38 unknown. 38 Phase 2, 8 Phase 3, 7 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 38 | Unknown 38 |
| Phase 3 | 8 | Unknown 8 |
| Phase 1 | 7 | Unknown 7 |
Follow Heredodegenerative Disorders, Nervous System: new trials by email Literature Review of Heredodegenerative Disorders, Nervous System trials Research Report on Heredodegenerative Disorders, Nervous System
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Node-Sparing Short-Course Radiation With CAPOX and Sintilimab for MSS Locally Advanced Colon Cancer: A Randomized, Prospective, Multicenter Study Node-Sparing Short-Course Radiotherapy Combined with CAPOX and Sintilimab, CAPOX Chemotherapy - Microsatellite Stable (MSS) Colon Cancer, Locally Advanced Colon Cancer | Phase 2 | Unknown | Sir Run Run Shaw Hospital | 140 | 2024-10-01 | 2024-09-19 |
| CVL237 Tablets for APDS/PASLI CVL237 tablets, CVL237 placebo tablets - PI3K and P110delta Hyperactivation Syndrome | Phase 2 / Phase 3 | Unknown | Convalife (Shanghai) Co., Ltd. | 35 | 2024-03-30 | 2024-03-05 |
| Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1 Tirzepatide - Wolfram Syndrome | Phase 2 | Unknown | Ospedale San Raffaele | 10 | 2024-01-01 | 2024-02-22 |
| Hereditary Sensory Neuropathy Serine Trial L-serine, Placebo - Hereditary Sensory Neuropathy Type I | Phase 2 | Unknown | University College, London | 50 | 2023-08-21 | 2023-11-02 |
| Toxicity and Efficacy Evaluation of Topical Minoxidil in Acne Vulgaris topical minoxidil in Acne Vulgaris - Acne Vulgaris | Phase 1 / Phase 2 | Unknown | Chang Gung Memorial Hospital | 26 | 2023-07-11 | 2023-11-03 |
| Research of SORD-CMT Natural History and Epalrestat Treatment Epalrestat - Charcot-Marie-Tooth Disease (CMT) | Phase 2 | Unknown | The Third Xiangya Hospital of Central South University | 30 | 2023-04-01 | 2023-04-18 |
| Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients VYNT-0126, Placebo - Rett Syndrome | Phase 2 | Unknown | Vyant Bio | 48 | 2023-03-01 | 2022-11-23 |
| Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1 EXG001-307 injection - Spinal Muscular Atrophy Type I | Phase 1 / Phase 2 | Unknown | Guangzhou Jiayin Biotech Ltd | 12 | 2023-02-16 | 2024-09-19 |
| A Multi-Center Study on the Use of Rho-Kinase Inhibitor to Reduce or Prevent PVR in RRD Eyes at High Risk for PVR Netarsudil 0.02% Ophthalmic Solution [RHOPRESSA], Glycerin 0.2%/Hypromellose 0.2%/Peg 1%/Soln,Oph,Ud - Rhegmatogenous Retinal Detachment, Proliferative Vitreoretinopathy | Phase 2 / Phase 3 | Unknown | Wills Eye | 80 | 2023-02-06 | 2024-07-30 |
| Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome Depakine - Wolfram Syndrome | Phase 2 | Unknown | Centre d'Etude des Cellules Souches | 23 | 2021-11-26 | 2023-11-28 |
| Phase 2 Study of Tanezumab in Subjects With Moderate to Severe Pain Due to Schwannomatosis Tanezumab, Placebo - Schwannomatosis, Pain | Phase 2 | Unknown | Massachusetts General Hospital | 9 | 2020-04-30 | 2024-03-13 |
| Clinical Extension Study for Safety and Efficacy Evaluation of Cellavita-HD Administration in Huntington's Patients. Cellavita-HD - Huntington Disease | Phase 2 / Phase 3 | Unknown | Azidus Brasil | 35 | 2020-02-10 | 2022-10-28 |
| Metformin in Patients With Fragile X Metformin, Placebo oral tablet - Fragile X Syndrome | Phase 1 / Phase 2 | Unknown | Rowan University | 40 | 2019-10-29 | 2019-10-31 |
| Treatment of NF1-related Plexiform Neurofibroma With Trametinib Trametinib - Neurofibromatosis 1, Child | Phase 2 | Unknown | Region Skane | 15 | 2019-06-10 | 2024-04-11 |
| Outcomes Mandate National Integration With Cannabis as Medicine Cannabis, Medical, RYAH-Medtech Inhaler - Chronic Pain, Chronic Pain Syndrome | Phase 2 | Unknown | OMNI Medical Services, LLC | 200000 | 2018-12-01 | 2022-06-02 |
| A Study to Examine the Efficacy of a Therapeutic THX-110 for Tourette Syndrome THX-110 (dronabinol plus PEA), Placebo - Tourette Syndrome | Phase 2 | Unknown | Neurothera Labs Inc. | 60 | 2018-08-01 | 2018-08-29 |
| A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy rAAV2-ND4 - Leber Hereditary Optic Neuropathy | Phase 2 / Phase 3 | Unknown | Huazhong University of Science and Technology | 159 | 2017-12-27 | 2020-09-17 |
| Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex Aspirin, AED, Placebo - Tuberous Sclerosis Complex, Aspirin | Phase 2 | Unknown | Peking Union Medical College Hospital | 98 | 2017-11-20 | 2020-06-09 |
| Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. Tridecanoic Acid - Rett Syndrome | Phase 2 | Unknown | Sheba Medical Center | 10 | 2017-04-01 | 2017-02-23 |
| Cabazitaxel in mCRPC Patients With AR-V7 Positive Circulating Tumor Cells (CTCs) Cabazitaxel, Antihistamine, Corticosteroid - Circulating Tumor Cell, Metastatic Prostate Cancer | Phase 2 | Unknown | Erasmus Medical Center | 140 | 2017-02-21 | 2021-08-20 |
Showing 20 of 38. Sign in to see all of them.
Hereditary Sensory and Autonomic Neuropathies (4) · Tuberous Sclerosis (4) · Bulbo-Spinal Atrophy, X-Linked (3) · Huntington Disease (3) · Rett Syndrome (3) · Spinocerebellar Degenerations (3) · Autistic Disorder (2) · Charcot-Marie-Tooth Disease (2) · Epilepsy (2) · Neurofibromatosis 1 (2) · Optic Atrophy, Hereditary, Leber (2) · Parkinson Disease (2) · Seizures (2) · Spinal Muscular Atrophies of Childhood (2) · Tourette Syndrome (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (38) · Nervous System Diseases (38) · Genetic Diseases, Inborn (37) · Neurodegenerative Diseases (32) · Central Nervous System Diseases (17) · Neuromuscular Diseases (15) · Congenital Abnormalities (13) · Neurologic Manifestations (13) · Brain Diseases (12) · Neoplasms (10) · Nervous System Malformations (10) · Spinal Cord Diseases (10) · Genetic Diseases, X-Linked (9) · Peripheral Nervous System Diseases (9)
Cell- and Tissue-Based Therapy (3) · Everolimus (2) · triheptanoin (2) · Adrenal Cortex Hormones (1) · Anastrozole (1) · Anticonvulsants (1) · Antiemetics (1) · Aromatase Inhibitors (1) · Aspirin (1) · Cyclosporine (1) · Dexamethasone (1) · Dronabinol (1) · Glycerol (1) · Histamine Antagonists (1) · Histamine H2 Antagonists (1)
Cardiff University (2) · Erasmus Medical Center (2) · University Hospital, Angers (2) · Azidus Brasil (1) · Center for Rare Neurological Diseases, Norcross, GA (1) · Centre d'Etude des Cellules Souches (1) · Chaitanya Hospital, Pune (1) · Chang Gung Memorial Hospital (1) · Convalife (Shanghai) Co., Ltd. (1) · General Hospital of Chinese Armed Police Forces (1) · Green Cross Corporation (1) · Guangzhou Jiayin Biotech Ltd (1) · Huazhong University of Science and Technology (1) · Juvantia Pharma Ltd (1) · Massachusetts General Hospital (1)
China (9) · United States (5) · France (4) · United Kingdom (3) · Italy (2) · Netherlands (2) · Austria (1) · Brazil (1) · Finland (1) · Germany (1) · India (1) · Iran (1) · Portugal (1) · South Korea (1) · Spain (1)
Amir Ali Hamidieh, MD (1) · Angelo E Schenone, MD (1) · Bin Li, PhD,MD (1) · Bruria Ben-Zeev, MD (1) · Cheng-Lung Hsu, Profesor (1) · Daniel Tarquinio, DO (1) · Dr. Ryan O Lakin, MD JD (1) · Fei Xu, MD (1) · GuangFu Chen, M.D.,Ph.D (1) · Hua Jiang, M.D,Ph.D (1) · Jason Hsu, MD (1) · Jiacai Zhuo (1) · Joyce Macedo, MD (1)
PMID 11231629, PMID 11399225, PMID 1463007, PMID 15496622, PMID 1674640, PMID 18441370, PMID 18441371, PMID 18774912, PMID 1928099, PMID 19387333, PMID 19892975, PMID 21525172, PMID 22612072, PMID 22644094, PMID 23063736, PMID 23151076, PMID 23845948, PMID 23966770, PMID 24396955, PMID 25950380, PMID 26480849, PMID 26606867, PMID 26892229, PMID 27386292, PMID 27389519, PMID 27426279, PMID 27749593, PMID 29398921, PMID 29454364, PMID 31209394, PMID 31217257, PMID 31237206, PMID 31294749, PMID 31320661, PMID 31434793, PMID 31463890, PMID 3201231, PMID 32165033, PMID 32332722, PMID 33316218, PMID 33607964, PMID 33692216, PMID 34725214, PMID 35020204, PMID 35118423, PMID 35427471, PMID 35660797, PMID 36038003, PMID 36633525, PMID 36657089
2018 (1)