EBRP spoke with Phoenix Tissue Repair’s Chief Medical Officer, Dr. Theresa Podrebarac, about their Phase 1/2 Trial of PTR-01, a protein replacement therapy for patients with RDEB. Read on to learn more about the trial, what’s required of patients, and how to enroll.
Q: What is PTR-01?
A: PTR-01 is recombinant Collagen VII, the protein that patients with RDEB are lacking. Living cells produce the protein in the laboratory, which is then purified and given to patients. This is a protein replacement therapy.
Q: How is PTR-01 delivered to patients?
A: The protein is delivered intravenously, so it is a systemic treatment. The infusion takes about an hour.
Q: How many clinical visits are required?
A: There are 9 visits to complete the study, but in many cases only 8 visits are required. There is 1 screening visit, 6 infusions and 2 follow ups. The screening visit may be combined with the first infusion if the patient is deemed eligible to begin the trial immediately. The whole process takes 127 days.
Q: Does every patient receive the treatment?
A: Yes. The study follows a crossover design. This means everyone will receive both Collagen VII and saline. Patients just aren’t aware of the order in which they receive the treatment or the saline. This helps us balance to see the true effect of the therapy.
Q: What must patients undergo during the trial?
A: The patient will undergo the 6 infusions, as well as blood draws and skin biopsies. During 6 of the visits, there are 2 biopsies done which are sent to 2 separate labs. One biopsy will test whether the Collagen VII has localized to the skin and the second will test if anchoring fibrils have formed, indicating that it’s functional. There are also questionnaires that take about 15-20 minutes with questions on quality of life, pain and itch, and daily challenges the patient faces. Lastly, photographs of up to 5 wounds will be taken to measure changes in wound size.
Q: How can patients enroll?
A: Interested patients should speak to their physician and see if the physician could be in touch with a trial center. This makes it easier to share records for a smoother process. Patients can also directly reach out to the study centers.
Q: Where are the study sites located?
A: There are 3 centers in the USA: Stanford University with Dr. Jean Tang, Children’s Hospital Colorado with Dr. Anna Bruckner, and Thomas Jefferson University with Dr. Dasgeb. Contact information is included below:
You can also view trial information on ClinicalTrials.gov and PhoenixTissueRepair.com.
Q: Are travel costs covered for patients?
A: All travel costs are covered for the patient and caregivers including flights, car, meals, mileage and gas fees, and hotel accommodations, as some visits require overnight stays for assessments. Everything can be booked through our white-glove concierge service, Greenphire, so patients don’t have to pay out of pocket and await reimbursement. Patients can also receive a prepaid card to assist with travel expenses.
Q: Will patients be able to receive the therapy after the trial is over?
A: Yes, we are providing Collagen VII with an open-label extension protocol.
Q: What is PTR-01?
A: PTR-01 is recombinant Collagen VII, the protein that patients with RDEB are lacking. Living cells produce the protein in the laboratory, which is then purified and given to patients. This is a protein replacement therapy.
Q: How is PTR-01 delivered to patients?
A: The protein is delivered intravenously, so it is a systemic treatment. The infusion takes about an hour.
Q: How many clinical visits are required?
A: There are 9 visits to complete the study, but in many cases only 8 visits are required. There is 1 screening visit, 6 infusions and 2 follow ups. The screening visit may be combined with the first infusion if the patient is deemed eligible to begin the trial immediately. The whole process takes 127 days.
Q: Does every patient receive the treatment?
A: Yes. The study follows a crossover design. This means everyone will receive both Collagen VII and saline. Patients just aren’t aware of the order in which they receive the treatment or the saline. This helps us balance to see the true effect of the therapy.
Q: What must patients undergo during the trial?
A: The patient will undergo the 6 infusions, as well as blood draws and skin biopsies. During 6 of the visits, there are 2 biopsies done which are sent to 2 separate labs. One biopsy will test whether the Collagen VII has localized to the skin and the second will test if anchoring fibrils have formed, indicating that it’s functional. There are also questionnaires that take about 15-20 minutes with questions on quality of life, pain and itch, and daily challenges the patient faces. Lastly, photographs of up to 5 wounds will be taken to measure changes in wound size.
Q: How can patients enroll?
A: Interested patients should speak to their physician and see if the physician could be in touch with a trial center. This makes it easier to share records for a smoother process. Patients can also directly reach out to the study centers.
Q: Where are the study sites located?
A: There are 3 centers in the USA: Stanford University with Dr. Jean Tang, Children’s Hospital Colorado with Dr. Anna Bruckner, and Thomas Jefferson University with Dr. Dasgeb. Contact information is included below:
- Stanford University - Redwood City, CA
- Contact: Melissa Barriga, 650-850-1674, [email protected]
- Principal Investigator: Jean Tang, MD, PhD
- Sub-Investigator: Peter Marinkovich, MD
- Children's Hospital Colorado - Aurora, CO
- Contact: Kathleen Peoples, 720-777-4708, [email protected]
- Principal Investigator: Anna Bruckner, MD
- Thomas Jefferson University - Philadelphia, PA
- Contact: Diana Clarkson, 215-503-7931, [email protected]
- Principal Investigator: Bahar Dasgeb, MD
You can also view trial information on ClinicalTrials.gov and PhoenixTissueRepair.com.
Q: Are travel costs covered for patients?
A: All travel costs are covered for the patient and caregivers including flights, car, meals, mileage and gas fees, and hotel accommodations, as some visits require overnight stays for assessments. Everything can be booked through our white-glove concierge service, Greenphire, so patients don’t have to pay out of pocket and await reimbursement. Patients can also receive a prepaid card to assist with travel expenses.
Q: Will patients be able to receive the therapy after the trial is over?
A: Yes, we are providing Collagen VII with an open-label extension protocol.