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Early Access Program With Arimoclomol in US Patients With NPC

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04316637
Expanded Access Status : Available
First Posted : March 20, 2020
Last Update Posted : September 30, 2021
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment
Niemann-Pick Disease, Type C Drug: Arimoclomol

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
Official Title: Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US

Intervention Details:
  • Drug: Arimoclomol
    Participants receive prescribed arimoclomol by oral administration

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  • 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 sexually active, it is agreed to use effective contraception.
  • Confirmed negative urine pregnancy test for sexually active female of child-bearing potential (post-menarche).
  • 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.

Exclusion Criteria:

  • Severe liver insufficiency.
  • Renal insufficiency.
  • Known or suspected allergy or intolerance to arimoclomol or its constituents.
  • The patient is pregnant, planning to become pregnant (while on the study) 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04316637

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Contact: Orphazyme Inc Medical Information +1-866-696-3346
Contact: Clinigen Customer service +1 877-768-4303

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United States, Alabama
University of Alabama Birmingham Available
Birmingham, Alabama, United States, 35233
Contact: Toni Seay    205-934-9508   
Contact: Joy Dean, MD    2059344983   
Principal Investigator: Joy Dean, MD         
United States, California
Children's Hospital Los Angeles/University of Southern California/Keck School of Medicine Available
Los Angeles, California, United States, 90027
Contact: Francesca Montellanos    323-361-5704   
Principal Investigator: Alvaro H Serrano Russi, MD         
UCSF Benioff Children's Hospital and Research Center/ UCSF Available
Oakland, California, United States, 94609
Contact: Veronica Cheung    510-428-3885 ext 2752      
Principal Investigator: Caroline Hastings, MD         
Children's Hospital of Orange County (CHOC) Available
Orange, California, United States, 92868
Contact: Nina Movsesyan, PhD    714-509-3008   
Contact: Raymond Wang, MD   
Principal Investigator: Raymond Wang, MD         
United States, Florida
Nicklaus Children's Hospital Available
Miami, Florida, United States, 33155
Contact: Marinellie Vega, BA, CCRC    786-624-3516   
Contact: Dainelys Pena Rodriguez   
Principal Investigator: Paula Schleifer, MD         
United States, Georgia
Emory University Available
Atlanta, Georgia, United States, 30322
Contact: Ami Rosen, MS, CGC    404-778-8536   
Contact: William Wilcox, MD   
Principal Investigator: William Wilcox, MD         
United States, Illinois
Rush University Medical Center Available
Chicago, Illinois, United States, 60612
Contact: Orsi Albert, MS    312-942-4036   
Contact: Elizabeth Berry-Kravis, MD, PhD   
Principal Investigator: Elizabeth Berry-Kravis, MD, PhD         
United States, Massachusetts
Boston Childrens Hospital Available
Boston, Massachusetts, United States, 02215
Contact: Eorna Maguire-Lobos    617-919-1399   
Contact: Olaf Bodamer, MD   
Sub-Investigator: Walla Al-Hertani, MD         
United States, Minnesota
Mayo Clinic Children's Center Available
Rochester, Minnesota, United States, 55905
Contact: Vanessa Morrow    507-538-0266   
Contact: Marc Patterson, MD   
Principal Investigator: Marc Patterson, MD, PhD         
United States, New York
New York University School of Medicine Available
New York, New York, United States, 10017
Contact: Claire MIller, MD    212-263-7744   
Contact: Danika Anganoo-Khan    929-455-5629   
Principal Investigator: Claire Miller, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Available
Cincinnati, Ohio, United States, 45229
Contact: Lisa Berry, LGC    800-647-4805   
Contact: Melissa Kahler    513-636-9938   
Principal Investigator: Loren Pena, MD, PhD         
United States, Pennsylvania
Children's Hospital of Philadelphia Available
Philadelphia, Pennsylvania, United States, 19104
Contact: Can Ficicioglu, MD, PhD    215-590-3376   
Contact: Emily Nolasco-Barrientos, MPH    657-549-2423   
Principal Investigator: Can Ficicioglu, MD, PhD         
UPMC Children's Hospital of Pittsburgh Available
Pittsburgh, Pennsylvania, United States, 15224
Contact: Nadene Hendersen    412-692-6378   
Contact: Kaylee Williams    412-692-8413   
Principal Investigator: Damara Ortiz, MD         
United States, Texas
Dell Children's Medical Center Available
Austin, Texas, United States, 78723
Contact: Kendra Koch, PhD    512-785-4442   
Contact: Karla Robles-Lopez    346-370-7735   
Principal Investigator: Kristina Julich, MD         
UT Health / McGovern Medical School; Division of Medical Genetics Available
Houston, Texas, United States, 77030
Contact: Heather Saavedra, MS, RD, LD    713-500-7098   
Contact: Ify Ibekwe    713-500-5064   
Principal Investigator: Paul Hillman, MD         
Sponsors and Collaborators
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Responsible Party: Orphazyme Identifier: NCT04316637    
Other Study ID Numbers: OR-ARI-EAP-NPC
First Posted: March 20, 2020    Key Record Dates
Last Update Posted: September 30, 2021
Last Verified: September 2021
Additional relevant MeSH terms:
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Pick Disease of the Brain
Aphasia, Primary Progressive
Frontotemporal Dementia
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Frontotemporal Lobar Degeneration
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
TDP-43 Proteinopathies
Neurodegenerative Diseases
Proteostasis Deficiencies
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases