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
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=20 IVIg for Small Fiber Neuropathy With Autoantibodies TS-HDS and FGFR3
Small Fiber Neuropathy · Idiopathic Peripheral Neuropathy
Primary: The Change in Nerve Fiber Density Between Visits 1 and 8. — 0.5; 0.6 fibers/mm
N/A N=148 Clinical Phenotyping and Genotyping of HIV-Associated Sensory Neuropathy: The HIV-POGO Study
HIV · Sensory Neuropathy · Neuropathic Pain
Primary: Neuropathic Element of Pain Using the Doleur Neuropathique 4 Interview — 81; 67 Participants
N/A N=10 Autofluorescent Flavoprotein Imaging of Intraepidermal Nerve Fibers: a Pilot Study
Small Fiber Neuropathy
Primary: AFI-intensity After Nociceptive Stimulation — 0.60; 0.25; 0.50 unitless
Phase 2 N=12 Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
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…
Early Phase 1 N=76 Autonomic Neuropathy, GI Motility, and Inflammation in HIV
HIV Disease
Primary: Change in Breath Test — 28; 14 ppm
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=21 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
N/A N=6 Testing Glial Pathways to HAAF in Human Subjects Using Carbon 13 Magnetic Resonance Spectroscopy
Diabetes Complications · Hypoglycemia
Primary: Percent Enrichment of Bicarbonate as Measured by Magnetic Resonance Spectroscopy (MRS) — 61.9 % enrichment — p=0.0053
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)
N/A N=15 Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy
Hereditary Spastic Paraplegia · Adrenomyeloneuropathy
Primary: Change From Baseline Walking Time in 10 Meter Walk Test to the Measurement Taken Directly After rTMS — -5.41; 1.70 seconds
Phase 3 N=44 An Intervention Trial for Cardiac Neuropathy in Type 1 Diabetes
Diabetic Autonomic Neuropathy
Primary: Global [11C]HED Retention Index (RI) — 0.081; 0.073; 0.070; 0.074 Retention index — p=0.32
N/A N=22 Hypoglycemia and Autonomic Nervous System Function
Hypoglycemia
Primary: Change in Baroreflex Sensitivity — 2.3; -1.6 ms/mmHg
Phase 2 N=29 A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
Muscular Atrophy, Spinal
Primary: Safety: Frequency of Adverse Events/Lab Abnormalities
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=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
N/A N=56 The Autonomic Nervous System and the Metabolic Syndrome
Obesity
Primary: Insulin Resistance — 3.8; 3.1 mg/kg/min
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=3 NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome
Menkes Disease · Occipital Horn Syndrome
Primary: Treatment Related Adverse Events as Assessed by CTCAE v4.0 — 10; 6 adverse events
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
Phase 2 N=45 Evaluation of Nicotinamide Riboside in Prevention of Small Fiber Axon Degeneration and Promotion of Nerve Regeneration
Small Fiber Neuropathy
Primary: Denervation of Skin — 0.1; 0.1 percent change — p=0.33
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 2 N=27 A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
Muscular Atrophy, Spinal
Primary: Efficacy: Functional Motor Testing, Including Gross Motor Function Measure (GMFM) and Timed Motor Tests
N/A N=10 Quantitative Assessment of Painful Diabetic Peripheral Neuropathy After High Frequency Spinal Cord Stimulation
Painful Diabetic Neuropathy
Primary: Pain Assessment — 6.5; 1.0 score on a scale
N/A N=53 Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS)
Congenital Sucrase-isomaltase Deficiency (CSID)
Primary: Prevalence of CSID Genetic Variants — 27; 0 Participants
Phase 2 N=6 A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
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
N/A N=161 Symptom Management Efficacy Study to Reduce Distal Neuropathic Pain
Neuropathic Pain · HIV Neuropathy · HIV/AIDS
Primary: Gracely Pain Scale (GPS) — -2.51; -1.96; -2.38; -0.99 GPS pain score on a scale
N/A N=44 Corneal Nerves Function and Structure
Diabetic Peripheral Neuropathy
Primary: Cornea Sensitivity — 0.177; 0.293 time ratio of eyelids closed vs. open — p=<0.001