Early Access Program With Arimoclomol in US Patients With NPC
Recruiting in Palo Alto (17 mi)
+15 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Academic
Waitlist Available
Sponsor: KemPharm Denmark A/S
No Placebo Group
Trial Summary
What is the purpose of 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
Inclusion Criteria
The patient is two years of age or above.
The patient has a confirmed diagnosis of NPC (NPC1 or NPC2)* and at least one neurological symptom.
If taking miglustat (Zavesca®), the patient must have been on the target dose for the past six weeks.
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Treatment Details
Interventions
- Arimoclomol (Heat Shock Protein Amplifier)
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
University of Alabama BirminghamBirmingham, AL
Children's Hospital of PhiladelphiaPhiladelphia, PA
Mayo Clinic Children's CenterRochester, MN
Cincinnati Children's Hospital Medical Center (CCH)Cincinnati, OH
More Trial Locations
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Who Is Running the Clinical Trial?
KemPharm Denmark A/SLead Sponsor
OrphazymeLead Sponsor
ZevraDenmarkLead Sponsor