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Clinical Trials & Research Studies Bulletin Board

The NBIA Disorders Association posts the information at this link for informational purposes only. While the organization supports and encourages the discovery of treatments for NBIA individuals and willingly posts information concerning research studies (such as questionnaires and clinical trial enrollment), we do not endorse specific studies. Nor do we advise NBIA individuals or their families to take part in a particular study. Rather, we believe that those decisions are best made by affected individuals and/or their families, in collaboration with their doctors.

May 4, 2017
Retrophin recruiting for PKAN drug study

Retrophin Inc. has begun to recruit patients for its planned clinical trial for PKAN patients.

The company plans to test a drug, fosmetpantotenate, the new name for RE-O24, to see if it can help patients with the most common NBIA disorder, PKAN. Retrophin had hoped to begin the study late last year, but a manufacturing problem caused a delay until now.

In the clinical trial, which takes its name from the drug and is being called the FORT Study, Retrophin plans to recruit 82 people diagnosed with PKAN in North America and Europe. Eligible patients must range in age from 6 to 65, be able to breathe without a ventilator, must be off of deferiprone for 30 days and cannot have had a deep brain stimulation device implanted within the past six months.

For information about the study, trial sites and screening requirements for participants, visit www.pkanfortstudy.com. Information about the study is also available on www.clinicaltrials.gov.

October, 2016
Health Questionnaire

Retrophin, a company working on a drug to treat individuals with PKAN, is seeking 33 patients and caregivers to help it gain a better understanding of how PKAN affects their daily lives.

The company has hired a consulting firm, Evidera, to interview PKAN individuals and caregivers using a health questionnaire and phone interviews. Retrophin is developing a potential treatment for individuals with PKAN, and the health questionnaire will be used in this program. Retrophin won’t receive the names of questionnaire participants to maintain patient confidentiality throughout the process.

To take part in the questionnaire, PKAN patients must be 16 or older. Eligible caregivers must be at least 18 and caring for a PKAN patient who is at least 6 years old. If you are an eligible patient or caregiver and want to participate, call Evidera toll-free at 800-257-8560. You will need to leave your name and number, with a good time to reach you. Participants will be compensated for their time.

If you aren’t sure about taking part but want to learn more about the questionnaire, contact Regina Buachie at Evidera at 240-235-2429 or via email at PKAN@evidera.com.




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Volunteering for Clinical Trials

The burden falls on us to volunteer for clinical trials if we are ever to have improved treatments or cures. This article contains information about the clinical trial process.


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