Seattle Children’s Expanded Access Policy for SCRI-CARB7H3(s)

At Seattle Children’s, we are dedicated to developing therapies that will transform the lives of patients with serious debilitating and/or life-threatening diseases. Our goal is to provide these treatments as quickly as possible to patients, once rigorous testing demonstrates our therapies are safe and effective for patients, and regulatory authorities, like the U.S. Food and Drug Administration (FDA), have approved these therapies.

Evaluation of investigational treatments in clinical trials plays a major part in achieving this important goal, and we apply strong scientific, legal and ethical principles to the evaluation of our investigational gene therapies. Patients, families, caregivers, healthcare professionals and patient advocacy organizations may find information about our clinical studies on Clinicaltrials.gov.

Under an Expanded Access Program (also referred to as “compassionate use” or “early access”) the FDA may allow patients and their healthcare providers to access investigational treatments outside of clinical trials.

It is important to understand that an Expanded Access Program involves an investigational treatment whose safety and effectiveness has not been established for the patient’s specific condition. As such, these investigational treatments have not been approved as safe and effective by the FDA. Expanded Access Programs are regulated by the U.S. FDA under Title 21 CFR Part 312 Subpart I, but do not take the place of a rigorously conducted clinical trial for an investigational product.

Under the 21st Century Cures Act, section 561A(f)(2) of the Federal Food, Drug, and Cosmetic Act, Seattle Children’s is required to make publicly available its expanded access policy for evaluating and responding to any expanded access requests for SCRI-CARB7H3(s) for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG) in children and young adults.

Generally, Seattle Children’s does not provide investigational cell therapy drugs to patients who are not enrolled in our clinical trials until there is sufficient data on the safety and efficacy of our products to allow patients and physicians to assess the risks and benefits of using these investigational drugs outside of a clinical trial context.

Due to the unique requirements of personalized cell manufacturing and the high-level of monitoring required for patients treated with this product, it is not feasible for Seattle Children’s to provide an expanded access program to this investigational therapy to patients at their local institution. Further information about this trial can be found at Clinicaltrials.gov/study/NCT04185038.

Seattle Children’s believes that the most appropriate way to access this investigational therapy is through participation in this trial: Phase 1 Study of B7-H3-Specific CAR T Cell Locoregional Immunotherapy for DIPG, DMG, or B7-H3-Positive Recurrent or Refractory Pediatric Central Nervous System Tumors-STUDY00001659. We encourage patients and their healthcare providers to discuss if participation in this trial, or in one of our other ongoing clinical trials, is appropriate.

In the future, this Expanded Access Policy may be reassessed as SCRI-CARB7H3 advances further in clinical trials and more data to support the safety and effectiveness become available.

Please contact us if you have any questions about our clinical trial programs or individual clinical trials: How to Participate.