Hematopoietic Stem Cell Processing Laboratory, CHLA

This site contains information, procedures, forms and contact information for marrow and Peripheral Blood Stem Cell purging and/or cryopreservation procedures carried out by this laboratory.

Expanded Access Purging Protocol

The Expanded Access Protocol for Purging of Peripheral Blood Stem Cells or Bone Marrow from Patients with High-Risk Neuroblastoma Prior to Autologous Transplantation was permanently closed effective June 13, 2008. Dr. Reynolds notified the FDA on June 13, 2008 that the Investigational Device Exemption (IDE) BB-IDE-2259 for purging was withdrawn because the COG A3973 study did not demonstrate a benefit for purging peripheral blood stem cells. Therefore, purging of bone marrow or peripheral blood stem cells under this IDE is no longer available.

There are patients that may still have purged stem cells infused, if medically indicated, but to use those stem cells a single patient IND will need to be filed with the FDA.

We recommend that you either develop an expanded access protocol for submission under a new IND application to be held by yourself or an appropriate alternate designate, or, that if the individual occasion arises to utilize the product for the intended patient, the responsible investigator at the involved institution submit to the FDA a request for a single patient IND to accommodate this action.

http://www.fda.gov/cder/cancer/singleIND.htm

Neuroblastoma is a malignant childhood tumor that often spreads to bone marrow. Neuroblastoma cells can be found in bone marrow or peripheral blood stem cells collected for re-infusion into patients (autologous transplantation) after myeloablative therapy. Purging of stem cells collected to support autologous transplant for high-risk neuroblastoma can be carried out by using monoclonal antibodies to attach small magnetic beads to neuroblastoma cells, allowing them to be separated from the stem cells using high-energy magnets.

The CHLA laboratory has carried out such tumor cells purging for > 10 years under an FDA Investigational Device Exception (IDE), supporting a number of different clinical trials, including the CCG 3891 trial that established myeloablative therapy followed by 13-cis-retinoic acid as standard of care for high-risk neuroblastoma (New Eng J Med 341:1165-1173, 1999). The most recent clinical trial using neuroblastoma purging from stem cells was the Children’s Oncology Group (COG) A3973 study which compared purged to non-purged peripheral blood stem cell transplantation in a randomized phase III clinical trial.

The Children's Oncology Group A3973 protocol has completed accrual and is closed. It will be years before the analysis of this protocol is completed. As the purging procedure has been used safely in over 1000 children with neuroblastoma and has been documented to remove neuroblastoma cells from bone marrow and PBSC, we have developed an "expanded access" protocol to allow patients to have PBSC or bone marrow purged with the purging device, which remains an investigational device.

The expanded access purging protocol has been approved by the FDA under BB IDE 2259. The expanded access protocol is approved by the CHLA Human Subjects Protection Committee (IRB). That protocol and the CHLA-IRB-approved consent and assent are provided as links below. The protocol must be approved by the IRB of any institution wishing to submit stem cells to CHLA for purging. While we recognize that individual IRB's will want variations on the consent, please stay as close to the example provided as possible. Critical is to include the portion about California Choice of Law as it is required by the CHLA administration.

To: All PI's at Participating Centers

From: Dr. C.P Reynolds / Dr. Judith Villablanca

DATE:12-03-2007

Please note that the compassionate exception purging protocol is temporarily suspended to patient accrual pending CHLA IRB review of the annual report.

A notice on this website will be posted as soon as IRB approval is received, and patient accrual will resume. If you have any questions regarding pending patients, please contact Dr. Villablanca at 323-361-4565 or 323-361-5654.

Annual Public Report Dec 2007

Opening the Study at your Institution - How To's

Current Documents

Shipping Forms

Dataforms

Archived Documents

The MOU between CHLA and institutions previously submitting cells to CHLA for purging will remain in effect and no further action is required.

In addition to obtaining local IRB approval for the Expanded Access purging protocol, the local PI for that protocol needs to submit an FDA 1572 form and CV, together with a copy of the letter of approval from their local IRB to: