Official Title
Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
Brief Summary

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.

Intermediate-size Population
Niemann-Pick Disease, Type C

Drug: Arimoclomol
Participants receive prescribed arimoclomol by oral administration

Eligibility Criteria

Inclusion Criteria: - 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. - If the patient is a sexually active female of child-bearing potential (post-menarche), it is agreed to use highly effective contraception during the EAP and until three weeks after the last dose of arimoclomol. - Confirmed negative urine pregnancy test for sexually active female of child-bearing potential (post-menarche). - All sexually active male patients with female partners of child-bearing potential (postmenarche) agree to use a condom in addition to the birth control used by their partners during treatment and until three weeks after the last dose of arimoclomol. - If the patient has a history of seizures, the condition must be adequately controlled, i.e., the pattern of seizure activity must be stable, and the patient must be on a stable dose and regimen of antiepileptic medication during one month prior to screening. - Patient or parent/guardian must provide written informed consent to participate in EAP. - In line with Patterson et al. 2017

Exclusion Criteria: - 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. - The patient will undergo treatment with another investigational drug*, whilst participating in the program or in the 4 weeks prior to commencing treatment with arimoclomol. - The patient is either eligible and able to participate in or is currently participating in an active interventional clinical trial within the indication. - The patient, in the opinion of the clinician, is unable to comply with the treatment or has a medical condition that would potentially increase the risk to the patient by participation. - The patient has a medical condition which hinders the clinician's assessment of arimoclomol safety and efficacy (e.g. certain epileptic conditions or severe cataplexy). - Including unlicensed product provided under an Early Access Program or equivalent compassionate use programs

Eligibility Gender
Eligibility Age
Minimum: 2 Years
United States

University of Alabama Birmingham
Birmingham, Alabama, 35233


Investigator: Toni Seay
Contact: 205-934-9508

Investigator: Joy Dean, MD
Contact: 2059344983

Investigator: Joy Dean, MD

Children's Hospital Los Angeles/University of Southern California/Keck School of Medicine
Los Angeles, California, 90027


Investigator: Francesca Montellanos
Contact: 323-361-5704

Investigator: Alvaro H Serrano Russi, MD

UCSF Benioff Children's Hospital and Research Center/ UCSF
Oakland, California, 94609


Investigator: Veronica Cheung, CRC
Contact: 510-428-3885


Investigator: Caroline Hastings, MD

Children's Hospital of Orange County (CHOC)
Orange, California, 92868


Investigator: Nina Movsesyan, PhD
Contact: 714-509-3008

Investigator: Raymond Wang, MD

Investigator: Raymond Wang, MD

Nicklaus Children's Hospital
Miami, Florida, 33155


Investigator: Marinellie Vega, BA, CCRC
Contact: 786-624-3516

Investigator: Dainelys Pena Rodriguez

Investigator: Paula Schleifer, MD

Emory University
Atlanta, Georgia, 30322


Investigator: Ami Rosen, MS, CGC
Contact: 404-778-8536

Investigator: William Wilcox, MD

Investigator: William Wilcox, MD

Rush University Medical Center
Chicago, Illinois, 60612


Investigator: Orsi Albert, MS
Contact: 312-942-4036

Investigator: Elizabeth Berry-Kravis, MD, PhD

Investigator: Elizabeth Berry-Kravis, MD, PhD

Boston Childrens Hospital
Boston, Massachusetts, 02215


Investigator: Eorna Maguire-Lobos
Contact: 617-919-1399

Investigator: Olaf Bodamer, MD

Investigator: Walla Al-Hertani, MD

Mayo Clinic Children's Center
Rochester, Minnesota, 55905


Investigator: Vanessa Morrow
Contact: 507-538-0266

Investigator: Marc Patterson, MD

Investigator: Marc Patterson, MD, PhD

New York University School of Medicine
New York, New York, 10017


Investigator: Claire MIller, MD
Contact: 212-263-7744

Investigator: Danika Anganoo-Khan
Contact: 929-455-5629

Investigator: Claire Miller, MD

Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229


Investigator: Farrah Jackson
Contact: 513-636-8093

Investigator: Lisa Berry, LGC
Contact: 800-647-4805

Investigator: Loren Pena, MD, PhD

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104


Investigator: Can Ficicioglu, MD, PhD
Contact: 215-590-3376

Investigator: Emily Nolasco-Barrientos, MPH
Contact: 657-549-2423

Investigator: Can Ficicioglu, MD, PhD

UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224


Investigator: Nadene Hendersen
Contact: 412-692-6378

Investigator: Kaylee Williams
Contact: 412-692-8413

Investigator: Damara Ortiz, MD

Dell Children's Medical Center
Austin, Texas, 78723


Investigator: Kendra Koch, PhD
Contact: 512-785-4442

Investigator: Karla Robles-Lopez
Contact: 346-370-7735

Investigator: Kristina Julich, MD

UT Health / McGovern Medical School; Division of Medical Genetics
Houston, Texas, 77030


Investigator: Daniela Bustamante
Contact: 713-500-5779

Investigator: Marilyn Garcia
Contact: 713 500 8937

Investigator: Paul Hillman, MD


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NCT Number
MeSH Terms
Pick Disease of the Brain
Aphasia, Primary Progressive
Frontotemporal Dementia
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C