Nieman-Pick Disease Type C Treatment with Arimoclomol

Early Access Program with Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US

Protocol Description

This early access program (EAP) makes the investigational medicine arimoclomal available for use outside of a clinical trial to treat patients with the life-threatening condition, Nieman-Pick Disease type C (NPC), when no comparable or satisfactory alternative treatment options are available. NPC is a rare, relentlessly progressive, neurological disease that is associated with serious morbidity and shortened life expectancy.

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting people with NPC, ages 2 and older.
Males and Females: Ages 2 years and older


Study participants will take arimoclomal 3 times a day until their doctor deterimes it no longer helps them, the participant withdraws, or the study is stopped for any reason. Blood samples are collected before treatment begins and at months 1, 4, 7 then as indicated, as long as the participant remains in the study.
Visits: Approximately every 3 months
Duration: Ongoing as long as the EAP continues and it is in the participant’s interest to remain in the study

Status: Open to Enrollment

Source(s) of Support

Orphazyme A/S

Primary Investigator

Damara Ortiz, MD

Contact Information

For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC