Pachyonychia Congenitia Project

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Phase 2/3 Clinical Trial – VALO

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Sign up at https://www.surveygizmo.com/s3/4854812/Phase2-3

PDF Documents to download about VALO study

Phase 2/3 VALO Study: Frequently Asked Questions

“Do I get paid for being in the study?”
Yes, you are paid for your time and effort. The amount is up to $1,200 if all visits and assessments are completed.

“How can I participate if I can’t take time off-of work to travel to clinic visits?”
At the end of September 2019, a new site opened that can see VALO patients on the weekends. If you would like to participate at this site, you can contact PC Project.

“Does the study provide financial reimbursement for babysitting?”
Yes, the study provides reimbursement for respite care (care for children, spouse, seniors, animals, etc.). You will have to provide receipts or documentation.

“The nearest site is 3 hours away, too short to fly. What if I can’t drive, don’t want to drive and/or don’t have someone to drive me?”
The study provides transportation to and from the clinical site for you and a companion. In this case, a car and driver would be provided to you to assist with the round trip. The study also provides financial support for transportation (train, bus, gas mileage, tolls, airfare), lodging and other costs incurred as a result of study participation.

“How can I find out if I think I might be taking a medication that I can’t take during the study?”
First, don’t stop any medications that have previously been approved by your doctor. If you are interested in participating, please contact PC Project so they can connect you with a clinical trial clinician that can answer your questions to see if you are a candidate for the study.

“When will study enrollment close?”
Study enrollment may close as early as the Feb 2020…but we need more patients! Please complete the short form at https://www.surveygizmo.com/s3/4854812/Phase2-3, if you are interested in participating to get started.

“What if the study drug works for me during the study, can I still take it after I finish the study?”
An extension study will be opening at the beginning of 2020, which will allow any patient that received study drug in the VALO study, and the patient and clinician believed helped the patient, to continue to use experimental PTX-022 (QTORIN™ topical rapamycin). The study drug will be provided at no cost to the patient.

Seeking Participants for Pachyonychia Congenita Clinical Research Trial

If you or someone you know has Pachyonychia Congenita (PC), you may be interested in participating in a Phase 2/3 clinical research trial for an experimental therapy that is applied to the soles of your feet.

Some of the requirements for participation include:

  • Must be able to travel to sites in the United States. Travel may include driving and flying
  • Must be fluent in English
  • Must be at least 18 years of age or older
  • Have a clinical diagnosis of PC, genetically confirmed to involve any of three keratin genes KRT6A, KRT6B, or KRT16
    • If you have not been genetically tested, or if you do not have a copy of your genetic testing report, please contact PC Project at study@pachyonychia.org for more information about genetic testing.
      NOTE: If you know your mutation because a family member was tested, you will need to do a confirming test through PC Project, so you will have your own report.
  • Have not participated in a clinical research study in the past sixty days
  • On a daily basis during the study, wear a wrist-based activity monitor and answer questions on a smartphone-based app.

Participants must be, in the opinion of the study doctor, able to understand the study, cooperate with the study procedures and willing to return to the clinic for all of the required visits.

If you qualify for study participation, you will be compensated for your time and your travel expenses to and from the clinic for you and a companion will be covered.

You can find more information about the study and participating sites on www.clinicaltrials.gov and www.valostudy.org.

If you are interested in participating, you can either:

Spread the word to help find the first treatment for Pachyonychia Congenita (PC)

  • Do you know someone who has PC? A family member or a friend?
  • Are they 18 years or older?
  • Are they interested in helping to test a potential first treatment for PC?

We need your help to inform your friends and family about the opportunity to take part in the VALO clinical trial and help us treat PC in the future. Participation is voluntary.

Please invite your family member or friend to sign up for the study here: https://www.surveygizmo.com/s3/4854812/Phase2-3

Invite your family member or friend to contact PC Project at 801-987-8758 or study@pachyonychia.org with questions or to learn more.