18 unknown. 18 Phase 3, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 18 | Unknown 18 |
| Phase 2 | 8 | Unknown 8 |
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 |
|---|---|---|---|---|---|---|
| Efficacy and Safety of NestaCell® in Huntington's Disease NestaCell® - Huntington Disease | Phase 3 | Unknown | Azidus Brasil | 120 | 2024-06-08 | 2023-10-24 |
| 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 |
| Home-based Training and Supplementation in DM1 Patients Mult-ingredient supplement, Placebo, Concurrent exercise training - Myotonic Dystrophy 1 | Phase 3 | Unknown | McMaster University | 60 | 2024-01-01 | 2023-10-19 |
| 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 |
| Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (RS)-baclofen, naltrexone hydrochloride and D-sorbitol, Placebo - Charcot-Marie-Tooth Disease | Phase 3 | Unknown | Pharnext S.C.A. | 350 | 2021-03-30 | 2024-04-03 |
| 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 |
| 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 |
| Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life Sirolimus - Vascular Malformations | Phase 3 | Unknown | Radboud University Medical Center | 75 | 2017-09-18 | 2021-01-29 |
| Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A PXT3003 - Charcot-Marie-Tooth Disease, Type IA | Phase 3 | Unknown | Pharnext S.C.A. | 187 | 2017-03-07 | 2024-02-20 |
| Intravenous Immunoglobulin for Unverricht-Lundborg Disease. Intravenous immunoglobulin - Unverricht-Lundborg Disease | Phase 3 | Unknown | Azienda Socio Sanitaria Territoriale di Mantova | 1 | 2015-12-09 | 2017-11-27 |
| Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease Sirolimus, Placebo - Polycystic Kidney, Type 1 Autosomal Dominant Disease, Polycystic Kidney, Type 2 Autosomal Dominant Disease | Phase 3 | Unknown | Medical University of Vienna | 68 | 2014-04-01 | 2018-10-31 |
| OnabotulinumtoxinA (onaBoNT-A) Versus Oral Oxybutynin ER onaBoNT-A, Oxybutynin ER - Overactive Bladder | Phase 3 | Unknown | Christopher Patrick Smith | 36 | 2013-08-01 | 2016-08-30 |
| Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex Everolimus, Placebo - Tuberous Sclerosis Complex, TSC Related Cognitive Disability | Phase 2 / Phase 3 | Unknown | Erasmus Medical Center | 60 | 2012-11-01 | 2015-05-05 |
| Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A) TreSPE, SPE - Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A | Phase 2 / Phase 3 | Unknown | University of Genova | 92 | 2011-02-01 | 2011-02-04 |
| Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103) RP103, Placebo - Huntington's Disease | Phase 2 / Phase 3 | Unknown | University Hospital, Angers | 96 | 2010-10-01 | 2014-04-02 |
| Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive Bilevel pressure ventilator - Myopathy, Muscular Weakness | Phase 3 | Unknown | Assistance Publique - Hôpitaux de Paris | 77 | 2010-10-01 | 2017-11-20 |
| First Clinical Study of Erbium - Yttrium Aluminium Garnet (YAG) Laser Vaporization of Cutaneous Neurofibromas Erbium-YAG laser vaporization - Cutaneous Neurofibromas | Phase 2 / Phase 3 | Unknown | Medical University of Vienna | 12 | 2006-04-01 | 2011-07-13 |
| Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome dextromethorphan, donepezil hydrochloride - Rett Syndrome | Phase 3 | Unknown | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 90 | 2004-09-01 | 2005-06-24 |
Hereditary Sensory and Autonomic Neuropathies (5) · Charcot-Marie-Tooth Disease (3) · Huntington Disease (3) · Myotonic Dystrophy (2) · Peripheral Nervous System Diseases (2) · Autistic Disorder (1) · Intellectual Disability (1) · Muscle Weakness (1) · Muscular Diseases (1) · Myoclonic Epilepsies, Progressive (1) · Neurofibromatosis 1 (1) · Optic Atrophy, Hereditary, Leber (1) · Polycystic Kidney Diseases (1) · Polycystic Kidney, Autosomal Dominant (1) · Respiratory Insufficiency (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Genetic Diseases, Inborn (18) · Nervous System Diseases (18) · Neurodegenerative Diseases (17) · Neuromuscular Diseases (11) · Congenital Abnormalities (9) · Nervous System Malformations (9) · Polyneuropathies (8) · Peripheral Nervous System Diseases (7) · Central Nervous System Diseases (5) · Brain Diseases (4) · Mental Disorders (4) · Basal Ganglia Diseases (3) · Chorea (3)
Sirolimus (2) · Botulinum Toxins, Type A (1) · Cell- and Tissue-Based Therapy (1) · Dextromethorphan (1) · Donepezil (1) · Everolimus (1) · Glycerol (1) · Hypromellose Derivatives (1) · Immunoglobulins, Intravenous (1) · Naltrexone (1) · Ophthalmic Solutions (1) · Polyethylene Glycols (1) · Sorbitol (1) · Tolterodine Tartrate (1) · netarsudil (1)
Azidus Brasil (2) · Medical University of Vienna (2) · Pharnext S.C.A. (2) · Assistance Publique - Hôpitaux de Paris (1) · Azienda Socio Sanitaria Territoriale di Mantova (1) · Christopher Patrick Smith (1) · Convalife (Shanghai) Co., Ltd. (1) · Erasmus Medical Center (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Huazhong University of Science and Technology (1) · McMaster University (1) · Radboud University Medical Center (1) · University Hospital, Angers (1) · University of Genova (1) · Wills Eye (1)
United States (5) · France (4) · Canada (3) · Netherlands (3) · Austria (2) · Belgium (2) · China (2) · Italy (2) · Spain (2) · Brazil (1) · Denmark (1) · Germany (1) · Israel (1) · United Kingdom (1)
Angelo E Schenone, MD (1) · Bin Li, PhD,MD (1) · Christopher P. Smith, MD (1) · Florian Thomas, MD PhD (1) · Gere Sunder-Plassmann, MD (1) · Jason Hsu, MD (1) · Joyce Macedo, MD (1) · Luciana Ferrara, Doctor (1) · Marianne de Visser, MD (1) · Mario Saporta, MD (1) · Mark Roberts, MD (1) · Markus Riegersperger, MD (1) · Maroeska Loo, te, Dr. (1)
PMID 1463007, PMID 1674640, PMID 18441370, PMID 18441371, PMID 18774912, PMID 1928099, PMID 22612072, PMID 22644094, PMID 23063736, PMID 24396955, PMID 25899445, PMID 25950380, PMID 26606867, PMID 26892229, PMID 27386292, PMID 27426279, PMID 27749593, PMID 31217257, PMID 31237206, PMID 3201231, PMID 37153086, PMID 39356039, PMID 42568743, PMID 5063796
2015 (1), 2016 (1), 2017 (1), P01HD024448 (1)