Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00579137
• Date of registration: 19/12/2007
• Prospective Registration: No
• Primary sponsor: Baylor College of Medicine
• Public title: Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders MASCI
• Scientific title: CD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency Disorders
• Date of first enrolment: October 2007
• Target sample size: 3
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00579137
• Study type: Interventional
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Malcolm Brenner, MD
• Affiliation: Baylor College of Medicine

• Name: Robert Krance, MD
• Affiliation: Baylor College of Medicine

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with a diagnosis of: Severe combined immunodeficiency disease

This includes patients whose SCID is characterized by gene specific mutations as well as
patients with clinically severe combined immunodeficiency without a defined genetic cause
in which the diagnosis will be determined by a combination of clinical course with
lymphocyte quantification and function assays.

OR

Severe primary immunodeficiency disorder, including undefined T cell deficiency disorder,
Wiskott-Aldrich syndrome, and other severe immunodeficiencies for which satisfactory
conventional therapy does not exist.

- Availability of an HLA mismatched (up to one haplotype) family member or an HLA
matched or mismatched (up to one antigen) unrelated donor.

- Creatinine < 2.5 x normal for age.

- Life expectancy greater than 6 weeks

- Lansky/Karnofsky greater than or equal to 70%

Exclusion Criteria:

- Patients with an HLA matched related donor

- Patients with symptomatic cardiac disease, or evidence of significant cardiac disease
by echocardiogram (i.e., shortening fraction less than 25%)

- Patients with known allergy to rat serum products

- Patients with a severe infection that on evaluation by the Principal Investigator
precludes ablative chemotherapy or successful transplantation

- HIV positive

- Pregnant



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Undefined T Cell Deficiency Disorder

Severe Combined Immunodeficiency Disease

Severe Primary Immunodeficiency Disorder

Wiskott-Aldrick Syndrome

Intervention(s)

Biological: Anti-CD45

Biological: Campath -1H

Drug: Fludarabine

Procedure: Stem cell infusion

Primary Outcome(s)

Number of Patients With Donor Engraftment [Time Frame: 100 Days]

Secondary Outcome(s)

Patients Alive at 1 Year [Time Frame: 1 Year]

Number of Patients With Grade III to IV Acute GVHD [Time Frame: 100 days]

Number of Patients With Grade III or IV Toxicity [Time Frame: 100 days]

Secondary ID(s)

21123-MASCI

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Center for Cell and Gene Therapy, Baylor College of Medicine

Texas Children's Hospital

Results

• Results available: Yes
• Date Posted: 02/07/2013
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00579137