Sickle Cell Disease Clinical Trial
Official title:
Hematopoietic Stem Cell Transplantation for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Doppler Ultrasound Using Reduced-Intensity Conditioning and T-Cell Depleted HSC From Partially Matched Family Donors
Verified date | February 2009 |
Source | St. Jude Children's Research Hospital |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Sickle cell disease is a life-long blood condition that can cause damage to the brain and
other organs of the body. Children may develop severe, debilitating clinical states, with
stroke or abnormal blood flow to the brain. Treatment generally includes chronic blood
transfusions which may cause iron overload, potentially leading to severe and sometimes
fatal complications.
Hematopoietic stem cell transplant using cells obtained from a sibling or an unrelated
volunteer donor who is a perfect HLA "match" (same tissue type) for the recipient has shown
to help, and possibly cure, sickle cell disease. Unfortunately, only about 10-20% of sickle
cell patients have a HLA matched sibling donor, and the likelihood of locating an
appropriate HLA matched unrelated donor through the various donor registries is limited.
Stem cells from partially HLA matched family members (also called haploidentical transplant)
is an option currently being explored for this patient population. This type of transplant
has been used and found to be successful in some patients, mostly those with cancers of the
blood. However, there can be significant complications with haploidentical transplant,
primarily infection, failure of the graft to grow (graft failure), and a disorder called
graft-versus-host disease. In addition, few patients with sickle cell disease have undergone
this procedure. Therefore, the risks and benefits of haploidentical transplants for patients
with sickle cell disorder are not as well established as those using an HLA identical
sibling or unrelated donor.
The primary objective of this study is to assess the safety of haploidentical stem cell
transplantation for children and adolescents with severe sickle cell disease and stroke or
abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy. The
treatment plan will be considered safe if there is not excessive toxicity. Toxicity for this
protocol is defined as graft failure/graft rejection, severe acute GVHD, or regimen related
death within 100 days after the last cellular product administered.
Of note, the protocol was closed to accrual in September 2007 as we had met the stopping
rules related to graft integrity (graft failure and graft rejection). Participants currently
enrolled continue to be followed per protocol.
Status | Completed |
Enrollment | 5 |
Est. completion date | January 2009 |
Est. primary completion date | December 2008 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 16 Years |
Eligibility |
Inclusion Criteria: - Hemoglobin SS or S-Beta Thalassemia Sickle Cell Disease. - Partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait) phenotype. - Stroke (persistent neurologic deficit lasting > 24 hours and present on MRI) or abnormal transcranial Doppler (TCD) ultrasonography requiring chronic transfusion therapy. A TCD is deemed abnormal when the velocity is greater than or equal to 200 cm/sec. Chronic transfusion therapy is defined as "packed red blood cell transfusions administered approximately every 3-5 weeks to decrease the percentage of sickle hemoglobin (Hemoglobin S) to prevent complications of sickle cell disease. This is used most commonly to treat/prevent stroke, acute chest syndrome, and/or pain crises. Exclusion criteria - Karnofsky or Lansky score < 60% - Acute hepatitis or evidence of moderate or severe portal fibrosis on biopsy. (Biopsy will be obtained if patient on chronic transfusion therapy > 6 months or ferritin > 1000 ng/ml) International normalized ratio (INR) less than 2 times normal. ALT and AST less than 3 times the upper limit of normal. - Severe renal impairment (as evidenced by GFR < 30% predicted normal) - Ejection fraction or shortening fraction below lower limit of normal for age. - Pregnancy - Lactating and pregnant females are excluded - Positive HLA crossmatch with donor. - No sickle cell chronic lung disease > Stage 2 |
Country | Name | City | State |
---|---|---|---|
United States | St. Jude Children's Research Hospital | Memphis | Tennessee |
Lead Sponsor | Collaborator |
---|---|
St. Jude Children's Research Hospital |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | To assess the safety of haploidentical stem cell transplantation for children and adolescents with severe sickle cell disease and stroke or abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy. | September 2007 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02227472 -
Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
|
||
Recruiting |
NCT06301893 -
Uganda Sickle Surveillance Study (US-3)
|
||
Recruiting |
NCT04398628 -
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
|
||
Completed |
NCT02522104 -
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
|
Phase 4 | |
Recruiting |
NCT04688411 -
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
|
N/A | |
Terminated |
NCT03615924 -
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
|
Phase 3 | |
Not yet recruiting |
NCT06300723 -
Clinical Study of BRL-101 in Severe SCD
|
N/A | |
Recruiting |
NCT03937817 -
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
|
||
Completed |
NCT04134299 -
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
|
N/A | |
Completed |
NCT04917783 -
Health Literacy - Neurocognitive Screening in Pediatric SCD
|
N/A | |
Completed |
NCT02580565 -
Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
|
||
Recruiting |
NCT04754711 -
Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition
|
N/A | |
Completed |
NCT04388241 -
Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD
|
N/A | |
Recruiting |
NCT05431088 -
A Phase 2/3 Study in Adult and Pediatric Participants With SCD
|
Phase 2/Phase 3 | |
Completed |
NCT01158794 -
Genes Influencing Iron Overload State
|
||
Recruiting |
NCT03027258 -
Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome
|
N/A | |
Withdrawn |
NCT02960503 -
Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease
|
Phase 1/Phase 2 | |
Completed |
NCT02565082 -
Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients
|
N/A | |
Completed |
NCT02567695 -
A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects
|
Phase 1 | |
Completed |
NCT02620488 -
A Brief Laboratory-Based Hypnosis Session for Pain in Sickle Cell Disease
|
N/A |