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Costimulation Blockade

Abatacept for Graft-versus-Host Disease Prevention (ASCENT Trial)

Phase 2
Waitlist Available
Led By John Horan, MD
Research Sponsored by Emory University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
- Previous clinical stroke, as evidenced by a neurological deficit lasting longer than 24 hours, which is accompanied by radiographic evidence of ischemic brain injury and cerebral vasculopathy.
Must have one of the following diseases: Glanzmann thrombasthenia, Chronic granulomatous disease, Severe congenital neutropenia (with resistance to granulocyte colony-stimulating factor (GCSF) or chronic requirement of GCSF doses ≥10 mcg/kg), Leukocyte adhesion deficiency, Shwachman-Diamond syndrome, Diamond-Blackfan Anemia (DBA; transfusion dependent, including steroid failure or inability to wean steroids), Thalassemia major, FA, Dyskeratosis congenita, Chediak Higashi syndrome, Acquired (immune; non-inherited, non-congenital) SAA.
Must not have
Pulmonary dysfunction defined as diffusing capacity of the lungs for carbon monoxide (DLCO; corrected for hemoglobin), forced expiratory volume in one second (FEV1), or forced vital capacity (FVC) < 40% of predicted. In a child unable to perform pulmonary function testing, a chronic need for supplemental oxygen will serve as the exclusionary criterion.
Uncontrolled viral, bacterial, fungal, or protozoal infection at the time of study enrollment.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 3 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new way to help prevent serious side effects from stem cell transplants in children with non-malignant blood diseases.

Who is the study for?
This trial is for children and adolescents with serious non-malignant hematologic diseases who are undergoing stem cell transplantation from an unrelated donor. Participants must be aged between 0-20.99 years, depending on their condition, and have a matched or nearly matched donor. They cannot join if they have certain organ dysfunctions, HIV, uncontrolled infections, recent clinical stroke, or are pregnant.
What is being tested?
The study tests whether extended use of abatacept can prevent acute and chronic graft-versus-host disease in pediatric patients receiving stem cell transplants without affecting engraftment or immune reconstitution. All participants will receive eight doses of abatacept along with standard medications tacrolimus and mycophenolate mofetil.
What are the potential side effects?
While the specific side effects of abatacept in this context aren't detailed here, generally it may cause increased risk of infections due to immune system suppression, possible allergic reactions during infusion, headache, nausea, and possibly high blood pressure.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have had a stroke confirmed by brain scans showing damage.
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I have a rare blood disorder or immune system condition.
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I have a diagnosed artery condition in my brain shown by specific tests.
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I have a genetic condition that has led to severe aplastic anemia.
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I have a blood disorder that has led to 3 hospital stays due to infection in the last 3 years.
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I've had 3 or more painful episodes a year needing strong pain relief before starting hydroxyurea.
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I have a donor match for a bone marrow or stem cell transplant.
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I have had 3 or more acute chest syndrome events requiring blood transfusions.
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I need monthly blood transfusions due to a hereditary or congenital red blood cell disorder.
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I am between 3-20 years old with sickle cell disease or under 21 years old with another disease.
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I have a blood platelet disorder and have had over 10 transfusions in the last 3 years.
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My sickle cell disease is severe, meeting at least one specific criterion.
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I am not pregnant and will use birth control during the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My lung function is significantly reduced, below 40% of what is expected.
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I do not have any ongoing serious infections.
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My heart's pumping ability is significantly reduced.
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I have a donor for my treatment, but tests show I have antibodies against the donor's HLA.
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I am HIV positive.
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I need considerable assistance and medical care.
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I understand the nature and risks of the HSCT process.
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My kidney function is reduced with a GFR less than 60.
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I have severe liver scarring.
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I have a family member who is a perfect match for my transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Event-free survival
Secondary study objectives
Acute GVHD
CMV Invasive Disease
Chronic GVHD
+12 more

Side effects data

From 2023 Phase 3 trial • 657 Patients • NCT03086343
24%
UPPER RESPIRATORY TRACT INFECTION
24%
HYPERTENSION
18%
BRONCHITIS
12%
OROPHARYNGEAL PAIN
12%
RASH
12%
ACUTE RESPIRATORY FAILURE
12%
WEIGHT INCREASED
12%
URINARY TRACT INFECTION
12%
INFLUENZA
12%
FALL
12%
ARTHRALGIA
12%
SINUSITIS
12%
MUSCLE SPASMS
12%
CHOLELITHIASIS
12%
NASOPHARYNGITIS
12%
LIVER FUNCTION TEST INCREASED
6%
VULVOVAGINAL MYCOTIC INFECTION
6%
LIGAMENT RUPTURE
6%
PATELLA FRACTURE
6%
ASTHMA
6%
COSTOCHONDRITIS
6%
DEHYDRATION
6%
NASAL CONGESTION
6%
PULMONARY EMBOLISM
6%
HYPONATRAEMIA
6%
CONSTIPATION
6%
RHINORRHOEA
6%
LEUKOCYTOSIS
6%
BLOOD PRESSURE INCREASED
6%
COUGH
6%
BONE CONTUSION
6%
HIP FRACTURE
6%
VOMITING
6%
PERIPHERAL SWELLING
6%
STAPHYLOCOCCAL INFECTION
6%
ORAL CANDIDIASIS
6%
PNEUMONIA
6%
FEELING HOT
6%
HEADACHE
6%
HAEMOGLOBIN DECREASED
6%
PHOTODERMATOSIS
6%
RHEUMATOID ARTHRITIS
6%
PARAESTHESIA
6%
PNEUMONIA BACTERIAL
6%
STOMATITIS
6%
SKIN LACERATION
6%
HYPOKALAEMIA
6%
GLAUCOMA
6%
FEMUR FRACTURE
6%
BACTERIAL SEPSIS
6%
CHEST PAIN
6%
FATIGUE
6%
ATRIOVENTRICULAR BLOCK FIRST DEGREE
6%
CANDIDA INFECTION
6%
TACHYCARDIA
6%
COVID-19
6%
CATARACT
6%
INFECTIOUS PLEURAL EFFUSION
6%
SJOGREN'S SYNDROME
6%
DIZZINESS
6%
DYSPHAGIA
6%
SWELLING
6%
SEBORRHOEIC KERATOSIS
6%
ACUTE KIDNEY INJURY
6%
CHRONIC OBSTRUCTIVE PULMONARY DISEASE
6%
HERPES ZOSTER
6%
OSTEOARTHRITIS
6%
NAUSEA
6%
NON-CARDIAC CHEST PAIN
6%
RHINITIS
6%
BLOOD CREATINE PHOSPHOKINASE INCREASED
6%
OSTEOPENIA
6%
INSOMNIA
6%
PRURITUS
6%
SUPRAVENTRICULAR TACHYCARDIA
6%
SCRATCH
6%
EYE HAEMATOMA
6%
ERYTHEMA
6%
ACTINIC KERATOSIS
6%
DERMATITIS ALLERGIC
6%
FLANK PAIN
6%
SCIATICA
6%
ANAEMIA
6%
BILIARY COLIC
6%
DERMATITIS CONTACT
6%
HEPATIC STEATOSIS
6%
DRUG HYPERSENSITIVITY
6%
HERPES SIMPLEX
6%
LOCALISED INFECTION
6%
PHARYNGITIS
6%
DIABETES MELLITUS
6%
VITAMIN D DEFICIENCY
6%
BACK PAIN
100%
80%
60%
40%
20%
0%
Study treatment Arm
Period 2, 30 mg Cohort: Upadacitinib 30 mg QD/Upadacitinib 30 mg QD
Period 2, Primary Cohort: Upadacitinib 15 mg QD/Upadacitinib 15 mg QD
Period 2, Primary Cohort: Abatacept/Upadacitinib 15 mg QD
Period 2, 30 mg Cohort: Abatacept/Upadacitinib 30 mg QD/Upadacitinib 15 mg QD
Period 1, 30 mg Cohort: Upadacitinib 30 mg QD
Period 2, 30 mg Cohort: Abatacept/Upadacitinib 30 mg QD
Period 2, 30 mg Cohort: Upadacitinib 30 mg QD/Upadacitinib 30 mg QD/Upadacitinib 15 mg QD
Period 1, Primary and 30 mg Cohorts: Abatacept
Period 1, Primary Cohort: Upadacitinib 15 mg QD

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Participants Receiving AbataceptExperimental Treatment1 Intervention
Pediatric participants who are undergoing URD HSCT for serious NMHD will receive 8 doses of abatacept in addition to conventional GVHD prophylaxis.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Abatacept
2005
Completed Phase 4
~112250

Find a Location

Who is running the clinical trial?

Sickle Cell Transplant Advocacy & Research Alliance (STAR)UNKNOWN
Aflac Cancer and Blood Disorders CenterUNKNOWN
Emory UniversityLead Sponsor
1,697 Previous Clinical Trials
2,603,788 Total Patients Enrolled

Media Library

Abatacept (Costimulation Blockade) Clinical Trial Eligibility Overview. Trial Name: NCT03924401 — Phase 2
Graft-versus-Host Disease Research Study Groups: Participants Receiving Abatacept
Graft-versus-Host Disease Clinical Trial 2023: Abatacept Highlights & Side Effects. Trial Name: NCT03924401 — Phase 2
Abatacept (Costimulation Blockade) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03924401 — Phase 2
~5 spots leftby Jan 2026
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