Early Access Program With Arimoclomol in US Patients With NPC
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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
|
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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 |
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 |

- Drug: Arimoclomol
Participants receive prescribed arimoclomol by oral administration

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.
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.
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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.
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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

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