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Showing 1–24 of 100 matching trials from the live ClinicalTrials.gov search.
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Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
Interventions
AR-42
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
4
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT, Elaprase
Drug
Lead sponsor
Takeda
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2024
U.S. locations
7
States / cities
Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
Completed Phase 2 Interventional Accepts healthy volunteers Results available

AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas

NCT02831257
Conditions
Neurofibromatosis 2, Meningioma
Interventions
AZD2014
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
2
States / cities
Boston, Massachusetts
Conditions
Neurofibromatosis 2
Interventions
Penetrating auditory brainstem implant
Device
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
3
States / cities
Los Angeles, California • Pasadena, California • Englewood, Colorado
Conditions
Craniofacial Microsomia, Hemifacial Microsomia, Oculo-Auriculo-Vertebral-Syndrome, Goldenhar Syndrome, Microtia
Interventions
Not listed
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
12 Months to 24 Months
Enrollment
417 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
5
States / cities
Los Angeles, California • Chicago, Illinois • Chapel Hill, North Carolina + 2 more
Conditions
Usher Syndrome, Retinitis Pigmentosa, Congenital Hearing Impairment
Interventions
Audiogram, Electroretinogram
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2002
U.S. locations
1
States / cities
New York, New York
Conditions
Microtia
Interventions
AUR-201
Combination Product
Lead sponsor
Auregen Biotherapeutics, SA
Industry
Eligibility
8 Years to 29 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
New York, New York
Conditions
Hunter Syndrome
Interventions
idursulfase-IT, No IT treatment
Biological · Other
Lead sponsor
Shire
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
No Longer Available No phase listed Expanded access

Compassionate Use Arm - ABI541 ABI for 10 NF2 Patients

NCT02589912
Conditions
Neurofibromatosis Type 2, Severe Profound Sensorineural Hearing Loss
Interventions
Nucleus Profile ABI541 Auditory Brainstem Implant
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
12 Years to 80 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Ototoxicity
Interventions
Placebo, SPI-1005 Ebselen 200mg Capsule x1, SPI-1005 Ebselen 200mg Capsule x2, SPI-1005 Ebselen 200mg Capsule x3
Drug
Lead sponsor
Sound Pharmaceuticals, Incorporated
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Charleston, South Carolina
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Active, not recruiting Phase 2 Interventional

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034
Conditions
Wolfram Syndrome
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
17 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Conditions
Vestibular Schwannoma, Acoustic Neuroma, Neurofibromatosis 2
Interventions
Aspirin, Placebo
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
12 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
5
States / cities
Stanford, California • Miami, Florida • Boston, Massachusetts + 2 more
Conditions
Retinitis Pigmentosa Syndromic, Congenital Deafness, Usher Syndrome, Retinitis Pigmentosa and Deafness, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
2 Years and older
Enrollment
249 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2019
U.S. locations
2
States / cities
Bethesda, Maryland • New York, New York
Conditions
Neurofibromatosis 2, Vestibular Schwannoma, Meningioma, Ependymoma, Glioma
Interventions
Selumetinib
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years to 45 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Sensorineural Hearing Loss, Long QT Syndrome
Interventions
Not listed
Lead sponsor
Pediatrix
Other
Eligibility
1 Day to 90 Days
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
12
States / cities
Anchorage, Alaska • Phoenix, Arizona • Waterford, Connecticut + 6 more
Conditions
Cleft Palate
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
Up to 24 Months
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2017
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Usher Syndrome, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
Portland, Oregon
Terminated Phase 1Phase 2 Interventional

AuriNovo for Auricular Reconstruction

NCT04399239
Conditions
Microtia
Interventions
AuriNovo
Combination Product
Lead sponsor
3DBio Therapeutics
Industry
Eligibility
6 Years to 25 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Beverly Hills, California • San Antonio, Texas
Conditions
CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma, Neurofibromatosis
Interventions
Sunitinib
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
6
States / cities
Boston, Massachusetts • Basking Ridge, New Jersey • Commack, New York + 3 more
Conditions
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions
Glycerol Tributyrate
Drug
Lead sponsor
George Washington University
Other
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Mitochondrial Disease
Interventions
N-Acetylcysteine
Drug
Lead sponsor
Michio Hirano, MD
Other
Eligibility
18 Years to 80 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
New York, New York
Conditions
Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma, Adult Grade II Meningioma, Adult Meningeal Hemangiopericytoma, Adult Papillary Meningioma, Neurofibromatosis Type 1, Neurofibromatosis Type 2, Recurrent Adult Brain Tumor
Interventions
bevacizumab
Biological
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2018
U.S. locations
5
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 2 more