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.
Drug: Arimoclomol
Participants receive prescribed arimoclomol by oral administration
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
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: Talaya Martinez, CRC
Contact: 323-361-4718
Investigator: Jose Camacho, MD
Investigator: Jose Camacho, 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: Danielle Friedman, MSN, CPNP-BC, CNRN
Contact: 617-919-1459
Investigator: Olaf Bodamer, MD
Investigator: Walla Al-Hertani, MD
Mayo Clinic Children's Center
Rochester, Minnesota, 55905
Investigator: Bridget Neja, CRC
Contact: 507-266-9150
Investigator: Marc Patterson, MD
Investigator: Marc Patterson, MD, PhD
New York University School of Medicine
New York, New York, 10017
Investigator: Nicolas Abreu, MD
Investigator: Danika Anganoo-Khan
Contact: 929-455-5629
Investigator: Nicolas Abreu, 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
Contact: 215-590-3376
Investigator: Genevieve Nesom, MPH, MBE
Contact: 267-426-1368
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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