Early Access Program With Arimoclomol in US Patients With NPC

Niemann-Pick Disease, Type C Clinical Trial

Official title:

Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT04316637
• Other study ID #: OR-ARI-EAP-NPC
• Status: Available

Study information

• Verified date: November 2023
• Source: ZevraDenmark
• Contact: Zevra Therapeutics Medical Information
• Phone: +1-888-289-5607
• Email: medicalaffairs[@]zevra.com
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Aphasia, Primary Progressive
• Frontotemporal Dementia
• Niemann-Pick Disease, Type C
• Niemann-Pick Diseases
• Pick Disease of the Brain

Intervention

Drug:
• Arimoclomol
Participants receive prescribed arimoclomol by oral administration

Locations

Country	Name	City	State
United States	Dell Children's Medical Center	Austin	Texas
United States	University of Alabama Birmingham	Birmingham	Alabama
United States	Boston Childrens Hospital	Boston	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	UT Health / McGovern Medical School; Division of Medical Genetics	Houston	Texas
United States	Nicklaus Children's Hospital	Miami	Florida
United States	New York University School of Medicine	New York	New York
United States	UCSF Benioff Children's Hospital and Research Center/ UCSF	Oakland	California
United States	Children's Hospital of Orange County (CHOC)	Orange	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Mayo Clinic Children's Center	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
ZevraDenmark

Country where clinical trial is conducted

United States,