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.

Available
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
All
Eligibility Age
Minimum: 2 Years
Countries
United States
Locations

University of Alabama Birmingham
Birmingham, Alabama, 35233

Available

Investigator: Toni Seay
Contact: 205-934-9508
tmseay@uabmc.edu


Investigator: Joy Dean, MD
Contact: 2059344983
joydean@uabmc.edu

Investigator: Joy Dean, MD

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

Available

Investigator: Francesca Montellanos
Contact: 323-361-5704
fmontellanos@chla.usc.edu

Investigator: Alvaro H Serrano Russi, MD

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

Available

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

2752 Veronica.Cheung@ucsf.edu

Investigator: Caroline Hastings, MD

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

Available

Investigator: Nina Movsesyan, PhD
Contact: 714-509-3008
nmovsesyan@choc.org


Investigator: Raymond Wang, MD
RaWang@choc.org

Investigator: Raymond Wang, MD

Nicklaus Children's Hospital
Miami, Florida, 33155

Available

Investigator: Marinellie Vega, BA, CCRC
Contact: 786-624-3516
marinellie.vega@nicklaushealth.org


Investigator: Dainelys Pena Rodriguez
Dainelys.PenaRodriguez@Nicklaushealth.org

Investigator: Paula Schleifer, MD

Emory University
Atlanta, Georgia, 30322

Available

Investigator: Ami Rosen, MS, CGC
Contact: 404-778-8536
arosen3@emory.edu


Investigator: William Wilcox, MD
william.wilcox@emory.edu

Investigator: William Wilcox, MD

Rush University Medical Center
Chicago, Illinois, 60612

Available

Investigator: Orsi Albert, MS
Contact: 312-942-4036
Orsolya_K_Albert@rush.edu


Investigator: Elizabeth Berry-Kravis, MD, PhD
Elizabeth_Berry-Kravis@rush.edu

Investigator: Elizabeth Berry-Kravis, MD, PhD

Boston Childrens Hospital
Boston, Massachusetts, 02215

Available

Investigator: Eorna Maguire-Lobos
Contact: 617-919-1399
Eorna.Maguire@childrens.harvard.edu


Investigator: Olaf Bodamer, MD
Olaf.Bodamer@childrens.harvard.edu

Investigator: Walla Al-Hertani, MD

Mayo Clinic Children's Center
Rochester, Minnesota, 55905

Available

Investigator: Vanessa Morrow
Contact: 507-538-0266
morrow.vanessa@mayo.edu


Investigator: Marc Patterson, MD
Patterson.Marc@mayo.edu

Investigator: Marc Patterson, MD, PhD

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

Available

Investigator: Claire MIller, MD
Contact: 212-263-7744
claire.miller@nyumc.org


Investigator: Danika Anganoo-Khan
Contact: 929-455-5629
Danika.Anganoo-Khan@nyulangone.org

Investigator: Claire Miller, MD

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

Available

Investigator: Farrah Jackson
Contact: 513-636-8093
Farrah.Jackson@cchmc.org


Investigator: Lisa Berry, LGC
Contact: 800-647-4805
Lisa.Berry@cchmc.org

Investigator: Loren Pena, MD, PhD

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Available

Investigator: Can Ficicioglu, MD, PhD
Contact: 215-590-3376
FICICIOGLU@email.chop.edu


Investigator: Emily Nolasco-Barrientos, MPH
Contact: 657-549-2423
nolascobae@email.chop.edu

Investigator: Can Ficicioglu, MD, PhD

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

Available

Investigator: Nadene Hendersen
Contact: 412-692-6378
Nadene.Hendersen@chp.edu


Investigator: Kaylee Williams
Contact: 412-692-8413
williamsk38@upmc.edu

Investigator: Damara Ortiz, MD

Dell Children's Medical Center
Austin, Texas, 78723

Available

Investigator: Kendra Koch, PhD
Contact: 512-785-4442
kdkoch@utexas.edu


Investigator: Karla Robles-Lopez
Contact: 346-370-7735
karla.robleslopez@austin.utexas.edu

Investigator: Kristina Julich, MD

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

Available

Investigator: Daniela Bustamante
Contact: 713-500-5779
Daniela.M.Bustamante@uth.tmc.edu


Investigator: Marilyn Garcia
Contact: 713 500 8937
Marilyn.Garcia@uth.tmc.edu

Investigator: Paul Hillman, MD

Contacts

Orphazyme Inc Medical Information
+1-866-696-3346
medicalaffairs@orphazyme.com

Clinigen Customer service
+1 877-768-4303
usmapoperations@clinigengroup.com

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