexate, sirolimus, high dose intravenous immunoglobulin \[IVIG\]), and/or other agents (e.g., TPO agonists) for at least 60 days prior to screening.
. The dose of any concomitant medication(s) aimed at treating cytopenia should be stable in the 60 days prior to screening. Stable is defined as:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
assess safety and tolerability of abacept given at double doses
Timeframe: To be measured at day 210
2
Clinical Efficacy of abatacept in normalizing cytopenias
Timeframe: To be measured at day 210
Trial details
NCT IDNCT03733067
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)
. Did not receive blood product transfusions within 30 days prior to screening.
. Did not receive abatacept within 60 days prior to screening.
. Did not receive rituximab within 3060 days of screening.
Exclusion criteria
. History of hypersensitivity to abatacept.
. Any live vaccines (including attenuated live vaccines) within 6 weeks of screening.
. History of acquired immunodeficiency diseases, including a positive HIV polymerase chain reaction (PCR) test result.
. Untreated chronic hepatitis B (positive PCR) or hepatitis C (positive PCR) infection. Patients with chronic hepatitis must be on medical treatment for at least 3 months before screening and have evidence of decreased viral loads after starting treatment.
. EBV viral load \> 4log on 2 or more laboratory checks greater than 1 month apart and within 6 months of screening.
. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of evidence of local recurrence or metastases.
. Current active infectious disease (bacterial or fungal) including evidence of tuberculosis (TB) infection as defined by a positive QuantiFERON TB-Gold test. Test results within the past 6 months will be accepted. If presence of latent TB is established, then treatment must be completed before the patient can be considered for enrollment. The patient may also be considered for enrollment after completing treatment of any other active bacterial or fungal infection.