Clinical trials: Polyneuropathies
Amyloidosis, Amyloid Cardiomyopathy
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause…
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- NCT06563895
- Testing
- Acoramidis
- Where
- 104 sites in United States, Argentina and 22 more
- Volunteers
- 587 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
This is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT05697861
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
Polyneuropathy, Inflammatory Demyelinating, Chronic
Real-world Study of Treatment Outcomes in Chronic Inflammatory Demyelinating Polyneuropathy/Polyradiculoneuropathy (CIDP)
This study is an observational, ambispective, descriptive, non-interventional study of people with a chronic inflammatory demyelinating polyneuropathy/polyradiculoneuropathy (CIDP) diagnosis in the United States with residual…
- Trial name
- ORBIT-CIDP (NCT06968975)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Sanofi
Neuropathic Pain, Distal Sensory Polyneuropathy
Effects of LY3848575 Versus Placebo in Participants With Painful Distal Sensory Polyneuropathy
The purpose of this study is to evaluate how safe and how well a treatment works compared to placebo for people with nerve pain that begins in their feet and moves up the leg to just below the knee.
- Trial name
- NCT06568042
- Testing
- LY3848575
- Where
- 76 sites in United States, Canada and 5 more
- Volunteers
- 558 people
- Sponsor
- Eli Lilly and Company
Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia
This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent…
- Conditions
- Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome, Polyneuropathy, Amyloidosis
- Trial name
- LOCUS (NCT06414746)
- Where
- 13 sites in Russia
- Volunteers
- 721 people
- Sponsor
- AstraZeneca
Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Two Dose Levels of Privigen in Pediatric CIDP
A randomized, open-label, prospective, multicenter study designed to investigate 2 dose levels in pediatric subjects 2 to ≤ 17 years of age with confirmed or possible CIDP, either previously exposed to IVIG treatment or unexposed…
- Trial name
- NCT03684018
- Testing
- IgPro10
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- CSL Behring
Neuromuscular Disease, Neuromuscular Diseases (NMD)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary
- Trial name
- NCT06672237
- Testing
- nexiguran ziclumeran
- Where
- 14 sites in Argentina, Brazil and 4 more
- Volunteers
- 60 people
- Sponsor
- Intellia Therapeutics
Chronic Inflammatory Demyelinating Polyneuropathy
A Study to Assess Adults With CIDP Transitioning From IVIg to Efgartigimod PH20 SC
This study will measure how adults with CIDP receiving IVIg treatment adjust to efgartigimod PH20 SC. The study duration for each participant will be approximately 17 to 19 weeks.
- Trial name
- NCT06637072
- Testing
- Efgartigimod PH20 SC
- Where
- 13 sites in United States
- Volunteers
- 23 people
- Sponsor
- argenx
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), CIDP
Pre-Approval Access for Efgartigimod PH20 SC in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
The aim of the Pre-Approval Access program is to provide efgartigimod PH20 SC treatment to patients with CIDP who are ineligible to participate in a clinical study and have unmet medical need despite available treatment options.
- Trial name
- NCT04777734
- Testing
- Efgartigimod PH20 SC
- Sponsor
- argenx
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
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT04601051
- Testing
- NTLA-2001
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
CIDP - Chronic Inflammatory Demyelinating Polyneuropathy
A First-in-Patient Clinical Trial of KINE-101 in Patients With Corticosteroid-Refractory CIDP
This is a multicenter, open-label, single-dose, dose-escalation study evaluating the safety and tolerability of intravenous (IV) KINE-101 in patients with corticosteroid-refractory chronic inflammatory demyelinating…
- Trial name
- NCT07343310
- Testing
- KINE-101
- Where
- 2 sites in South Korea
- Volunteers
- 9 people
- Sponsor
- Kine Sciences Co., Ltd.
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
- Trial name
- NCT05071300
- Testing
- Eplontersen
- Where
- 33 sites in United States, Argentina and 11 more
- Volunteers
- 151 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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
Chronic Inflammatory Demyelinating Polyneuropathy
An Expanded Access IND for the Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
This is an Individual Patient Expanded Access IND to evaluate the safety and preliminary efficacy of autologous HB-adMSCs for treating a single patient Chronic Inflammatory Demyelinating Polyneuropathy, CIDP.
- Trial name
- NCT04825626
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Transthyretin Amyloid Polyneuropathy
To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy
The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy
- Trial name
- NCT06940336
- Testing
- Tafamidis Meglumine Soft Capsules
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
National, multicenter, epidemiological, longitudinal protocol to investigate the hATTR prevalence in an at-risk population for Hereditary Transthyretin Amyloidosis (hATTR) and subjects diagnosed with hATTR, to monitor the…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Polyneuropathies
- Trial name
- TRAMmoniTTR (NCT03237494)
- Where
- 84 sites in Austria, Germany and 1 more
- Volunteers
- 5,028 people
- Sponsor
- CENTOGENE GmbH Rostock
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)
This is the open-label extension study of phase II ARGX-113-1802 to evaluate the long-term safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.
- Trial name
- ADHERE+ (NCT04280718)
- Testing
- Efgartigimod PH20 SC
- Where
- 147 sites in United States, Austria and 22 more
- Volunteers
- 229 people
- Sponsor
- argenx
Chronic Inflammatory Demyelinating Polyneuropathy, CIDP
Vitaccess Real CIDP Registry
Vitaccess Real CIDP (VRCIDP) is a patient registry designed to capture longitudinal observational data on chronic inflammatory demyelinating polyneuropathy (CIDP), its treatment, and impact on symptoms, daily activities, and…
- Trial name
- VRCIDP (NCT07006597)
- Volunteers
- 300 people
- Sponsor
- Vitaccess 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