Trial record 1 of 8 for:
orphazyme
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 : July 27, 2021
|
Sponsor:
Orphazyme
Information provided by (Responsible Party):
Orphazyme
- Study Details
- Tabular View
- Disclaimer
- How to Read a Study Record
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 |
| 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 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Niemann-Pick disease
CHMP2B-related frontotemporal dementia
Frontotemporal dementia with parkinsonism-17
Inclusion body myopathy with early-onset Paget disease and frontotemporal dementia
GRN-related frontotemporal lobar degeneration
Genetic and Rare Diseases Information Center resources:
Frontotemporal Dementia
Frontotemporal Dementia, Ubiquitin-positive
Primary Progressive Aphasia
Semantic Dementia
Niemann-Pick Disease
Niemann-Pick Disease Type A
Niemann-Pick Disease Type C1
Behavioral Variant of Frontotemporal Dementia
Sphingolipidosis
Non-Langerhans-Cell Histiocytosis
Intervention Details:
- Drug: Arimoclomol
Participants receive prescribed arimoclomol by oral administration
Information from the National Library of Medicine
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 |
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
| Contact: Orphazyme Inc Medical Information | +1-866-696-3346 | USMedInfo@orphazyme.com | |
| Contact: Clinigen Customer service | +1 877-768-4303 | usmapoperations@clinigengroup.com |
Locations
| 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: Francesca Montellanos 323-361-5704 fmontellanos@chla.usc.edu | |
| Principal Investigator: Alvaro H Serrano Russi, MD | |
| UCSF Benioff Children's Hospital and Research Center/ UCSF | Available |
| Oakland, California, United States, 94609 | |
| Contact: Natalie Nardone, PhD Natalie.Nardone@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: Eorna Maguire-Lobos 617-919-1399 Eorna.Maguire@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: Vanessa Morrow 507-538-0266 morrow.vanessa@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: Claire MIller, MD 212-263-7744 claire.miller@nyumc.org | |
| Contact: Danika Anganoo-Khan 929-455-5629 Danika.Anganoo-Khan@nyulangone.org | |
| Principal Investigator: Claire Miller, MD | |
| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center | Available |
| Cincinnati, Ohio, United States, 45229 | |
| Contact: Lisa Berry, LGC 800-647-4805 Lisa.Berry@cchmc.org | |
| Contact: Melissa Kahler 513-636-9938 Melissa.Kahler@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, PhD 215-590-3376 FICICIOGLU@email.chop.edu | |
| Contact: Emily Nolasco-Barrientos, MPH 657-549-2423 nolascobae@email.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: Michele Graham 412-692-6378 Michele.Graham@chp.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: Heather Saavedra, MS, RD, LD 713-500-7098 Heather.Saavedra@uth.tmc.edu | |
| Contact: Ify Ibekwe 713-500-5064 Ifeoma.C.Ibekwe@uth.tmc.edu | |
| Principal Investigator: Paul Hillman, MD | |
Sponsors and Collaborators
Orphazyme
| 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: | July 27, 2021 |
| Last Verified: | July 2021 |
Additional relevant MeSH terms:
|
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 |