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ClinicalTrials.gov public records Last synced Oct 17, 2026, 11:52 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 121–144 of 270 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Neuropathies Clear all
Active, not recruiting Phase 2 Interventional

9-ING-41 in Patients With Advanced Cancers

NCT03678883
View directory record Official record
Conditions
Cancer, Pancreatic Cancer, Sarcoma, Renal Cancer, Refractory Cancer, Refractory Neoplasm, Refractory Non-Hodgkin Lymphoma, Pancreatic Adenocarcinoma, Resistant Cancer, Neoplasm Metastasis, Neoplasm of Bone, Neoplasm, Breast, Neoplasm of Lung, Neoplasms,Colorectal, Neoplasms Pancreatic, Malignant Glioma, Malignancies, Malignancies Multiple, Bone Metastases, Bone Neoplasm, Bone Cancer, Pancreas Cancer, Pancreatic Neoplasms, Breast Neoplasms, Acute T Cell Leukemia Lymphoma
Interventions
9-ING-41, Gemcitabine - 21 day cycle, Doxorubicin., Lomustine, Carboplatin., Nab paclitaxel., Paclitaxel., Gemcitabine - 28 day cycle, Irinotecan
Drug
Lead sponsor
Actuate Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
40
States / cities
Phoenix, Arizona • Tucson, Arizona • Orange, California + 34 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study of the Experimental Drug BKM120 With Paclitaxel in Patients With HER2 Negative, Locally Advanced or Metastatic Breast Cancer, With or Without PI3K Activation

NCT01572727
View directory record Official record
Conditions
Breast Cancer
Interventions
Paclitaxel, BKM120 matching placebo, BKM120
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Female only
Enrollment
416 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
21
States / cities
Chandler, Arizona • Phoenix, Arizona • Fayetteville, Arkansas + 17 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2017 · Synced Oct 17, 2026, 11:52 AM EDT
Terminated Phase 1 Interventional Results available

Safety, Tolerability and Pharmacokinetics of Cobimetinib in Combination With Pictilisib in Patients With Locally Advanced or Metastatic Solid Tumors

NCT00996892
View directory record Official record
Conditions
Solid Tumors
Interventions
Cobimetinib, Pictilisib
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
18 Years and older
Enrollment
178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
6
States / cities
Baltimore, Maryland • Boston, Massachusetts • Detroit, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 30, 2016 · Synced Oct 17, 2026, 11:52 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

NCT02532244
View directory record Official record
Conditions
Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
Interventions
sample collection
Other
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
1 Month and older
Enrollment
230 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2024
U.S. locations
3
States / cities
Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 3 Interventional Results available

RESCUE and REVERSE Long-term Follow-up

NCT03406104
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Sham Intravitreal Injection
Genetic · Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Pasadena, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 1 Interventional

An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease

NCT00357786
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal agalsidase alfa, Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
39 Years to 45 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 17, 2011 · Synced Oct 17, 2026, 11:52 AM EDT
Active, not recruiting Phase 1 Interventional

A Phase I Study, Evaluating the Safety, Pharmacokinetics and Efficacy of PRJ1-3024 in Subjects With Advanced Solid Tumors

NCT05159700
View directory record Official record
Conditions
Advanced Solid Tumor, Advanced Solid Malignancies
Interventions
PRJ1-3024
Drug
Lead sponsor
Zhuhai Yufan Biotechnologies Co., Ltd
Industry
Eligibility
18 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
6
States / cities
Denver, Colorado • Orlando, Florida • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 17, 2026, 11:52 AM EDT
Recruiting No phase listed Observational

Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)

NCT06669949
View directory record Official record
Conditions
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 1Phase 2 Interventional

RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

NCT00457964
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
RAD001
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 25, 2013 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 2 Interventional

Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease

NCT00876863
View directory record Official record
Conditions
Alzheimer's Disease
Interventions
CERE-110: Adeno-Associated Virus injection Surgery, Placebo Surgery
Drug · Procedure
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
55 Years to 80 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Oct 17, 2026, 11:52 AM EDT
Terminated Phase 2 Interventional Results available

Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities

NCT04572893
View directory record Official record
Conditions
Primary Familial Dilated Cardiomyopathy
Interventions
danicamtiv
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years to 80 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
12
States / cities
La Jolla, California • Washington D.C., District of Columbia • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated May 21, 2025 · Synced Oct 17, 2026, 11:52 AM EDT
Recruiting Not applicable Interventional

Neuromodulation to Enhance Motor Function in HSP

NCT07417943
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
transcutaneous spinal cord stimulation
Device
Lead sponsor
Rahul Sachdeva
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 3 Interventional Results available

Glutamic Acid in Reducing Nerve Damage Caused by Vincristine in Young Patients With Cancer

NCT00369564
View directory record Official record
Conditions
Kidney Cancer, Leukemia, Lymphoma, Neurotoxicity, Peripheral Neuropathy, Sarcoma
Interventions
glutamic acid, placebo
Drug · Other
Lead sponsor
University of South Florida
Other
Eligibility
3 Years to 20 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
6
States / cities
Fort Myers, Florida • Grand Rapids, Michigan • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2021 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

NCT00411619
View directory record Official record
Conditions
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Interventions
Everolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 20, 2014 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 1 Interventional

A Study to Assess the Safety and Tolerability of AZD1390 Given With Radiation Therapy in Patients With Brain Cancer

NCT03423628
View directory record Official record
Conditions
Recurrent Glioblastoma Multiforme, Primary Glioblastoma Multiforme, Brain Neoplasms, Malignant, Leptomeningeal Disease (LMD)
Interventions
Radiation Therapy, AZD1390
Radiation · Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
5
States / cities
Boston, Massachusetts • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Completed No phase listed Observational

Biomarker Testing and Treatment Patterns Among Patients With PIK3CA Mutation in Advanced Breast Cancer

NCT05982886
View directory record Official record
Conditions
Breast Cancer
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
1,093 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Aug 9, 2023 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan

NCT02985710
View directory record Official record
Conditions
Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
Interventions
Sudoscan, Skin biopsy, QSART
Device · Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
8 Years to 70 Years
Enrollment
102 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 7, 2021 · Synced Oct 17, 2026, 11:52 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors

NCT04657068
View directory record Official record
Conditions
Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer, Endometrial Cancer, Metastatic Colorectal Cancer, Pancreatic Ductal Adenocarcinoma, Acinar Cell Carcinoma
Interventions
ART0380, Gemcitabine, Irinotecan
Drug
Lead sponsor
Artios Pharma Ltd
Industry
Eligibility
18 Years and older
Enrollment
542 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
41
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 33 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)

NCT05103358
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Not listed Phase 3 Interventional

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

NCT04762758
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
(RS)-baclofen, naltrexone hydrochloride and D-sorbitol, Placebo
Drug
Lead sponsor
Pharnext S.C.A.
Other
Eligibility
16 Years to 65 Years
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
20
States / cities
Los Angeles, California • Sacramento, California • New Britain, Connecticut + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 17, 2026, 11:52 AM EDT
Completed Phase 3 Interventional

REVEAL - Research With I-124 EVuzamitide to Elucidate Cardiac AmyLoidosis

NCT06788535
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
I-124 evuzamitide
Drug
Lead sponsor
Sharmila Dorbala
Other
Eligibility
18 Years and older
Enrollment
204 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
20
States / cities
Scottsdale, Arizona • Duarte, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy

NCT07750678
View directory record Official record
Conditions
Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
Interventions
GEB-101 Injection
Genetic
Lead sponsor
GenEditBio Limited
Industry
Eligibility
18 Years to 70 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Ultrasound Therapy In Cardiac Amyloidosis

NCT04667494
View directory record Official record
Conditions
Amyloidosis Cardiac
Interventions
Sonotherapy
Combination Product
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 17, 2026, 11:52 AM EDT
No Longer Available No phase listed Expanded access

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

NCT02300753
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
EPI-743
Drug
Lead sponsor
Edison Pharmaceuticals Inc
Industry
Eligibility
8 Years to 65 Years
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 25, 2014 · Synced Oct 17, 2026, 11:52 AM EDT