Clinical trials: Polyneuropathies
- Condition Polyneuropathies
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)
This is an open label, Phase 1/2 study 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 Overgrowth…
- Trial name
- MOSAIC (NCT03094832)
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 50 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Chronic Inflammatory Demyelinating Polyneuropathy
Evaluating the Effectiveness of Telemonitoring System in the Management of Patients With CIDP
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a chronic demyelinating polyneuropathy of autoimmune origin with a progressive or relapsing course.
- Trial name
- HELIPAD 1 (NCT03779828)
- Testing
- HOME LINK
- Where
- 10 sites in France
- Volunteers
- 11 people
- Sponsor
- CSL Behring
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
Guillain-Barre Syndrome
An Open Label Clinical Study to Evaluate Tanruprubart (Also Commonly Known as ANX005) in Participants With Guillain-Barré Syndrome (FORWARD Study)
The goal of this open label study is to measure pharmacokinetics, pharmacodynamics, early efficacy, and safety of tanruprubart in adult and pediatric participants, in the United States, Canada, and Europe.
- Trial name
- NCT07020819
- Testing
- Tanruprubart
- Where
- 30 sites in United States, Denmark and 3 more
- Volunteers
- 30 people
- Sponsor
- Annexon, Inc.
Transthyretin Familial Amyloid Polyneuropathy
Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions [ADRs]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received…
- Trial name
- NCT02146378
- Testing
- Vyndaqel
- Volunteers
- 500 people
- Sponsor
- Pfizer
PIK3CA-Related Overgrowth Spectrum (PROS)
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the…
- Trial name
- EPIK-P1 (NCT04285723)
- Testing
- alpelisib
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 57 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.
Tumor, Tumor, Solid
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
- Conditions
- Tumor, Tumor, Solid, Metastasis, Metastatic Cancer
- Trial name
- NCT05103358
- Testing
- nab-sirolimus
- Where
- 166 sites in United States, Puerto Rico and 1 more
- Volunteers
- 128 people
- Sponsor
- Aadi Bioscience, Inc.
Myasthenia Gravis, CIDP - Chronic Inflammatory Demyelinating Polyneuropathy
A Worldwide Pregnancy Safety Study to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to Efgartigimod During Pregnancy and/or Breastfeeding.
This is a multi-country, prospective safety study of pregnant women exposed to efgartigimod or efgartigimod PH20 SC any time within 25 days prior to conception or any time during pregnancy.
- Trial name
- NCT06299748
- Testing
- Efgartigimod
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 279 people
- Sponsor
- argenx
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Guillain-Barre Syndrome
Efficacy and Safety of ANX005 in Subjects With Guillain-Barré Syndrome
This study is intended to evaluate the efficacy and safety of ANX005 administered by intravenous (IV) infusion to participants recently diagnosed with Guillain-Barré Syndrome (GBS).
- Trial name
- NCT04701164
- Testing
- ANX005
- Where
- 11 sites in Bangladesh, Philippines
- Volunteers
- 242 people
- Sponsor
- Annexon, Inc.
Pediatric Chronic Inflammatory Demyelinating Polyneuropathy
Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
- Trial name
- NCT04929236
- Testing
- Panzyga
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Octapharma
Polyneuropathies
Individual Patient Expanded Access IND to Treat Polyneuropathy
This Individual Patient Expanded Access IND has been created as requested by an 58-year-old man who suffers from Polyneuropathy due to Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal gammopathy, and Skin abnormalities…
- Trial name
- NCT04064983
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
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
Guillain-Barré Syndrome
Efficacy and Safety Study of GB-0998 for Guillain-Barré Syndrome
This study will carry out to assess the efficacy of GB-0998 (intravenous immunoglobulin;400mg/kg/day for five days) in the treatment of the Guillain-Barré Syndrome based on the changes in Hughes Functional Grade (FG) as primary…
- Trial name
- NCT02342184
- Testing
- GB-0998
- Where
- Japan
- Volunteers
- 21 people
- Sponsor
- Japan Blood Products Organization
Invasive Group B Streptococcus Disease, Group B Streptococcus
Comprehensive Outcomes Monitoring for Peri- and Postnatal Invasive Group B Strep Sequelae (COMPPASS) Registry
The goal of this registry is to collect and store important medical and non-health information on children from 0 to 18 years of age affected by early- (up to 24 hours after birth) and late-onset (up to 90 days after birth)…
- Conditions
- Invasive Group B Streptococcus Disease, Group B Streptococcus, Group B Streptococcal Infection, Group B Streptococcus (GBS) Disease
- Trial name
- COMPPASS (NCT06896526)
- Volunteers
- 10,000 people
- Sponsor
- Precia Group
Advanced Stage Solid Tumors
ATRi Transition Rollover Study
The main purpose of the study was to monitor the safety of participant receiving long-term treatment of Berzosertib as monotherapy.
- Trial name
- NCT03309150
- Testing
- Berzosertib
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Prostatic Neoplasms
Prevalence of HRR-related Genes Mutations and Prognosis in Metastatic Castration Resistant Prostate Cancer (mCRPC) Patients in Real World Setting
The purpose of this study is to investigate the prevalence of tissue homologous recombination repair (HRR)-related gene mutations (positive/negative/Variant of uncertain significance (VUS)), clinical outcome such as…
- Trial name
- ZENSHIN (NCT04425200)
- Where
- 26 sites in Japan
- Volunteers
- 205 people
- Sponsor
- AstraZeneca
Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHMBP2, when administered by intrathecal injection.
- Trial name
- NCT07223632
- Testing
- VCA-894A
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Vanda Pharmaceuticals