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Early Access Program With Arimoclomol in US Patients With NPC

Primary Purpose

Niemann-Pick Disease, Type C

Status
Available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Arimoclomol
Sponsored by
ZevraDenmark
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Niemann-Pick Disease, Type C

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All Sexes

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

Sites / Locations

  • University of Alabama Birmingham
  • UCSF Benioff Children's Hospital and Research Center/ UCSF
  • Children's Hospital of Orange County (CHOC)
  • Nicklaus Children's Hospital
  • Rush University Medical Center
  • Boston Childrens Hospital
  • Mayo Clinic Children's Center
  • New York University School of Medicine
  • Cincinnati Children's Hospital Medical Center
  • Children's Hospital of Philadelphia
  • UPMC Children's Hospital of Pittsburgh
  • Dell Children's Medical Center
  • UT Health / McGovern Medical School; Division of Medical Genetics

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
March 18, 2020
Last Updated
June 28, 2023
Sponsor
ZevraDenmark
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1. Study Identification

Unique Protocol Identification Number
NCT04316637
Brief Title
Early Access Program With Arimoclomol in US Patients With NPC
Official Title
Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
Study Type
Expanded Access

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ZevraDenmark

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Niemann-Pick Disease, Type C

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Arimoclomol
Intervention Description
Participants receive prescribed arimoclomol by oral administration

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Zevra Therapeutics Medical Information
Phone
+1-866-696-3346
Email
medicalaffairs@zevra.com
First Name & Middle Initial & Last Name or Official Title & Degree
Clinigen Customer service
Phone
+1 877-768-4303
Email
usmapoperations@clinigengroup.com
Facility Information:
Facility Name
University of Alabama Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Toni Seay
Phone
205-934-9508
Email
tmseay@uabmc.edu
First Name & Middle Initial & Last Name & Degree
Joy Dean, MD
Phone
2059344983
Email
joydean@uabmc.edu
First Name & Middle Initial & Last Name & Degree
Joy Dean, MD
Facility Name
UCSF Benioff Children's Hospital and Research Center/ UCSF
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Veronica Cheung, CRC
Phone
510-428-3885
Ext
2752
Email
Veronica.Cheung@ucsf.edu
First Name & Middle Initial & Last Name & Degree
Caroline Hastings, MD
Facility Name
Children's Hospital of Orange County (CHOC)
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nina Movsesyan, PhD
Phone
714-509-3008
Email
nmovsesyan@choc.org
First Name & Middle Initial & Last Name & Degree
Raymond Wang, MD
Email
RaWang@choc.org
First Name & Middle Initial & Last Name & Degree
Raymond Wang, MD
Facility Name
Nicklaus Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marinellie Vega, BA, CCRC
Phone
305-968-5491
Email
marinellie.vega@nicklaushealth.org
First Name & Middle Initial & Last Name & Degree
Paola Gracia, CRC
Phone
305.794.5071
Email
Paola.Gracia@NicklausHealth.org
First Name & Middle Initial & Last Name & Degree
Paula Schleifer, MD
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Orsi Albert, MS
Phone
312-942-4036
Email
Orsolya_K_Albert@rush.edu
First Name & Middle Initial & Last Name & Degree
Elizabeth Berry-Kravis, MD, PhD
Email
Elizabeth_Berry-Kravis@rush.edu
First Name & Middle Initial & Last Name & Degree
Elizabeth Berry-Kravis, MD, PhD
Facility Name
Boston Childrens Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Danielle Friedman, MSN, CPNP-BC
Phone
617-919-1459
Email
Danielle.Friedman@childrens.harvard.edu
First Name & Middle Initial & Last Name & Degree
Walla Al-Hertani, MD
Email
walla.al-hertani@childrens.harvard.edu
First Name & Middle Initial & Last Name & Degree
Olaf Bodamer, MD
Facility Name
Mayo Clinic Children's Center
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Bridget Neja, CRC
Phone
507-266-9150
Email
Neja.Bridget@mayo.edu
First Name & Middle Initial & Last Name & Degree
Marc Patterson, MD
Email
Patterson.Marc@mayo.edu
First Name & Middle Initial & Last Name & Degree
Marc Patterson, MD, PhD
Facility Name
New York University School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10017
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nicolas Abreu, MD
Email
nicolas.abreu@nyulangone.org
First Name & Middle Initial & Last Name & Degree
Danika Anganoo-Khan
Phone
929-455-5629
Email
Danika.Anganoo-Khan@nyulangone.org
First Name & Middle Initial & Last Name & Degree
Nicolas Abreu, MD
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Farrah Jackson
Phone
513-636-8093
Email
Farrah.Jackson@cchmc.org
First Name & Middle Initial & Last Name & Degree
Lisa Berry, LGC
Phone
800-647-4805
Email
Lisa.Berry@cchmc.org
First Name & Middle Initial & Last Name & Degree
Loren Pena, MD, PhD
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Can Ficicioglu, MD
Phone
215-590-3376
Email
FICICIOGLU@email.chop.edu
First Name & Middle Initial & Last Name & Degree
Genevieve Nesom, MPH, MBE
Phone
267-426-1368
Email
NesomG@chop.edu
First Name & Middle Initial & Last Name & Degree
Can Ficicioglu, MD, PhD
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nadene Hendersen, MS, LCGC
Phone
412-692-3475
Email
Nadene.Hendersen@chp.edu
First Name & Middle Initial & Last Name & Degree
Elizabeth McCracken, MS, CGC
Phone
412-692-5662
Email
elizabeth.mccracken@chp.edu
First Name & Middle Initial & Last Name & Degree
Damara Ortiz, MD
Facility Name
Dell Children's Medical Center
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kendra Koch, PhD
Phone
512-785-4442
Email
kdkoch@utexas.edu
First Name & Middle Initial & Last Name & Degree
Karla Robles-Lopez
Phone
346-370-7735
Email
karla.robleslopez@austin.utexas.edu
First Name & Middle Initial & Last Name & Degree
Kristina Julich, MD
Facility Name
UT Health / McGovern Medical School; Division of Medical Genetics
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Daniela Bustamante
Phone
713-500-5779
Email
Daniela.M.Bustamante@uth.tmc.edu
First Name & Middle Initial & Last Name & Degree
Marilyn Garcia
Phone
713 500 8937
Email
Marilyn.Garcia@uth.tmc.edu
First Name & Middle Initial & Last Name & Degree
Paul Hillman, MD

12. IPD Sharing Statement

Learn more about this trial

Early Access Program With Arimoclomol in US Patients With NPC

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