Niemann-Pick Disease, Type CNCT04316637
Early Access Program With Arimoclomol in US Patients With NPC
A study testing Arimoclomol in people with Niemann-Pick Disease, Type C, run by ZevraDenmark at 13 sites in United States.
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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Niemann-Pick Disease, Type C
- What is being tested?
- Arimoclomol
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 13 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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).
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- 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
Who cannot join
- 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.
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- 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
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
- Available
Dell Children's Medical Center
Austin, Texas, 78723
Kendra Koch, PhD, 512-785-4442, kdkoch@utexas.edu
Karla Robles-Lopez, 346-370-7735, karla.robleslopez@austin.utexas.edu
- Available
University of Alabama Birmingham
Birmingham, Alabama, 35233
Toni Seay, 205-934-9508, tmseay@uabmc.edu
Joy Dean, MD, 2059344983, joydean@uabmc.edu
- Available
Boston Childrens Hospital
Boston, Massachusetts, 02215
Danielle Friedman, MSN, CPNP-BC, 617-919-1459, Danielle.Friedman@childrens.harvard.edu
Olaf Bodamer, MD
- Available
Rush University Medical Center
Chicago, Illinois, 60612
Giulia DiMarco, CRC, 312-942-9841, giulia_dimarco@rush.edu
Elizabeth Berry-Kravis, MD, PhD, Elizabeth_Berry-Kravis@rush.edu
- Available
Cincinnati Children's Hospital Medical Center (CCH)
Cincinnati, Ohio, 45229
Farrah Jackson, 513-636-8093, Farrah.Jackson@cchmc.org
Mollie Hemminger, 513-636-3974, Mollie.Hemminger@cchmc.org
- Available
UT Health / McGovern Medical School; Division of Medical Genetics
Houston, Texas, 77030
Emily Garcia, o Emily.Garcia@uth.tmc.edu
Marilyn Garcia, 713 500 8937, Marilyn.Garcia@uth.tmc.edu
- Available
Nicklaus Children's Hospital
Miami, Florida, 33155
Paola Gracia, CRC, 305-794-5071, Paola.Gracia@NicklausHealth.org
Paula Schleifer, MD
- Available
New York University School of Medicine
New York, New York, 10017
Nicolas Abreu, MD, nicolas.abreu@nyulangone.org
Danika Anganoo-Khan, 929-455-5629, Danika.Anganoo-Khan@nyulangone.org
- Available
UCSF Benioff Children's Hospital and Research Center/ UCSF
Oakland, California, 94609
Veronica Cheung, CRC, 510-428-3885 ext. 2752, Veronica.Cheung@ucsf.edu
Caroline Hastings, MD
- Available
Children's Hospital of Orange County (CHOC)
Orange, California, 92868
Nina Movsesyan, PhD, 714-509-3008, nmovsesyan@choc.org
Raymond Wang, MD, RaWang@choc.org
- Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Juan Ramos, ramosjf@chop.edu
Genevieve Nesom, MPH, MBE, 267-426-1368, NesomG@chop.edu
- Available
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Nadene Hendersen, MS, LCGC, 412-692-3475, Nadene.Hendersen@chp.edu
Elizabeth McCracken, MS, CGC, 412-692-5662, elizabeth.mccracken@chp.edu
- Available
Mayo Clinic Children's Center
Rochester, Minnesota, 55905
Bridget Neja, CRC, 507-266-9150, Neja.Bridget@mayo.edu
Marc Patterson, MD, Patterson.Marc@mayo.edu
About this study
Official title: Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
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.
- Conditions
- Niemann-Pick Disease, Type C
- Being tested
- Arimoclomol
- Medical subject headings
- Niemann-Pick Disease, Type C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ArimoclomolDrug
Participants receive prescribed arimoclomol by oral administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Niemann-Pick Disease, Type CStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- ZevraDenmark
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04316637
- Sponsor's protocol ID
- OR-ARI-EAP-NPC
- Status last verified
- Aug 2024
- First submitted
- 18 Mar 2020
- Last updated
- 16 Aug 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
- ClinicalTrials.gov ID
- NCT04316637
- Acronym / sponsor ID
- OR-ARI-EAP-NPC
- Conditions
- Niemann-Pick Disease, Type C
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- ZevraDenmarkIndustry (pharma, biotech, devices)
- First posted
- 20 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ArimoclomolParticipants receive prescribed arimoclomol by oral administration | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Aug 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Arimoclomol in people with Niemann-Pick Disease, Type C, run by ZevraDenmark at 13 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Zevra Therapeutics Medical Information (+1-888-289-5607, medicalaffairs@zevra.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Aug 2024.