Severe Combined ImmunodeficiencyPhase 1NCT00152100
Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
A Phase 1 study testing Stem cell transplant, Filgrastim, Alemtuzumab and Miltenyi CliniMACS in people with Severe Combined Immunodeficiency, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages Up to 2 years
- Conditions the study is for
- Severe Combined Immunodeficiency
- What is being tested?
- Stem cell transplant, Filgrastim, Alemtuzumab and Miltenyi CliniMACS
- Type of treatment
- Medical device, Surgery or procedure
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2004to Aug 2007
- Phase
- Phase 1
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 Up to 2 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patient with confirmed severe combined immunodeficiency
- Two years of age or younger
- A suitable matched sibling donor is not available
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: 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.
- Conditions
- Severe Combined Immunodeficiency
- Being tested
- Stem cell transplant, Filgrastim, Alemtuzumab, Miltenyi CliniMACS
- Keywords
- Severe combined immunodeficiency, Transplantation, Haploidentical
- Medical subject headings
- Severe Combined Immunodeficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem cell transplantProcedure or surgery
Filgrastim, AlemtuzumabDrug
Miltenyi CliniMACSDevice
What is being measured
The primary outcome is the main question the study is designed to answer.
To investigate safety issues related to use of haploidentical highly purified CD133+ hematopoietic cells in patients with SCID
To study the effects (good and bad) of this procedure
To learn if this procedure will result in normal immune function in children with SCID
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2005 (estimated)
- Study startFeb 2004
- Primary completionAug 2007
- Study completionAug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Combined ImmunodeficiencyStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Investigators
- Kimberly Kasow, DO, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00152100
- Sponsor's protocol ID
- ALSCID
- Status last verified
- May 2009
- First submitted
- 7 Sep 2005
- Last updated
- 20 May 2009
- Results posted
- No
- Data monitoring committee
- No