Clinical trials: Peripheral Neuropathy Hereditary
Hereditary Spastic Paraplegia
Disease Natural History and Biomarkers of SPG3A, SPG4A, and SPG31
Background: Hereditary spastic paraplegia (HSP) usually progresses slowly. Researchers want to learn more about how its symptoms change over time.
- Trial name
- NCT02859428
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Breast Cancer
A Single Arm, Phase Ib/II Trial of Single Agent Pacritinib in Patients With 1q21.3 Amplified Solid Tumors Enriching for Interleukin-1 Receptor-associated Kinase 1 Pathway Activation (PAIR)
This is a single arm, open-label, lead in phase Ib dose confirmation, followed by phase II study with 2 parallel study cohorts. Patients will be pre-screened for presence of 1q21.3 copy number amplification in plasma samples…
- Trial name
- NCT04520269
- Testing
- Pacritinib
- Where
- Singapore
- Volunteers
- 74 people
- Sponsor
- National University Hospital, Singapore
Neonatal Brachial Plexus Palsy
Forced Lefthandedness in Neonatal Brachial Plexus Palsy (NBPP) Children
The aim of the project is to assess the neurophysiological and functional consequences of forced lefthandedness in a population with right neonatal brachial plexus palsy (NBPP).
- Trial name
- NBPP (NCT04376372)
- Testing
- Functional testing, Magnetic resonance imaging, Electromyography
- Where
- Belgium
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- University Hospital, Ghent
Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial
To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).
- Conditions
- Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial, Amyloid Cardiomyopathy, Amyloid Cardiomyopathy, Transthyretin-Related
- Trial name
- NCT07116473
- Testing
- Acoramidis (AG10)
- Volunteers
- 250 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
NF1 Mutation, Neurofibroma Plexiform
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1).
- Conditions
- NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)
- Trial name
- NCT06961565
- Testing
- PAS-004 Tablets
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 56 people
- Sponsor
- Pasithea Therapeutics Corp.
Neurofibromatosis 1
Analysis of Plasma for Diagnosis and Follow-up of Neurofibromatosis Type 1
The purpose of this study is to find blood plasma based biomarkers of disease progression in neurofibromatosis type 1 (NF1). NF1 is associated with the development of benign cutaneous tumors as well as a variety of malignancies.
- Trial name
- NCT02680431
- Testing
- Blood sample
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Juha Peltonen
Heart Transplantation, Dilated Cardiomyopathy (DCM)
Multimodal Analysis of Endomyocardial Biopsies
The goal of this observational study is to pursue a multimodal approach to identify the molecular signatures and immune signalling molecules of various myocardial diseases and thereby contribute to improving diagnosis and…
- Conditions
- Heart Transplantation, Dilated Cardiomyopathy (DCM), Hypertrophic Cardiomyopathy (HCM), Myocarditis, Pericarditis
- Trial name
- NCT07359690
- Where
- Germany
- Volunteers
- 216 people
- Sponsor
- University Hospital, Essen
Burns
Determination of Nerve Growth Factor (NGF) Expression in Pediatric Patients With Severe Burns: a Pilot Study
The goal of this observational study is to determine the blood values of NGF in severely burned patients, to describe any changes over time and finally to correlate the expression of NGF to the severity of the burn.
- Trial name
- NCT05751174
- Testing
- pediatric patients affected by severe burns
- Where
- Italy
- Volunteers
- 12 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Leukemia, Juvenile Myelomonocytic, JMML
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML).
- Conditions
- Leukemia, Juvenile Myelomonocytic, JMML, JCML, Neurofibromatosis 1
- Trial name
- NCT05849662
- Testing
- Trametinib, Azacitidine, Fludarabine
- Where
- 21 sites in United States
- Volunteers
- 58 people
- Sponsor
- Therapeutic Advances in Childhood Leukemia Consortium
Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1
Prevalence and Risk Evaluation of Diabetic Complications of the Foot in A Large Canadian Population
The study aims to assess foot complications among patients with diabetes in Canada, using patient data collected during diabetes foot assessments performed by the LMC Chiropody Team between February 27, 2018 and April 17, 2019.
- Conditions
- Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Foot Deformities, Onychauxis
- Trial name
- PEDAL (NCT04327622)
- Where
- Canada
- Volunteers
- 5,084 people
- Sponsor
- LMC Diabetes & Endocrinology Ltd.
Aortic Stenosis Symptomatic, Amyloidosis Cardiac
ATTR Amyloidosis in Elderly Patients With Aortic Stenosis
Severe aortic stenosis is defined with a mean transvalvular pressure gradient (MTPG) > 40mmHg and a calculated aortic valve area of < 1cm2.
- Trial name
- NCT04061213
- Testing
- Tc-99m-DPD scintigraphy
- Where
- Switzerland
- Volunteers
- 489 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Glenohumeral Subluxation, Neonatal Brachial Plexus Palsy
Investigation of the Relationship Between Shoulder Alpha Angle and Humeral Head Displacement Percentage Measured by Ultrasonography in Neonatal Brachial Plexus Palsy Infants With Clinical Parameters
This study will examine infants with a birth-related nerve injury called neonatal brachial plexus palsy, which can affect movement in the arm and sometimes cause the shoulder joint to shift out of its normal position…
- Conditions
- Glenohumeral Subluxation, Neonatal Brachial Plexus Palsy, Brachial Plexus Injuries
- Trial name
- NCT07768904
- Testing
- Shoulder Ultrasonography, Standardized Clinical Examination
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Fatih Sultan Mehmet Training and Research Hospital
Advanced Solid Tumor
A Study to Evaluate ARV-6723 Alone and With Pembrolizumab in Participants With Advanced Solid Tumors
This is a study to evaluate the safety and potential anti-tumor activity of an investigational drug called ARV-6723, in participants with advanced solid tumors.
- Trial name
- NCT07749586
- Testing
- ARV-6723, Pembrolizumab, SOC
- Where
- 4 sites in United States
- Volunteers
- 499 people
- Sponsor
- Arvinas Inc.
Neurofibromatosis 1, NF1
Reliability of Functional Outcome Measures in Neurofibromatosis 1
Neurofibromatosis 1 (NF1) is a common inherited condition that affects the skin, bone and nervous system. The complications of NF1 are widespread and can lead to varied difficulties dependent on the location of neurofibromas and…
- Trial name
- NCT02479360
- Where
- United Kingdom
- Volunteers
- 49 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Heart Failure NYHA Class II, Heart Failure NYHA Class III
CMR T1 Mapping for Diagnosis of Cardiac Amyloidosis
The study aims to test the diagnostic accuracy of T1 mapping for the diagnosis of cardiac amyloidosis prospectively. The hypothesis is that T1 mapping in older patients with symptomatic heart failure, increased LV wall thickness…
- Conditions
- Heart Failure NYHA Class II, Heart Failure NYHA Class III, Heart Failure NYHA Class IV, Heart Failure With Preserved Ejection Fraction
- Trial name
- CMR for CA (NCT04862273)
- Testing
- Native T1 CMR, Web-based ATTR probability estimator (Pfizer, New York), 99mTc-DPD scintigraphy
- Where
- Germany
- Volunteers
- 112 people
- Sponsor
- University of Leipzig
Cardiac Amyloidosis
AI Echocardiographic Screening of Cardiac Amyloidosis
Recent advances in machine learning and image processing techniques have shown that machine learning models can identify features unrecognized by human experts and accurately assess common measurements made in clinical practice.
- Trial name
- NCT06664866
- Testing
- EchoNet-LVH Assessment
- Where
- 4 sites in United States
- Volunteers
- 500 people
- Sponsor
- Cedars-Sinai Medical Center
Amyloid Cardiomyopathy
Unmasking the Prevalence of AC in an Unselected Echocardiographic Population
This study will investigate the prevalence of echocardiographic red-flags of amyloid cardiomyopathy (AC) in patients undergoing clinically-indicated echocardiography (observational phase) and the prevalence of AC among…
- Trial name
- AC-TIVE (NCT04738266)
- Testing
- Amyloid Cardiomyopathy
- Where
- 16 sites in Italy
- Volunteers
- 381 people
- Sponsor
- University of Trieste
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Charcot-Marie-Tooth Disease, Type IA
Biomarkers and Validation of Selected Outcome Measures (CMTBiomarker)
CMT is a rare disease for which novel treatments are being developed. Evaluation of intervention efficacy is hampered by slow progression and lack of sensitive outcome measures.
- Trial name
- NCT03386266
- Where
- Germany
- Volunteers
- 156 people
- Sponsor
- University Medical Center Goettingen
Arthritis, Osteoarthritis
Tanezumab In Osteoarthritis Of The Knee (2)
The purpose of this study is to test the efficacy and safety of 2 doses of tanezumab compared with naproxen and placebo in patients with osteoarthritis.
- Trial name
- NCT00830063
- Testing
- tanezumab 10 mg, tanezumab 5 mg, naproxen
- Where
- 94 sites in United States
- Volunteers
- 832 people
- Sponsor
- Pfizer