|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
12 December 2020 |
|
Main ID: |
NCT01182675 |
|
Date of registration:
|
09/08/2010 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim
|
|
Scientific title:
|
Hematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & Filgrastim |
|
Date of first enrolment:
|
August 2010 |
|
Target sample size:
|
7 |
|
Recruitment status: |
Terminated |
|
URL:
|
https://clinicaltrials.gov/show/NCT01182675 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Christopher C Dvorak, M.D. |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University of California, San Francisco |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Patients with classic SCID phenotype (<400 CD3/ul or maternally engrafted and <10% of
normal PHA lymphoproliferative response). Genotypic identification is preferable, but
not required.
- Patients must have an acceptable stem cell donor (HLA matched relative, 9 or 10/10
HLA-matched unrelated, or haplocompatible relative).
Exclusion Criteria:
- Patients with "leaky" SCID syndromes, Omenn's Syndrome, reticular dysgenesis, ADA
deficiency
- Lansky score <60%
- Patient with expected survival <4 weeks (including disseminated CMV infection
involving lungs and/or CNS)
Age minimum:
N/A
Age maximum:
3 Years
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Severe Combined Immunodeficiency
|
|
Intervention(s)
|
|
Drug: Transplant Conditioning with Mobilization Only
|
|
Drug: Transplant Conditioning with Mobilization and Alemtuzumab
|
|
Primary Outcome(s)
|
|
Engraftment of Donor B-cells in Blood by STR Testing
[Time Frame: 1 Year]
|
|
Secondary Outcome(s)
|
|
Incidence of Acute GVHD
[Time Frame: 100 Days]
|
|
Incidence of Chronic GVHD
[Time Frame: 2 Years]
|
|
Number of Patients With Engraftment of Donor Stem Cells in Bone Marrow by STR Testing
[Time Frame: 1 Year]
|
|
Percentage of Patients Who Become Independent From Regular IVIG Infusion
[Time Frame: 2 Years]
|
|
Number of Patients Who Achieve Engraftment of Donor T-cells in Blood by STR Testing
[Time Frame: 1 Year]
|
|
Secondary ID(s)
|
|
UCSF10-00701
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|