Cryo-Cell is committed to improving patient outcomes through cellular therapies derived from umbilical cord blood and cord tissue.
We understand that some patients may not qualify for investigational clinical trials (Investigational New Drug [IND]) and may lack alternative treatment options. In such cases, an Expanded Access Program (also known as compassionate use) becomes a potential pathway to allow patients with serious or life-threatening conditions to access investigational products when no satisfactory alternatives exist. Regulators and health authorities, like the U.S. Food and Drug Administration (FDA), may grant permission for Cryo-Cell to provide an investigational product to a treating physician for a patient in need. These requests are referred to as “expanded access”. Cryo-Cell hopes to to offer this expanded access program in 2025.
It’s crucial to recognize that these investigational products have not yet received regulatory approval by the FDA, which means their risks and benefits remain uncertain. Physicians and patients should carefully weigh all potential advantages and risks when considering treatment with an investigational product via an expanded access program.
Please note: Cryo-Cell is completing the establishment of a new infusion clinic and laboratory facility in Durham, NC which will be used for cord blood banking, manufacturing of cell therapies from cord blood and cord tissue, and treatment of patients enrolled in Cryo-Cell’s clinical trials and expanded access protocols. This facility is planned to open in 2025.
Cryo-Cell will consider providing a requesting physician and their patient with expanded access to an IND outside of an investigational clinical trial for the treatment of an individual patient at our facility when certain conditions are met. These conditions include the following:
- The patient must have a medical diagnosis of Cerebral Palsy (GMFCS Level I-IV).
- The patient must be ineligible for ongoing clinical trials and have no satisfactory alternative therapy available.
- The patient must meet the eligibility criteria specified in the therapeutic protocol for the expanded access program.
- The potential benefit of the investigational product for the patient should outweigh the potential risk, as evaluated by the patient’s healthcare provider and discussed thoroughly with the patient.
- The patient must be treated at the Cryo-Cell Clinical facility in Durham, NC.
- A cord blood unit meeting quality criterion must be available in the public inventory or have been stored privately by the patient’s family.
- A child must be 6 months of age or older to receive an infusion from a sibling or unrelated donor cord blood unit.
- Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access.
- The cost of the cord blood unit from a third party and the related services necessary for the infusion will be borne by the patient.
At Cryo-Cell, we are dedicated to ensuring a fair and equitable evaluation of all Expanded Access requests we receive. Each request is carefully reviewed on a case-by-case basis. In some instances, we may seek additional detailed information related to a specific expanded access program request to more thoroughly assess the request. Unfortunately, we cannot guarantee that all access requests will be granted, even if eligibility criteria are met.
Patients and healthcare providers seeking access to a Cryo-Cell investigational treatment should submit their requests via Infusion Clinic Inquiry Form, providing all information requested by Cryo-Cell for that expanded access program. Cryo-Cell will contact you to request additional information so that the patient’s eligibility can be determined. Once eligibility is determined, a treatment may be scheduled at our facility in Durham, NC. Please note that treatment availability is dependent on the Cryo-Cell clinic availability.
Cryo-Cell reserves the right to revise this policy at any time and may establish distinct expanded access policies for each investigational product currently in development. Additionally, Cryo-Cell may terminate expanded access to an investigational product at any time, or access may be discontinued by the FDA or a patient’s healthcare provider. For further details about expanded access in the United States, please refer to the: FDA Expanded Access information page.