Clinical trials: Inherited Neuropathies
- Condition Inherited Neuropathies
- Funder type Industry (pharma, biotech, devices)
- Accepts healthy volunteers yes
Asthma
A Study to Evaluate the Safety, Tolerability and Absorption to the Blood After Administration of Single and Multiple Doses of AZD8154 in Healthy Participants.
This study is a Phase 1, first in human (FiH) study, consisting of 3 parts (Part 1, Part 2, and Part 3) in healthy male and female participants of non-childbearing potential, performed at a single study center.
- Trial name
- NCT03436316
- Testing
- AZD8154
- Where
- Germany
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Breast Cancer
Validation of Molecular Diagnostic Assays to Detect Cancer Biomarkers in Blood & Primary Tumor in ER+/HER2- MBC
Conventional biopsy and surgical tumor resection are invasive procedures that capture only one instance of the progression of the tumor. However, the genome of tumor is not static, but it is constantly altered during treatment.
- Trial name
- NCT04480814
- Testing
- PIK3CA kit
- Where
- 4 sites in Greece
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Pharmassist Ltd
Muscle Spasticity, Hypertonia, Muscle
Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals
To assess the safety and tolerability of single and multiple doses of MTR-601 in normal healthy volunteers under fed and fasted conditions. To evaluate the plasma and urine pharmacokinetics (PK) of MTR-601.
- Conditions
- Muscle Spasticity, Hypertonia, Muscle, Cerebral Palsy, Multiple Sclerosis
- Trial name
- NCT06117020
- Testing
- MTR-601
- Where
- United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Motric Bio
Amyloid Cardiomyopathy, Transthyretin-Related
Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
- Trial name
- NCT03294707
- Testing
- AG10 oral tablet
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Healthy
Study to Evaluate Safety, Tolerability and Pharmacokinetics of rhNGF Eye Drops in Healthy Volunteers
The primary objective of this study is to assess the safety and tolerability of single and multiple ascending doses of rhNGF when administered as eye drops in healthy subjects.
- Trial name
- NGF0112 (NCT01744704)
- Testing
- rhNGF 0.5 µg/mL Sentinel, rhNGF 5 µg/mL Sentinel, rhNGF 20 µg/mL Sentinel
- Where
- 2 sites in Switzerland, United Kingdom
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Dompé Farmaceutici S.p.A
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Study of Electrical Impedance Myography (EIM) in ALS
This trial is studying Electrical Impedance Myography (EIM) for measuring muscle health. The trial is studying people with Amyotrophic Lateral Sclerosis (ALS), other neuromuscular diseases, and healthy volunteers to see if the…
- Conditions
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
- Trial name
- NCT02011204
- Testing
- Electrical impedance myography (EIM)
- Where
- 6 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Skulpt, Inc.
Retinitis Pigmentosa, Macula Off
Transcorneal Electrical Stimulation Therapy for Retinal Disease
Transcorneal stimulation may enable neurons to survive degeneration processes via enhanced secretion of neurotrophic substances and direct stimulation of neurons.
- Conditions
- Retinitis Pigmentosa, Macula Off, Primary Open Angle Glaucoma, Hereditary Macular Degeneration
- Trial name
- NCT00804102
- Testing
- Transcorneal Electrical Stimulation, DTL-electrode attached without energy
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Okuvision GmbH
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
- Trial name
- NCT02797847
- Testing
- ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Charcot-Marie-Tooth Disease
Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls
The primary aim of this study is to discover whether patients with CMT type 1 and 2 have neuromuscular junction transmission deficits.
- Trial name
- ESTABLISH (NCT04980807)
- Where
- 2 sites in United States, Denmark
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- NMD Pharma A/S
Skin Cancer, Skin Diseases
Multi-modal Quantitative Imaging of the Skin
This clinical trial is about evaluating the suitability of multi-modal imaging technology, which covers virtually the entire body, to evaluate the possibility of screening and early detection of disease.
- Conditions
- Skin Cancer, Skin Diseases, Atopic Dermatitis, Psoriasis
- Trial name
- DermaFlow (NCT06989216)
- Where
- 3 sites in Sweden
- Volunteers
- 8,000 people, healthy volunteers welcome
- Sponsor
- HJN Sverige AB/Neko Health
Familial Amyloid Polyneuropathy (FAP)
Study of SOM0226 in Familial Amyloid Polyneuropathy
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
- Trial name
- NCT02191826
- Testing
- SOM0226
- Where
- Spain
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- SOM Innovation Biotech SA
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS
Healthy Volunteers
A Study to Determine the Relative Bioavailability of Capsule and Tablet Formulations of GDC-0941 and the Effect of Food and Proton Pump Inhibition on GDC-0941 Tablet Pharmacokinetics in Healthy Volunteers
This is a Phase I, randomized, two-part, single-center pharmacokinetics (PK) study to assess the relative bioavailability of capsule and tablet formulations of GDC-0941 in the fasted state (Part 1) and to determine the effect of…
- Trial name
- NCT00999128
- Testing
- GDC-0941, rabeprazole
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Genentech, Inc.