CTLA4 HaploinsufficencyPhase 1/2NCT03733067
Safety and Efficacy of Abatacept for Treating Chronic Cytopenia in Cytotoxic T-Lymphocyte Antigen 4 (CTLA4) Haploinsufficiency
A Phase 1/2 study testing abatacept in people with CTLA4 Haploinsufficency and Chronic Cytopenia, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 8 to 65 years
- Conditions the study is for
- CTLA4 Haploinsufficency, Chronic Cytopenia
- What is being tested?
- abatacept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Nov 2023to 30 Nov 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Individuals must meet all of the following criteria to be eligible for study participation:
- 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:
- ANC < 750 cells/microL.
- Platelet count < 75,000 cells/microL.
Show all 21Show fewer
- Hemoglobin < 7.5 g/dL.
- 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:
- No new concomitant medications for cytopenia were initiated.
- No dose increase of the medication was required.
- 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.
- Did not receive alemtuzumab at any time.
- Has access to healthcare provider at home.
- Able to provide informed consent.
- Willing to allow storage of biological specimens for future use in medical research.
- Females of childbearing potential must agree to use appropriate birth control methods when engaging in sexual activities that can result in pregnancy, beginning Day -30 through 30 days after the last dose of study agent. Appropriate methods should include 2 forms of contraception, one from each of the following categories:
- Hormonal contraception or placement of an intrauterine device or intrauterine system.
- Barrier method: Condom or occlusive cap (diaphragm or cervical/vault cap) with a spermicide.
Who cannot join
- Patients meeting any of the following criteria are not eligible for this study:
- 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.
Show all 11Show fewer
- 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.
- Contraindication to PFT or CT scan.
- Pregnancy or breastfeeding.
- Any condition that, in the opinion of the investigator, contraindicates participation in this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Phase 1/2 Randomized, Double-Blind, Placebo-Controlled Study of Safety and Efficacy of Abatacept for Treating Chronic Cytopenia in Cytotoxic T-lymphocyte Antigen 4 (CTLA4) Haploinsufficiency
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.
Read more
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.
Read the detailed description
Rare heterozygous mutations in cytotoxic T-lymphocyte antigen-4 (CTLA4) lead to a monogenetic defect that presents with a heterogeneous clinical phenotype of recurrent infections, lymphoproliferation, autoimmunity, and lymphocytic infiltration of target organs. Management is challenging and focuses on treating infections, autoimmune complications, and end organ damage due to lymphocytic infiltrates. Experience with the natural history of the disease and therapies for underlying complications are limited. There is no established standard of care for these patients.
Abatacept is a biologically engineered CTLA4-mimetic that is approved as an intravenous (IV) infusion to treat adult rheumatoid arthritis, adult psoriatic arthritis, and juvenile idiopathic arthritis. Given that abatacept mimics CTLA4 function, we hypothesize that the drug will prevent T-cell hyper-activation, restore regulatory T-cell function, and thereby treat the autoimmune and immune dysregulatory manifestations of CTLA4 deficiency.
This study is a phase 1/2, double-blind, randomized, intra-patient dose-escalation, placebo-controlled trial designed to evaluate the safety and efficacy of abatacept in participants with CTLA4 deficiency and cytopenia. Participants will come to the NIH Clinical Center monthly for 210 days to receive infusions of study agent or placebo and to undergo safety and research evaluations, including blood draw for cytopenia evaluation and scoring of disease severity. Before and after the treatment period, participants will also have imaging, pulmonary function testing, bone marrow biopsy, and endoscopy (symptomatic participants only). Primary endpoints will be evaluated at Day 210. After completing the blinded treatment trial, participants will be offered the option to enroll in a 6-month open-label extension study for long-term safety evaluation of abatacept.
- Conditions
- CTLA4 Haploinsufficency, Chronic Cytopenia
- Being tested
- abatacept, Placebo
- Keywords
- Regulatory T Cells, T-cell hyperactivation, Autoimmune Cytopenia, Adult Rheumatoid Arthritis, Juvenile Idiopathic Arthritis
- Medical subject headings
- Arthritis, Rheumatoid, Arthritis, Juvenile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
abataceptDrug
Double-blind, placebo-controlled, intra-patient dose-escalation trial of abatacept for treating cytopenia in CTLA4 deficiency. Abatacept or placebo will be administered for 3 doses over 30 days, followed by 5 more months of administration at double the initial dose. If the participant s hematologic parameters normalize at Day 120 or onwards, the investigator may taper or discontinue concomitant medications aimed at treating cytopenia.
PlaceboOther
Saline packaged identically to abatacept, with volume matching that of the abatacept dosing by weight, and will be administered via IV infusion identically to abatacept
| Study group | Role | What they receive |
|---|---|---|
| abatacept | Experimental | Adult and pediatric dosing will be based on weight per protocol Receives: Drug: abatacept |
| placebo | Placebo comparator | will be given as the same IV volume as abatacept Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
assess safety and tolerability of abacept given at double doses
All safety parameters (including possibly to definitely related AEs and SAEs, physical exam, vital signs, ECG, safety laboratories [hematology, blood chemistry, urinalysis]) and incidence and severity of infections at initial and doubled doses of abatacept at Day 210.
Measured: To be measured at day 210
Clinical Efficacy of abatacept in normalizing cytopenias
A complete response defined by the normalization of all listed hematologic parameters:A) ANC > 1,000 cells/microliter; andB) Platelet count > 100,000 cells/microliter; andC) Hemoglobin > 10 g/dLParameters will be measured at Day 210 and must be met without any transfusions and concomitant medications (immunomodulatory therapy, exogenous growth factor, steroids, hematopoietic/TPO mimetic agent) aimed at treating cytopenias during the past 28 days.
Measured: To be measured at day 210
Secondary outcomes (2)
measure of time to cytopenia recurrence
Measured: From initiating the taper or discontinuation of concomitant medications while on treatment
Clinical efficacy of abatacept in improving but not normalizing cytopenais
Partial hematologic response defined as an improvement in one or more of the listed parameters but not meeting complete hematologic response:A) ANC: a 100% increase from baseline or an ANC >= 750 cells/microL.B) Platelet: for baseline count >= 20,000/microL but < 75,000/microL: a 100% increase in platelet count or a platelet count > 75,000/ (Micro)L; for baseline platelet count < 20,000/micro: a 100% increase in platelet count or a platelet count > 20,000/microL.C) Any increase in hemoglobin by 2 g/dL.All of the parameters will be measured at Day 210 and must be met without any transfusions and concomitant medications (immunomodulatory therapy, exogenous growth factor, steroids, hematopoietic/TPO mimetic agent) aimed at treating cytopenias during the past 28 days. OR A normalization of hematologic parameters as defined in the primary endpoint AND a dose reduction or discontinuation of baseline concomitant medications.
Measured: To be measured at Day 210
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2018
- Study start30 Nov 2023
- Primary completion30 Nov 2023
- Study completion30 Nov 2023
Why it stopped: No enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for CTLA4 HaploinsufficencyStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Gulbu Uzel, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03733067
- Sponsor's protocol ID
- 190015
- Other IDs
- 19-I-0015
- Status last verified
- Nov 2023
- First submitted
- 6 Nov 2018
- Last updated
- 4 Dec 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lo B, Zhang K, Lu W, Zheng L, Zhang Q, Kanellopoulou C, Zhang Y, Liu Z, Fritz JM, Marsh R, Husami A, Kissell D, Nortman S, Chaturvedi V, Haines H, Young LR, Mo J, Filipovich AH, Bleesing JJ, Mustillo P, Stephens M, Rueda CM, Chougnet CA, Hoebe K, McElwee J, Hughes JD, Karakoc-Aydiner E, Matthews HF, Price S, Su HC, Rao VK, Lenardo MJ, Jordan MB. AUTOIMMUNE DISEASE. Patients with LRBA deficiency show CTLA4 loss and immune dysregulation responsive to abatacept therapy. Science. 2015 Jul 24;349(6246):436-40. doi: 10.1126/science.aaa1663.
PubMed 26206937Lee S, Moon JS, Lee CR, Kim HE, Baek SM, Hwang S, Kang GH, Seo JK, Shin CH, Kang HJ, Ko JS, Park SG, Choi M. Abatacept alleviates severe autoimmune symptoms in a patient carrying a de novo variant in CTLA-4. J Allergy Clin Immunol. 2016 Jan;137(1):327-330. doi: 10.1016/j.jaci.2015.08.036. Epub 2015 Oct 21. No abstract available.
PubMed 26478010Salman J, Ius F, Knoefel AK, Sommer W, Siemeni T, Kuehn C, Tudorache I, Avsar M, Nakagiri T, Preissler G, Hatz R, Greer M, Welte T, Haverich A, Warnecke G. Association of Higher CD4+ CD25high CD127low , FoxP3+ , and IL-2+ T Cell Frequencies Early After Lung Transplantation With Less Chronic Lung Allograft Dysfunction at Two Years. Am J Transplant. 2017 Jun;17(6):1637-1648. doi: 10.1111/ajt.14148. Epub 2017 Jan 24.
PubMed 27931084
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2 Randomized, Double-Blind, Placebo-Controlled Study of Safety and Efficacy of Abatacept for Treating Chronic Cytopenia in Cytotoxic T-lymphocyte Antigen 4 (CTLA4) Haploinsufficiency
- ClinicalTrials.gov ID
- NCT03733067
- Acronym / sponsor ID
- 190015
- Conditions
- CTLA4 Haploinsufficency, Chronic Cytopenia
- Keywords
- Regulatory T Cells, T-cell hyperactivation, Autoimmune Cytopenia, Adult Rheumatoid Arthritis, Juvenile Idiopathic Arthritis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No enrollment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 30 Nov 2023 (actual)
- Primary completion
- 30 Nov 2023 (actual)
- Study completion
- 30 Nov 2023 (actual)
- First posted
- 7 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Rare heterozygous mutations in cytotoxic T-lymphocyte antigen-4 (CTLA4) lead to a monogenetic defect that presents with a heterogeneous clinical phenotype of recurrent infections, lymphoproliferation, autoimmunity, and lymphocytic infiltration of target organs. Management is challenging and focuses on treating infections, autoimmune complications, and end organ damage due to lymphocytic infiltrates. Experience with the natural history of the disease and therapies for underlying complications are limited. There is no established standard of care for these patients.
Abatacept is a biologically engineered CTLA4-mimetic that is approved as an intravenous (IV) infusion to treat adult rheumatoid arthritis, adult psoriatic arthritis, and juvenile idiopathic arthritis. Given that abatacept mimics CTLA4 function, we hypothesize that the drug will prevent T-cell hyper-activation, restore regulatory T-cell function, and thereby treat the autoimmune and immune dysregulatory manifestations of CTLA4 deficiency.
This study is a phase 1/2, double-blind, randomized, intra-patient dose-escalation, placebo-controlled trial designed to evaluate the safety and efficacy of abatacept in participants with CTLA4 deficiency and cytopenia. Participants will come to the NIH Clinical Center monthly for 210 days to receive infusions of study agent or placebo and to undergo safety and research evaluations, including blood draw for cytopenia evaluation and scoring of disease severity. Before and after the treatment period, participants will also have imaging, pulmonary function testing, bone marrow biopsy, and endoscopy (symptomatic participants only). Primary endpoints will be evaluated at Day 210. After completing the blinded treatment trial, participants will be offered the option to enroll in a 6-month open-label extension study for long-term safety evaluation of abatacept.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| abatacept | Experimental | Drug: abataceptAdult and pediatric dosing will be based on weight per protocol |
| placebo | Placebo comparator | Other: Placebowill be given as the same IV volume as abatacept |
Who may qualify
- Ages
- 8 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (21 listed)
- Individuals must meet all of the following criteria to be eligible for study participation:
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| abataceptDouble-blind, placebo-controlled, intra-patient dose-escalation trial of abatacept for treating cytopenia in CTLA4 deficiency. Abatacept or placebo will be administered for 3 doses over 30 days, followed by 5 more months of administration at double the initial dose. If the participant s hematologic parameters normalize at Day 120 or onwards, the investigator may taper or discontinue concomitant medications aimed at treating cytopenia. | Drug | abatacept |
| PlaceboSaline packaged identically to abatacept, with volume matching that of the abatacept dosing by weight, and will be administered via IV infusion identically to abatacept | Other | placebo |
Primary outcome
assess safety and tolerability of abacept given at double doses
Time frame: To be measured at day 210
All safety parameters (including possibly to definitely related AEs and SAEs, physical exam, vital signs, ECG, safety laboratories [hematology, blood chemistry, urinalysis]) and incidence and severity of infections at initial and doubled doses of abatacept at Day 210.
Clinical Efficacy of abatacept in normalizing cytopenias
Time frame: To be measured at day 210
A complete response defined by the normalization of all listed hematologic parameters:A) ANC > 1,000 cells/microliter; andB) Platelet count > 100,000 cells/microliter; andC) Hemoglobin > 10 g/dLParameters will be measured at Day 210 and must be met without any transfusions and concomitant medications (immunomodulatory therapy, exogenous growth factor, steroids, hematopoietic/TPO mimetic agent) aimed at treating cytopenias during the past 28 days.
2 secondary outcomes
measure of time to cytopenia recurrence
Time frame: From initiating the taper or discontinuation of concomitant medications while on treatment
Clinical efficacy of abatacept in improving but not normalizing cytopenais
Time frame: To be measured at Day 210
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2023.
- Background: PubMed 26206937Lo B, Zhang K, Lu W, Zheng L, Zhang Q, Kanellopoulou C, Zhang Y, Liu Z, Fritz JM, Marsh R, Husami A, Kissell D, Nortman S, Chaturvedi V, Haines H, Young LR, Mo J, Filipovich AH, Bleesing JJ, Mustillo P, Stephens M, Rueda CM, Chougnet CA, Hoebe K, McElwee J, Hughes JD, Karakoc-Aydiner E, Matthews HF, Price S, Su HC, Rao VK, Lenardo MJ, Jordan MB. AUTOIMMUNE DISEASE. Patients with LRBA deficiency show CTLA4 loss and immune dysregulation responsive to abatacept therapy. Science. 2015 Jul 24;349(6246):436-40. doi: 10.1126/science.aaa1663.
- Background: PubMed 26478010Lee S, Moon JS, Lee CR, Kim HE, Baek SM, Hwang S, Kang GH, Seo JK, Shin CH, Kang HJ, Ko JS, Park SG, Choi M. Abatacept alleviates severe autoimmune symptoms in a patient carrying a de novo variant in CTLA-4. J Allergy Clin Immunol. 2016 Jan;137(1):327-330. doi: 10.1016/j.jaci.2015.08.036. Epub 2015 Oct 21. No abstract available.
- Background: PubMed 27931084Salman J, Ius F, Knoefel AK, Sommer W, Siemeni T, Kuehn C, Tudorache I, Avsar M, Nakagiri T, Preissler G, Hatz R, Greer M, Welte T, Haverich A, Warnecke G. Association of Higher CD4+ CD25high CD127low , FoxP3+ , and IL-2+ T Cell Frequencies Early After Lung Transplantation With Less Chronic Lung Allograft Dysfunction at Two Years. Am J Transplant. 2017 Jun;17(6):1637-1648. doi: 10.1111/ajt.14148. Epub 2017 Jan 24.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gulbu Uzel, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing abatacept in people with CTLA4 Haploinsufficency and Chronic Cytopenia, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 4 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
assess safety and tolerability of abacept given at double doses (time frame: To be measured at day 210). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03733067, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2023.