Clinical trials: Peripheral Nerve Disorder
- Condition Peripheral Nerve Disorder
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Hereditary Transthyretin Amyloidosis
hATTR Polyneuropathy in Russia
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There…
- Trial name
- PRIMER (NCT06365593)
- Where
- 3 sites in Russia
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
PIK3CA-Related Overgrowth Spectrum
A Study of the Epidemiology and Hospital Management of Patients With PROS in France
The purpose of this study was to estimate the incidence and prevalence of a group of genetic disorders known as PIK3CA-Related Overgrowth Spectrum (PROS) in France.
- Trial name
- EpiPROS (NCT07222423)
- Where
- United States
- Volunteers
- 3,605 people
- Sponsor
- Novartis Pharmaceuticals
Alström Syndrome
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with…
- Trial name
- NCT03184584
- Testing
- PBI-4050
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Liminal BioSciences Ltd.
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory…
- Trial name
- CMT4J (NCT03810508)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Neurogene Inc.
Peripheral Nerve Injuries
Continuous Observational Monitoring Program for Allay™ Hydrogel Cap Safety and Success Study
is is a multi-center, open-label, observational registry study (both prospective and retrospective) designed to evaluate the safety and performance of the allay™ Hydrogel Cap.
- Trial name
- COMPASS (NCT07246980)
- Testing
- Nerve Cap
- Where
- 5 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Tulavi Therapeutics,Inc.
Carcinoma, Midline
A Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of GSK525762 in Subjects With NUT Midline Carcinoma (NMC) and Other Cancers
This study is divided into two parts; Part 1 of the study is a dose escalation phase to select the recommended dose for Part 2 based on the safety, pharmacokinetic, and pharmacodynamic profiles observed after oral administration…
- Trial name
- NCT01587703
- Testing
- GSK525762
- Where
- 17 sites in United States, Australia and 5 more
- Volunteers
- 196 people
- Sponsor
- GlaxoSmithKline
Ewing's Sarcoma/Soft Tissue Sarcoma, Neuroblastoma
A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone
This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages: Stage 1 is a dose-escalation study.
- Conditions
- Ewing's Sarcoma/Soft Tissue Sarcoma, Neuroblastoma, Brain Tumors
- Trial name
- NCT01288573
- Testing
- plerixafor
- Where
- 27 sites in Belgium, Czechia and 10 more
- Volunteers
- 46 people
- Sponsor
- Genzyme, a Sanofi Company
Cerebral Palsy (CP), Spastic Paraplegia
Retrospective Study on the WalkOn AFO in Children or Teenagers With Neurological Pathologies
The goal of this observational study is to assess the interest of prefabricated carbon fiber ankle foot orthoses (AFO) in children and teenagers with central or peripheral neurological pathologies.
- Conditions
- Cerebral Palsy (CP), Spastic Paraplegia, Spinal Cord Injuries (SCI), Spina Bifida
- Trial name
- NCT06740006
- Where
- France
- Volunteers
- 65 people
- Sponsor
- Otto Bock France SNC
Neuroblastoma
Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow
Children and adults diagnosed with high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and/or bone marrow will be treated for up to 101 weeks with naxitamab and…
- Trial name
- NCT03363373
- Testing
- GM-CSF + Naxitamab
- Where
- 20 sites in United States, Canada and 7 more
- Volunteers
- 122 people
- Sponsor
- Y-mAbs Therapeutics
NF type1 With Inoperable Plexiform Neurofibromas
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable…
- Trial name
- NCT03259633
- Testing
- Selumetinib
- Where
- United States
- Sponsor
- AstraZeneca
Neuroblastoma in Children, Neuroblastoma, Recurrent, Refractory
Expanded Access Protocol for the Use of SL-28 in the Treatment of Advanced Solid Tumors
This is an Expanded Access Program (EAP) that will give the participants access to the drug SL-28 before it is approved by the FDA.
- Conditions
- Neuroblastoma in Children, Neuroblastoma, Recurrent, Refractory, Advanced Cancer, Advanced Malignant Neoplasm
- Trial name
- NCT06872489
- Testing
- SL-28.
- Sponsor
- Second Life Therapeutics
Neuroblastoma, Ewing's Sarcoma
Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
This study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy.
- Conditions
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor, Retinoblastoma
- Trial name
- NCT01598454
- Testing
- racotumomab
- Where
- Argentina
- Volunteers
- 15 people
- Sponsor
- Laboratorio Elea Phoenix S.A.
Neurofibromatosis 1, Plexiform Neurofibroma
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT04954001
- Testing
- FCN-159
- Where
- 14 sites in United States, China and 1 more
- Volunteers
- 160 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)
This is a study of the safety and tolerability of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA)-related…
- Trial name
- NCT04980872
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 60 people
- Sponsor
- Merck Sharp & Dohme LLC
Pheochromocytoma, Neuroblastoma
Meta-Iodobenzylguanidine (123I mIBG) Scintigraphy in Patients Being Evaluated for Phaeochromocytoma or Neuroblastoma
The study is designed to study the effectiveness of 123I-mIBG as a diagnostic imaging agent in evaluating patients with known or suspected neuroblastoma or phaeochromocytoma.
- Trial name
- NCT00126412
- Testing
- 123I-mIBG (meta-iodobenzylguanidine)
- Where
- United States
- Volunteers
- 251 people
- Sponsor
- GE Healthcare
Pain, Pain, Acute
Assessing Opioid Care Practices Using CPV Patient Simulation Modules
This study will test the quality of opioid-related physician care decisions using a patient-simulation based measurement and feedback approach that combines multiple-choice care decisions with real-time, personalized scoring and…
- Conditions
- Pain, Pain, Acute, Pain, Chronic, Pain, Neuropathic
- Trial name
- NCT04080037
- Testing
- Quality IQ Patient Simulations
- Where
- United States
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- Qure Healthcare, LLC
Brain and Central Nervous System Tumors, Neuroblastoma
Radiolabeled MAB Therapy in Patients With Refractory, Recurrent, or Advanced CNS or Leptomeningeal Cancer
The purpose of this study is to test the feasibility and toxicity of administering intrathecal immunotherapy for patients with central nervous system/leptomeningeal (CNS/LM) malignancies.
- Conditions
- Brain and Central Nervous System Tumors, Neuroblastoma, Sarcoma
- Trial name
- NCT00089245
- Testing
- Iodine I 131 MOAB 8H9
- Where
- United States
- Volunteers
- 177 people
- Sponsor
- Y-mAbs Therapeutics
Medulloblastoma, Rhabdomyosarcoma
A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other…
- Conditions
- Medulloblastoma, Rhabdomyosarcoma, Neuroblastoma, Hepatoblastoma
- Trial name
- NCT01125800
- Testing
- LDE225
- Where
- 14 sites in United States, Australia and 4 more
- Volunteers
- 76 people
- Sponsor
- Novartis Pharmaceuticals