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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.


Retrophin adds clinical trial sites

Retrophin Inc. has added more clinical trial locations to test the drug it developed as a possible treatment for Pantothenate Kinase-Associated Neurodegeneration, or PKAN, the most common form of NBIA.

Retrophin is now testing in 10 sites, split evenly between Europe and the United States, with more clinical trial locations expected to open in the second quarter of this year.

We reported in our August/September 2017 newsletter that the company had begun enrolling patients in three North American sites for its Phase 3 FORT trial of fosmetpantotenate, also known as RE-024. It is the first test of a medication targeting the underlying cause of PKAN.

In addition, Retrophin anticipates getting approval for a change in protocol that would drop the age for trial participation from 19 to 18 in the United States. Pediatric enrollment is expected to open by June 30.

RE-024 has been granted orphan drug status by the U.S. Food and Drug Administration and the European Commission, as well as expedited “fast track” approval in the U.S. to speed up the development and review process.

Retrophin also has an agreement with the FDA for a Special Protocol Assessment, an advanced declaration that says the FDA will allow an uncompleted Phase III design, clinical endpoints and statistical analyses. The special assessment does not guarantee that the agency will approve the drug, but assures the company that the FDA won’t object to its trial design.

To judge whether the drug is effective, the trial’s primary endpoint will be to measure whether patients do better on a PKAN Activities of Daily Living scale after 24 weeks. That scale is an adaptation of Part II of the comprehensive and widely-referenced Unified Parkinson’s Disease Rating Scale adapted for PKAN patients, with input from experts, patients, advocacy leaders and regulators.

People with PKAN cannot make enough of an essential enzyme, pantothenate kinase 2 (PANK2), needed to turn vitamin B5 into Coenzyme A, an essential substance for metabolism. Deficiency in Coenzyme A is believed to cause PKAN symptoms. The drug delivers phosphopantothenate to the cells, which could help restore the PANK 2 enzyme, and, in turn, boost Coenzyme A levels.

To stay up-to-date on site openings and for more information on the FORT study, visit the study’s website at www.pkanfortstudy.com or access information on clinicaltrials.gov.

Clinical Trial Sites Now Open for Retrophin Study

  • Barcelona, Spain
  • Boston, Mass.
  • Chicago, Ill.
  • Decatur, Georgia
  • London, England
  • Milan, Italy
  • Munich, Germany
  • New York, N.Y.
  • Pittsburgh, Penn.
  • Prague, Czech Republic



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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