Stealth BioTherapeutics is committed to bringing therapies to patients for the treatment of rare and life-threatening diseases involving mitochondrial dysfunction. We conduct clinical trials to evaluate the safety and effectiveness of our investigational drugs in order to obtain regulatory approval to provide these investigational therapies to the broadest group of patients. Regulatory authorities will permit us to provide patients with broad access to these investigational therapies only after we have demonstrated that they are safe and effective.
Enrolling in a clinical trial is the customary way for patients to access Stealth’s investigational drugs. However, in some rare and very specific circumstances when patients are not eligible and/or it is not feasible to enroll them into a clinical trial, physicians caring for patients with a serious, life-threatening or imminently debilitating condition may seek special access to investigational therapies. Only in these rare and very specific circumstances patients may be able to receive investigational therapies outside the clinical trial process through an expanded access program (also known as compassionate use, named patient, or preapproval access). Additionally, under certain circumstances emergency access may be considered. For emergency consideration the licensed treating physician should contact Stealth at firstname.lastname@example.org or call 617.600.8888 and specify the emergency access need. Details of Stealth’s Expanded Access protocol can be found at www.ClinicalTrials.gov with identifier NCT04689360. Stealth BioTherapeutics will consider Expanded Access requests to an investigational drug outside of a clinical trial on a case-by-case basis for patients with a serious, life-threatening, or imminently debilitating condition when there is no therapeutic alternative, provided the initial request meets the following criteria:
- The patient (or patient’s legal guardian) and a licensed physician are both willing to participate and complete regulatory requirements
- The patient’s physician determines that there is no comparable or satisfactory therapy available to treat the patient’s condition
- In the opinion of the treating physician, the probable risk to the patient from the investigational drug is not greater than the probable risk from the disease or condition
- There is sufficient evidence of the safety and effectiveness of the investigational therapy to support its use in the treatment of the patient’s condition
- Providing the investigational therapy will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval
- The patient meets inclusion criteria for participation based on their clinical status
- The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial
Procedure for Submitting a Request
For Patients / Caregivers / Patient Advocates
Enrolling in a clinical trial is the customary way for patients to access Stealth’s investigational drugs. To participate in a clinical study, you must meet certain eligibility criteria. You should discuss participation in a clinical trial with the physician who knows your medical history and status. If you are not eligible for a clinical trial and would like to request Expanded Access to a Stealth BioTherapeutics investigational therapy, the licensed physician overseeing your care must submit this request directly to Stealth BioTherapeutics.
For Healthcare Professionals
Patients who are not eligible and/or able to participate in a clinical trial of the investigational therapy may be able to gain access through the Stealth Expanded Access Program. For the patient to be considered, a qualified and licensed physician must submit the initial request for Expanded Access in writing directly to Stealth BioTherapeutics.
- Qualified physician submits request via email@example.com
- Stealth BioTherapeutics will acknowledge receipt of request and provide a Patient Information Intake Form for the provision of additional data including at least the following information:
- Demographics of the proposed Expanded Access patient
- Relevant medical history, including all therapies administered to date for the relevant indication
- Recent laboratory assessments including genetic confirmation of mitochondrial disease when appropriate
- Assessment of the patient’s prognosis
- Rationale for requesting Expanded Access to the investigational drug
- Complete Patient Information Intake Form is returned via provided e-mail
- Stealth BioTherapeutics will notify requesting physician of initial decision of patient eligibility based on the Patient Information Intake Form and any other supplemental information reviewed.
- If the decision is an approval, Stealth BioTherapeutics will provide requesting physician with additional Expanded Access Program details.
- There is no appeal process for a request that has been denied by Stealth BioTherapeutics.
- Access to the investigational therapy will generally continue as long as the patient continues to receive benefit as determined by the treating physician and will end should the investigational therapy become commercially available for the relevant indication in the patient’s country of residence. Stealth BioTherapeutics reserves the right to discontinue, at any time, access to an investigational therapy.