Phase 2 N=4 Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Charcot Marie Tooth Disease
Primary: Number of Participants With a Decrease in Cramp Duration — 2; 0 participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=80 Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
Charcot-Marie-Tooth Disease · Hereditary Neuropathy With Liability to Pressure Palsies · Genetic Disorders
Primary: Safety and Tolerability of PXT3003 — 21; 21; 19; 19 Participants
Phase 2 N=12 The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
CMT
Primary: Safety of Intramuscular (IM) Injections of Engensis in Participants — 1; 1; 1; 1 Participants
Phase 3 N=323 Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
Charcot-Marie-Tooth Disease Type 1A
Primary: Overall Neuropathy Limitation Scale (ONLS) Total Score — 3.33; 3.05; 3.23; 3.25 Scores on the ONLS — p=0.008
Phase 2 N=18 L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale
Phase 1 N=15 Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
Spinal Muscular Atrophy 1
Primary: Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical…
Phase 2 N=38 CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Spinal Muscular Atrophy Type I
Primary: Laboratory Safety Data
Phase 2 N=14 MC-5A for Chemotherapy Induced Peripheral Neuropathy
Neuropathy, Paraneoplastic
Primary: Change in Mechanical Visual Analog Scale (mVAS) Using Quantitative Sensory Pain Testing (QSPT) — 5.14; -2.00; 1.25; 6.76 Millimeters
Phase 2 N=132 Topiramate for Cryptogenic Sensory Peripheral Neuropathy in Metabolic Syndrome (CSPN)
Cryptogenic Sensory Peripheral Neuropathy in Metabolic Syndrome
Primary: Intraepidermal Nerve Fiber Density (IENFD) — -0.6064; -0.396 fibers/mm/year (52 weeks)
Phase 3 N=61 Targeting Inflammation With Salsalate in Type 1 Diabetes Neuropathy
Type 1 Diabetes · Peripheral Neuropathy
Primary: Skin Biopsy - Intraepidermal Nerve Fiber Density (IENFD) - Distal Thigh — 12; 14 Percentage change in IENFD
Phase 3 N=22 Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
SMA - Spinal Muscular Atrophy · Gene Therapy
Primary: Achievement of Independent Sitting for at Least 30 Seconds — 13 Participants — p=<0.0001
Phase 2 N=69 Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1
Primary: Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance — 12.40; 20.11 meters
Phase 3 N=33 Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
SMA
Primary: Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds — 14 Participants
N/A N=44 Reversing Diabetic Peripheral Neuropathy Through Exercise
Peripheral Neuropathy · Diabetes
Primary: Body Mass Index (BMI) — 30.7 kg/m^2
N/A N=41 Neurogenic Inflammation in Diabetes
Diabetes Mellitus · Polyneuropathies · Arthropathy, Neurogenic
Primary: Induration Size as a Response to Intracutaneous Candida Albicans. — 4; 6; 5; 4 millimeters — p=0.84
Phase 2 N=45 Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
GLUT1DS1 · Epilepsy · Glut1 Deficiency Syndrome 1, Autosomal Recessive
Primary: Neuropsychological Score of Sustained Attention — 11 Participants
Phase 4 N=402 Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations
Cryptogenic Sensory Polyneuropathy
Primary: Co-Primary Measures: Percent of Patients With at Least a 50% Decrease in Likert Pain Scale From Baseline to Week 12 Follow Up and Percent of Patients That Quit — 34; 29…
Phase 2 N=42 Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
Myotonic Dystrophy
Primary: Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance — 17.44; 7.25 Meters
Phase 4 N=12 Growth and Development Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm
Phase 3 N=2 Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Spinal Muscular Atrophy Type I
Primary: Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds — 1 Participants
Phase 3 N=130 The Effect of Diflunisal on Familial Amyloidosis
Familial Amyloid Polyneuropathy · Familial Amyloidosis
Primary: Neurologic Impairment Score + 7 (NIS+7) — 8.2; 26.3; 6.2; 12.5 units on a scale — p=<0.001
Phase 2 N=8 Pilot Study to Determine Effects of Salsalate in Type 1 Diabetes and Painful Peripheral Neuropathy
Peripheral Neuropathy · Type 1 Diabetes
Primary: Intra-epidermal Nerve Fiber Density (IENFD) Fibers Per mm — 6.3; 3.0; 5.2; 3.0 fibers per mm