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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.
ClinicalTrials.gov Identifier: NCT04316637
Expanded Access Status : Available
First Posted : March 20, 2020
Last Update Posted : August 22, 2022
Information provided by (Responsible Party):
KemPharm Denmark A/S

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:

  • 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

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 ClinicalTrials.gov identifier (NCT number): NCT04316637

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Contact: Orphazyme Inc Medical Information +1-866-696-3346 medicalaffairs@orphazyme.com
Contact: Clinigen Customer service +1 877-768-4303 usmapoperations@clinigengroup.com

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United States, Alabama
University of Alabama Birmingham Available
Birmingham, Alabama, United States, 35233
Contact: Toni Seay    205-934-9508    tmseay@uabmc.edu   
Contact: Joy Dean, MD    2059344983    joydean@uabmc.edu   
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: Talaya Martinez, CRC    323-361-4718    tamartinez@chla.usc.edu   
Contact: Jose Camacho, MD       jocamacho@chla.usc.edu   
Principal Investigator: Jose Camacho, MD         
UCSF Benioff Children's Hospital and Research Center/ UCSF Available
Oakland, California, United States, 94609
Contact: Veronica Cheung, CRC    510-428-3885 ext 2752    Veronica.Cheung@ucsf.edu   
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    nmovsesyan@choc.org   
Contact: Raymond Wang, MD       RaWang@choc.org   
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    marinellie.vega@nicklaushealth.org   
Contact: Dainelys Pena Rodriguez       Dainelys.PenaRodriguez@Nicklaushealth.org   
Principal Investigator: Paula Schleifer, MD         
United States, Georgia
Emory University Available
Atlanta, Georgia, United States, 30322
Contact: Ami Rosen, MS, CGC    404-778-8536    arosen3@emory.edu   
Contact: William Wilcox, MD       william.wilcox@emory.edu   
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    Orsolya_K_Albert@rush.edu   
Contact: Elizabeth Berry-Kravis, MD, PhD       Elizabeth_Berry-Kravis@rush.edu   
Principal Investigator: Elizabeth Berry-Kravis, MD, PhD         
United States, Massachusetts
Boston Childrens Hospital Available
Boston, Massachusetts, United States, 02215
Contact: Danielle Friedman, MSN, CPNP-BC, CNRN    617-919-1459    Danielle.Friedman@childrens.harvard.edu   
Contact: Olaf Bodamer, MD       Olaf.Bodamer@childrens.harvard.edu   
Sub-Investigator: Walla Al-Hertani, MD         
United States, Minnesota
Mayo Clinic Children's Center Available
Rochester, Minnesota, United States, 55905
Contact: Bridget Neja, CRC    507-266-9150    Neja.Bridget@mayo.edu   
Contact: Marc Patterson, MD       Patterson.Marc@mayo.edu   
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: Nicolas Abreu, MD       nicolas.abreu@nyulangone.org   
Contact: Danika Anganoo-Khan    929-455-5629    Danika.Anganoo-Khan@nyulangone.org   
Principal Investigator: Nicolas Abreu, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Available
Cincinnati, Ohio, United States, 45229
Contact: Farrah Jackson    513-636-8093    Farrah.Jackson@cchmc.org   
Contact: Lisa Berry, LGC    800-647-4805    Lisa.Berry@cchmc.org   
Principal Investigator: Loren Pena, MD, PhD         
United States, Pennsylvania
Children's Hospital of Philadelphia Available
Philadelphia, Pennsylvania, United States, 19104
Contact: Can Ficicioglu, MD    215-590-3376    FICICIOGLU@email.chop.edu   
Contact: Genevieve Nesom, MPH, MBE    267-426-1368    NesomG@chop.edu   
Principal Investigator: Can Ficicioglu, MD, PhD         
UPMC Children's Hospital of Pittsburgh Available
Pittsburgh, Pennsylvania, United States, 15224
Contact: Nadene Hendersen    412-692-6378    Nadene.Hendersen@chp.edu   
Contact: Kaylee Williams    412-692-8413    williamsk38@upmc.edu   
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    kdkoch@utexas.edu   
Contact: Karla Robles-Lopez    346-370-7735    karla.robleslopez@austin.utexas.edu   
Principal Investigator: Kristina Julich, MD         
UT Health / McGovern Medical School; Division of Medical Genetics Available
Houston, Texas, United States, 77030
Contact: Daniela Bustamante    713-500-5779    Daniela.M.Bustamante@uth.tmc.edu   
Contact: Marilyn Garcia    713 500 8937    Marilyn.Garcia@uth.tmc.edu   
Principal Investigator: Paul Hillman, MD         
Sponsors and Collaborators
KemPharm Denmark A/S
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Responsible Party: KemPharm Denmark A/S
ClinicalTrials.gov Identifier: NCT04316637    
Other Study ID Numbers: OR-ARI-EAP-NPC
First Posted: March 20, 2020    Key Record Dates
Last Update Posted: August 22, 2022
Last Verified: August 2022
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