Early Access Program With Arimoclomol in US Patients With NPC
ConditionNiemann-Pick Disease, Type C
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age2+
SponsorZevraDenmark
About this trial
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.
Eligibility criteria
Qualifiers
The patient has a confirmed diagnosis of NPC (NPC1 or NPC2)* and at least one neurological symptom.
The patient is two years of age or above.
The patient is a permanent resident of US.
If taking miglustat (Zavesca®), the patient must have been on the target dose for the past six weeks.
Disqualifiers
Severe liver insufficiency.
Renal insufficiency.
The patient has a known or suspected allergy or intolerance to arimoclomol or its constituents.
The patient is pregnant, planning to become pregnant (while on the EAP program) or is currently breastfeeding.
Trial design
Treatments tested in this trial
- Arimoclomol
Treatment groups
No treatment groups listed
Sponsors and collaborators
Source ClinicalTrials.gov