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Trial record 5 of 12 for:    arimoclomol

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 : May 12, 2020
Sponsor:
Information provided by (Responsible Party):
Orphazyme

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

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

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 ClinicalTrials.gov identifier (NCT number): NCT04316637


Contacts
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Contact: Orphazyme Inc Medical Information +1 312-282-7033 usmedicalaffairs@orphazyme.com
Contact: Clinigen Customer service +1 877-768-4303 usmapoperations@clinigengroup.com

Locations
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United States, California
Children's Hospital of Orange County (CHOC) Available
Orange, California, United States, 92868
Contact: Nina Movsesyan, PhD    714-509-3008    nmovsesyan@choc.org   
Principal Investigator: Raymond Wang, MD         
Sponsors and Collaborators
Orphazyme
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Responsible Party: Orphazyme
ClinicalTrials.gov Identifier: NCT04316637    
Other Study ID Numbers: OR-ARI-EAP-NPC
First Posted: March 20, 2020    Key Record Dates
Last Update Posted: May 12, 2020
Last Verified: May 2020
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
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Aphasia
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
Signs and Symptoms
TDP-43 Proteinopathies
Neurodegenerative Diseases
Proteostasis Deficiencies
Metabolic Diseases
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Histiocytosis, Non-Langerhans-Cell
Histiocytosis