December 2, 2024 Posting:

New Clinical Trial Information For Families and Their Physicians

Enrollment in The New Progerinin Clinical Trial Has Begun

Enrollment is now open for the progerinin trial! The title of this research study is: A Phase 2a, Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of progerinin in Patients with Hutchinson-Gilford Progeria Syndrome (HGPS). The study site is Boston Children’s Hospital (BCH). The study sponsor is the Korean-based company PRG Science & Technology (PRG S&T). PRF is collaborating with BCH and PRG S&T on this trial.  As part of the collaboration, PRF will be arranging for travel to and from the trial site in Boston and lodging while in Boston. Trial information is also posted on the website clinicaltrials.gov

Information For Patients with Progeria and their Doctors

• This research trial involves an investigational new drug called progerinin that may be taken in addition to lonafarnib.
• Progerinin has not yet been proven to be safe and/or effective for the treatment of Progeria (HGPS) and has not yet been approved by the FDA or other international authorities for sale.
• The goal of this study is to determine progerinin’s side effects, optimal dose and to start to look at whether progerinin can help treat patients with Progeria.
• Progerinin showed benefits when tested by scientists on Progeria cells and Progeria mice. It has been given to adults without Progeria either once (one dose) or for a few weeks.  It has never been given to children and young adults with Progeria.
• Progerinin is given as a powder dissolved in water and is taken twice a day.
• The trial team anticipates that between 10 and 16 children and young adults will be enrolled who satisfy the specific enrollment criteria established by the study protocol. Enrollment will start with 10 children and young adults with Progeria (HGPS).
• 8 trial participants will take progerinin by mouth twice per day and also continue to take lonafarnib as usual; 2 participants will not take progerinin but will continue to take lonafarnib. The trial team will not know which patients are in the progerinin treatment group or the lonafarnib-only group until it is randomly assigned by a computer.

• If a patient is assigned to take progerinin along with their usual dose of lonafarnib:
  • • The patient will be required to make 4 trips to Boston for testing (4 months between each visit). This means the patient would travel to Boston for their first visit, the patient would return 4 months after the first visit, then 8 months after the first visit, and finally 12 months after the first visit.
    • The first dose of progerinin is a starting dose. Between the first 2 visits to Boston, there will be home requirements while the patient is on this first dose level. The patient will have check-in phone calls or zoom calls with the BCH trial team on days 7, 14, and 28, Month 2, and Month 3.  These calls are meant to monitor any side effects of progerinin.  The patient will also be in close contact with the BCH trial team in case there are side effects between calls.
    • Patients will have blood tests locally where the patient lives between visits to Boston. They will be arranged between BCH and the patient’s home doctor at week 2, week 4, month 2 and month 3. The blood will be drawn from the patient’s arm and the total amount needed at each visit is about half of a teaspoon (2.5 ml). The results of these tests will be sent from the patient’s local doctor’s office to the BCH trial team.

• If a patient is assigned to take only lonafarnib:
  • • The patient will visit Boston 2 times, at baseline and at month 12.
    • The patient may give a blood sample locally to home 4 months after they join the trial. BCH and the patient’s local doctor would arrange for this blood sample to be taken.
• Thirty days after the last in-person visit, all subjects will be asked to complete another phone call with the BCH study team.
• Each patient chooses whether or not to participate in the trial. The clinical trial is completely voluntary.

If you would like to learn more, or if you are interested in being contacted by BCH about enrolling in the trial, please contact The Progeria Research Foundation and we will put you in touch with the BCH trial team.

Shelby Phillips Patient Programs Coordinator, The Progeria Research Foundation
Email: sphillips@progeriaresearch.org

Cell Phone for WhatsApp, Telegram, WeChat: 1-978-876-2407
Office Phone: 978-548-5308

Best Regards,
Shelby Phillips, Patient Programs Coordinator and
Dr. Leslie Gordon, Medical Director
The Progeria Research Foundation