Expanded Access Policy

Expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. It is important to note that pafolacianine sodium injection is considered an investigational drug to be used adjunctly during ovarian or lung cancer surgery to aid in the detection of cancerous lesions.

It is critical that the patient and his/her licensed physician consider all possible risks when pursuing use of an investigational product as they have not yet been approved by the FDA nor has the FDA found these products to be safe and effective for their specific use. Furthermore, the investigational product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

The FDA emphasizes that whenever possible, an investigational medical product should be used as part of a clinical trial. However, if patient enrollment is not possible, expanded access offers a possible route for gaining access to an investigational medical product.

On Target has developed an expanded use policy to help provide the investigational product to patients who meet the criteria for such use.

To access On Target Laboratories’ Expanded Access program, patients should contact the company representatives (Timothy Biro, COO or Kimberly Fabrizio, VP of Regulatory Affairs and Quality Assurance) directly via email at expandedaccess@ontargetlabs.com or via phone at 765-588-4547 . The patient seeking expanded use will be required to travel to an approved clinical site, where the investigator has completed the necessary training to administer the product and has access to the required near-infrared camera device.

This would include any clinical investigators who have been approved to participate and have enrolled subjects in either Phase 2 or Phase 3 clinical trials with the product. The patient may qualify to be enrolled in an open study, if available. On Target will not include this subject in the randomization plan and their data will not be included in the efficacy analysis of the open study, however. All open clinical trials can be found on clinicaltrials.gov.

In the event enrollment in an open trial is not possible, an Expanded Access Submission would be required to be submitted under the open IND 118215 as a Protocol Amendment. On Target Laboratories will work with the selected clinical investigator to generate the submission, which will include the required information defined in CFR 312.305, including the rationale for the intended use of the drug, criteria for the patient selection, and the benefit risk of utilizing the investigational product and device.

The lung cancer patient selection criteria would match the inclusion and exclusion criteria defined in the Lung Cancer Phase 3 trial as noted below:

Inclusion Criteria:
To be considered eligible to participate in this study, a patient must meet all the inclusion criteria listed below:

1. Male and Female patients 18 years of age and older

2. Have a primary diagnosis, or a high clinical suspicion, of cancer in the lung warranting surgery based on CT/PET or other imaging

3. Are scheduled to undergo surgical thoracoscopy for diagnostic wedge resection followed by anatomic lung resection

4. Female patients of childbearing potential or less than 2 years postmenopausal agree to use an acceptable form of contraception from the time of signing informed consent until 30 days after study completion

5. Ability to understand the requirements of the study, provide written informed consent and authorization of use and disclosure of protected health information, and agree to abide by the study restrictions and to return for the required assessments

Exclusion Criteria:
Patients will be excluded if they meet any of the exclusion criteria listed below:

1. Previous exposure to pafolacianine sodium injection (OTL38)

2. Any medical condition that in the opinion of the investigators could potentially jeopardize the safety of the patient

3. History of anaphylactic reactions to folate, including synthetic folic acid (pteroylmonoglutamic acid) and contrast agents containing indocyanine green for near infrared imaging. Patients with a medical history of ‘idiopathic anaphylaxis’ will require evaluation.

4. History of allergy to any of the components of pafolacianine sodium injection (OTL38), including folic acid

5. A positive serum pregnancy test at Screening or a positive urine pregnancy test on the day of surgery or day of admission for female subjects of childbearing potential

6. Clinically significant abnormalities on electrocardiogram (ECG) at screening

7. Presence of any psychological, familial, sociological condition or geographical challenges potentially hampering compliance with the study protocol and follow-up schedule

8. Impaired renal function defined as eGFR< 50 mL/min/1.73m2

9. Impaired liver function defined as values > 3x the upper limit of normal (ULN) for alanine aminotransferase (ALT) or aspartate aminotransferase (AST), alkalinephosphatase (ALP), or >2x ULN for total bilirubin excluding those patients with Gilbert’s syndrome.

10. Received an investigational agent in another investigational drug or vaccine trial within 30 days prior to the administration of study drug

11. Known sensitivity to fluorescent light

Upon receipt of an expanded use access requests, On Target Laboratories will review the request and provide acknowledgement to the patient or requesting physician within 3-5 business. On Target will discuss the benefit risks with the treating physician and patient prior granting access and the company will make every effort possible to grant the access request.

Inclusion Criteria:
The ovarian patient selection criteria would match the inclusion and exclusion criteria defined in the Ovarian Cancer Phase 3 trial as noted below:

1. Female patients 18 years of age and older

2. Have a primary diagnosis, or at high clinical suspicion, of primary ovarian cancer(of epithelial type), planned for primary surgical cytoreduction, interval debulking, or have recurrent ovarian cancer, and:

    • Who are scheduled to undergo laparotomy for the debulking surgery OR
    • Who are scheduled to undergo laparoscopy and pre-authorized to undergo laparotomy for the debulking surgery if cancer is detected on the laparoscopy

3. A negative serum pregnancy test at Screening followed by a negative urine pregnancy test on the day of surgery or day of admission for female patients of childbearing potential

4. Female patients of childbearing potential, or less than 2 years postmenopausal, agree to use an acceptable form of contraception from the time of signing informed consent until 30 days after study completion

5. Ability to understand the requirements of the study, provide written informed consent or participation in the study and authorization of use and disclosure of protected health information, and agree to abide by the study restrictions and to return for the required assessments

Exclusion Criteria:
Patients will be excluded if they meet any of the exclusion criteria listed below:

1. Previous exposure to pafolacianine sodium injection (OTL38)

2. Known FR-negative ovarian cancer

3. Planned surgical debulking via laparoscopy or robotic surgery, with no intent of laparotomy

4. Patients with known ovarian cancer miliary disease determined pre-operatively to be inoperable

5. Any medical condition that in the opinion of the investigators could potentially jeopardize the safety of the patient

6. History of anaphylactic reactions

7. History of allergy to any of the components of pafolacianine sodium injection (OTL38), including folic acid

8. Pregnancy or positive pregnancy test

9. Clinically significant abnormalities on the electrocardiogram (ECG)

10. Presence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule

11. Impaired renal function defined as eGFR< 50 mL/min/1.73m2

12. Impaired liver function defined as values > 3x the upper limit of normal (ULN) for alanine aminotransferase(ALT),aspartate aminotransferase (AST), alkaline phosphatase(ALP),or total bilirubin.

13. Known Stage IV ovarian cancer with brain metastases

14. Received an investigational agent in another clinical trial within 30 days prior to surgery

15. Known sensitivity to fluorescent light

 

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