Trials & Expanded Access
Clinical Trials
Miltenyi Biomedicine is investigating multiple disease areas across our investigational cell therapy programs and next-generation platform technologies, including selected blood cancers, solid tumors, and autoimmune diseases.
Information on Miltenyi Biomedicine–sponsored clinical studies (including study status, eligibility criteria, and participating sites) is available in public clinical trial registries, including ClinicalTrials.gov and, where applicable, other national registries.
View our clinical trialsExpanded Access
At Miltenyi Biomedicine, our purpose is to advance effective cell therapies for patients with serious and difficult-to-treat diseases. Our primary focus is the development of investigational therapies through rigorous clinical research. We understand that individuals and healthcare professionals may seek access to our investigational therapies outside of our clinical development programs and prior to regulatory approval.
When patients are unable to participate in a clinical trial and have limited treatment options, we may consider requests for Expanded Access. This Expanded Access Policy outlines our approach to such requests.
Our approach to Expanded Access reflects our commitment to putting patients first, making evidence-driven decisions, and maintaining the highest standards of scientific and regulatory integrity. Requests are evaluated on a case-by-case basis, considering patient safety, available clinical and scientific data, regulatory requirements, and the feasibility of providing investigational therapies without compromising ongoing development programs. Treatment centers will need to have the appropriate clinical expertise and infrastructure to administer investigational cellular therapies.
Through this approach, we seek to responsibly address individual patient needs while continuing to generate the evidence required to bring innovative cell therapies to a broader patient population.
Investigational medicines
Our investigational CAR-T therapies are currently undergoing clinical evaluation and have not yet received marketing approval by regulatory authorities such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Thus, as investigational products, their safety, efficacy, and appropriate use have not yet been established.
Information about our clinical trials, together with links to the relevant studies listed on ClinicalTrials.gov can be found here - Research Pipeline
Expanded Access consideration
We may consider providing early access to an investigational therapy outside of a clinical trial when all of the following criteria are met:
- The patient has a serious or life-threatening condition with no satisfactory alternative treatment.
- The request is supported by a CAR-T certified treatment center.
- The patient is not eligible to participate in a clinical trial.
- The requested investigational therapy is part of an active clinical development program, and Expanded Access will be not compromise ongoing clinical studies or regulatory submissions.
- Sufficient preliminary efficacy and safety evidence is available to assess the benefit-risk profile for the requested condition/patient.
- The Expanded Access request complies with all applicable laws and regulations in the patient’s country.
Geographic scope
Expanded access is considered on a country-by-country basis and is currently limited, subject to local regulations. For example:
- United States: Requests may be submitted under the FDA’s Expanded Access pathway. At this time, this program is limited to Individual Patient Expanded Access INDs (also known as single patient INDs). To learn more about FDA expanded access programs, please visit: FDA Expanded Access
- European Union: Access may be available via national Compassionate Use Programs.
We do not guarantee availability in all countries and regions, and we can only offer access at CAR-T certified treatment centers. We are actively working to expand access to additional regions globally.
How to request access
Only Healthcare professionals may submit requests on behalf of patients by contacting us via e-mail at [email protected]. Please include all relevant clinical information and a justification for the request. We aim to acknowledge receipt and will review and respond as promptly as possible.
Important disclaimers
- Access to investigational therapies is not guaranteed and subject to internal review.
- Clinical trials are generally the preferred way to access investigational medicines when a suitable trial is available.
- Our CAR-T therapies are investigational and have not been approved for commercial use.
- Treatment can only be delivered at CAR-T certified treatment centers.
- Investigational therapies under Expanded Access are generally provided without charge. In limited circumstances, charges may be permitted in accordance with applicable local law and regulatory requirements. Patients may still be responsible for standard-of-care medical services and other healthcare costs, which are not covered under Expanded Access.
- This policy is not intended to promote or advertise any product.