- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00152100
Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
Treatment for severe combined immunodeficiency (SCID) is a medical emergency. A stem cell transplant (immature blood cells that can make other blood cells) from a (MSD) matched sibling donor (brother or sister who is a "match" for your child's immune (HLA) type), usually results in complete correction of immune function. However, most patients lack a matched sibling donor, requiring the use of an alternate donor source.
Transplantation of cells from haploidentical family donors (typically parents) has resulted in immune system correction in the majority of SCID individuals. However, only 65-80% of patients survive greater than one year after this procedure. Failure results from life-threatening infections, graft versus host disease (GvHD) or post-transplant treatment-related effects. Also, for patients that survive beyond one year, B-cell (type of blood cell that fights infection) and natural killer cell function (cell that attacks infections and cancer cells) frequently fail to work, resulting in the need for long-term treatment with intravenous gamma-globulin (IVIg).
In this study, in an effort to restore the overall cell function in patients with SCID, researchers will use a highly purified CD133+ hematopoietic cell graft (stem cell transplant without many mature donor white cells, called T-cells) obtained via use of the Miltenyi CliniMACS device, a device not FDA approved.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
Study Locations
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Tennessee
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Memphis, Tennessee, United States, 38105
- St. Jude Children's Research Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patient with confirmed severe combined immunodeficiency
- Two years of age or younger
- A suitable matched sibling donor is not available
Exclusion Criteria:
- An available matched sibling donor or a confirmed matched unrelated donor
- Patients with DiGeorge syndrome, Zap70, MHC Class II deficiency, or cartilage-hair hypoplasia
- Patients with a Lansky performance score of less than 10, evidence of HIV or a congenital rubella infection or a documented neoplasm
- Patients in whom it is not possible to perform a peripheral blood cell harvest on a haploidentical family member
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
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To investigate safety issues related to use of haploidentical highly purified CD133+ hematopoietic cells in patients with SCID
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To study the effects (good and bad) of this procedure
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To learn if this procedure will result in normal immune function in children with SCID
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Collaborators and Investigators
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ALSCID
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Severe Combined Immunodeficiency
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Great Ormond Street Hospital for Children NHS Foundation...UnknownX-linked Severe Combined ImmunodeficiencyUnited Kingdom
-
National Institute of Allergy and Infectious Diseases...RecruitingX-linked Severe Combined Immunodeficiency (XSCID)United States
-
St. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...SuspendedSevere Combined Immunodeficiency Disease, X-linkedUnited States
-
Assistance Publique - Hôpitaux de ParisCompletedX-linked Severe Combined ImmunodeficiencyFrance
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Shenzhen Geno-Immune Medical InstituteUnknownAdenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)China
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David WilliamsActive, not recruitingGene Therapy | Severe Combined Immunodeficiency, X LinkedUnited States, United Kingdom
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National Institute of Allergy and Infectious Diseases...TerminatedGrowth Failure | X-linked Severe Combined Immunodeficiency (XSCID) | Growth Hormone ResistenceUnited States
-
Great Ormond Street Hospital for Children NHS Foundation...RecruitingSevere Combined Immunodeficiency, X-LinkedUnited Kingdom
-
National Institute of Allergy and Infectious Diseases...UnknownX-Linked Combined Immunodeficiency DiseasesUnited States
-
National Institute of Allergy and Infectious Diseases...CompletedSevere Combined ImmunodeficiencyUnited States
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Royal Marsden NHS Foundation TrustCompleted
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Royal Marsden NHS Foundation TrustCompleted
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