Early Access Program With Arimoclomol in US Patients With NPC
Available · NCT04316637
Conditions: Niemann-Pick Disease, Type C
At a glance
- Research payments reported, 2025
- $423,933
- U.S. investigators named
- 11
- Sites paid
- 12
About the trial
- Sponsor
- ZevraDenmark
- Tested
- Arimoclomol (drug)
- Results posted
- Not yet
NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.
From ClinicalTrials.gov, last updated 2024-08-16. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Zevra Therapeutics Inc reported $423,933 in research payments for this study in 2025 (37 payments).
Products named: Miplyffa
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Caroline Hastings, MD Pediatric Hematology & Oncology · Oakland, CA
- Paula P. Schleifer, MD Child Neurology · Miami, FL
- Elizabeth Berry-kravis, M.D. Child Neurology · Chicago, IL
- Kristina K. Julich, MD Neurology · Boston, MA
- Melinda J. Peters, M.D. Medical Genetics · Boston, MA
- Marc C. Patterson, MD Neurology · Rochester, MN
- Nicolas J. Abreu, M.D. Child Neurology · New York, NY
- Nancy D. Leslie, M.D. Medical Genetics · Cincinnati, OH
- Can H. Ficicioglu, M.D. Medical Genetics · Philadelphia, PA
- Damara Ortiz, M.D. Medical Genetics · Pittsburgh, PA
- Paul Hillman, MD/PHD Medical Genetics · Houston, TX
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Boston Medical Center, MA $214,060
- Mayo Clinic Childrens Center $63,295
- Children's Hospital of Orange County, CA $46,050
- Children's Hospital of Philadelphia, PA $20,576
- Ronald Reagan UCLA Medical Center, CA $19,242
- Cincinnati Children's Hospital Medical Center, OH $17,380
- UPMC Children's Hospital of Pittsburgh, PA $14,141
- Dell Children's Medical Center, TX $11,698
- Nicklaus Children's Hospital, FL $6,025
- Harris County Psychiatric Center, TX $4,006
- New York University School of Medicine $3,975
- Rush University Medical Center, IL $3,485
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.