Transplantation with autologous CD34+ stem cells corrected with X1XCGD lentiviral vector after myeloreductive conditioning

Granulomatous Disease, Chronic, X-linked

The investigational product is patient-specific and corresponds to autologous CD34+ cells transduced ex vivo with the G1XCGD vector in their final suspension. The starting materials used for the production of the investigational product consist of the viral vector and the patient's CD34+ cells.
The G1XCGD vector is used to transduce autologous CD34+ cells ex vivo. These transduced cells are then frozen, and an aliquot tested and characterized for quality. If the cell product passes release criteria, it is thawed at bedside and infused into the patient after the patient has received myelo-ablative conditioning. The cell/product dose will consist of at least 3 x 10^6 cells per kg of body weight transduced ex vivo with 1 x 10^8 IG/mL of lentiviral vector to achieve > 0.3 integrated copies per cell.

Lentiviral G1XCGD Gene Therapy

University of California, Los Angeles (UCLA)

National Institutes of Health

Children's Hospital Boston

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Record clinical adverse events
Evaluate overall incidence of adverse events
Record clinically significant laboratory abnormalities
Monitor the incidence of serious adverse events

Measuring percentage of subjects who have ≥ 10% oxidase positive granulocytes by DHR flow cytometry

Results will be published in scientific literature once trial is completed and data analysis is done.

Donald B. Kohn, M.D.

Professor

University of California, Los Angeles

Boston Children’s Hospital

National Institute of Allergy and Infectious Diseases (NIAID)

Genethon

California Institute for Regenerative Medicine (CIRM)

Patients will receive one (1) four (4) week cycle of weekly IV PS-341 followed by a standard of care radical prostatectomy 24 to 72 hours later.

PS-341

Age of participants is reported as age at the time of study enrollment.

Age, Customized

Sex: Female, Male

Race/Ethnicity, Customized

Region of Enrollment

Pour wound healing is defined in the protocol as dehiscence of fascia during the first postoperative week. Excessive bleeding is defined in the protocol as greater than 2 units of blood required during the first 24 hours after surgery.

The Purpose of the Present Study is to Assess the Safety of PS-341 as a Pretreatment in Patients Who Are to Undergo a Radical Prostatectomy. Poor Wound Healing and Excessive Bleeding, With Historical Rates of <1% and 10% Respectively Will be Measured.

Overall Study

Dr. Teresa Hayes - Associate Professor

Prostate Neoplasms

PS-341 is a dipeptidyl boronic acid inhibitor with high specificity for the proteasome developed by Millennium Pharmaceuticals Inc. to treat human malignancies. It is the first member of this class of anti-tumor agents to come to human trials. Patients will receive PS-341 (1.6mg/m2/dose) by intravenous push weekly for 4 consecutive weeks followed by a 24-72 hour rest. This schedule consists of one treatment cycle. Upon the completion of 4 weeks of PS-341 followed by a 24-72 hour rest period, radical prostatectomy will be performed.

PS-341 (bortezomib)

Scott Department of Urology Clinical Trials

Scott Department of Urology, Baylor College of Medicine

PS-341 Followed by Removal of Prostate for Those With Prostate Cancer

H-11047: A Study of PS-341 Followed by Radical Prostatectomy for Patients With Adenocarcinoma of the Prostate (Spore #: 11-01-30-13)

Teresa Hayes

Associate Professor

Baylor College of Medicine

Millennium Pharmaceuticals, Inc.

Acute Myelogenous Leukaemia (AML)

Myelodysplastic Syndrome (MDS)

5 azacytidine - VALPROIC acid- Retinoic acid

Chu Saint Louis

5-azacytidine Valproic Acid and ATRA in AML and High Risk MDS

Multi Centers, Open-trial Phase II Study Evaluating 5-azacytidine (Vidaza®) + Valproic Acid (Depakine ®) Before Administration of Retinoic Acid (Vesanoid®) in Patients With Acute Myelogenous Leukemia and High Risk Myelodysplasia.

myelodysplastic syndromes/protease

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

PS-341 Followed by Removal of Prostate for Those With Prostate Cancer

5-azacytidine Valproic Acid and ATRA in AML and High Risk MDS

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