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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hereditary Sensory and Autonomic Neuropathies Clear all
Recruiting Phase 2 Interventional

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)

NCT07175038
View directory record Official record
Conditions
Pulmonary Arterial Hypertension (PAH), Pulmonary Hypertension, Interstitial Lung Disease
Interventions
ROC-101
Drug
Lead sponsor
AllRock Bio, Inc.
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
10
States / cities
Phoenix, Arizona • Rancho Cordova, California • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Recruiting No phase listed Observational

The Natural History of Familial Dysautonomia

NCT03920774
View directory record Official record
Conditions
Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies, Hereditary Sensory and Autonomic Neuropathy 3
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
4 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Jul 23, 2026, 12:52 PM EDT
Terminated Phase 2 Interventional Results available

Javelin BRCA/ATM: Avelumab Plus Talazoparib in Patients With BRCA or ATM Mutant Solid Tumors

NCT03565991
View directory record Official record
Conditions
Locally Advanced or Metastatic Solid Tumors, Genes, BRCA 1
Interventions
Avelumab, Talazoparib
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
72
States / cities
Palo Alto, California • Stanford, California • Alpharetta, Georgia + 35 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2023 · Synced Jul 23, 2026, 12:52 PM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate the Safety and Tolerability of TOS-358 in Women With HR+ HER2- Breast Cancer

NCT05683418
View directory record Official record
Conditions
HR+/HER2-negative Breast Cancer
Interventions
TOS-358, Fulvestrant, Palbociclib, Ribociclib
Drug
Lead sponsor
Totus Medicines
Industry
Eligibility
18 Years and older · Female only
Enrollment
241 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
9
States / cities
Chicago, Illinois • Boston, Massachusetts • Las Vegas, Nevada + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)

NCT03094832
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Interventions
Miransertib
Drug
Lead sponsor
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Industry
Eligibility
2 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
6
States / cities
Atlanta, Georgia • Chicago, Illinois • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2023 · Synced Jul 23, 2026, 12:52 PM 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 8, 2023 · Synced Jul 23, 2026, 12:52 PM EDT
Completed Phase 1Phase 2 Interventional

Combination Trial of Tipifarnib and Alpelisib in Adult Recurrent/ Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)

NCT04997902
View directory record Official record
Conditions
HNSCC
Interventions
Tipifarnib, Alpelisib
Drug
Lead sponsor
Kura Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
11
States / cities
Duarte, California • Orlando, Florida • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

First-in-Human Study of Tersolisib (STX-478) as Monotherapy and in Combination With Other Antineoplastic Agents in Participants With Advanced Solid Tumors

NCT05768139
View directory record Official record
Conditions
Breast Cancer, Solid Tumors, Adult
Interventions
STX-478, Fulvestrant, Ribociclib, Palbociclib, Letrozole, Anastrozole, Exemestane, Tamoxifen, Abemaciclib, Imlunestrant, Metformin
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years and older
Enrollment
880 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
28
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Active, not recruiting Phase 2 Interventional

24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs

NCT07543822
View directory record Official record
Conditions
Vascular Anomalies, Vascular Anomaly, TIE2/PIK3CA Pathway Driven Vascular Anomalies
Interventions
alpelisib (BYL719)
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 30 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Completed Phase 1 Interventional

Dose Escalation of RMC-5552 Monotherapy in Relapsed/Refractory Solid Tumors

NCT04774952
View directory record Official record
Conditions
Solid Tumors
Interventions
RMC-5552
Drug
Lead sponsor
Revolution Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
8
States / cities
Irvine, California • Sacramento, California • San Francisco, California + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2025 · Synced Jul 23, 2026, 12:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

Evaluating Efficacy of Tivozanib (AV-951) in Biliary Tract Cancers

NCT04645160
View directory record Official record
Conditions
Cholangiocarcinoma, Bile Duct Neoplasm, Biliary Tract Malignancy
Interventions
Tivozanib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Multi-Center Study on the Use of Rho-Kinase Inhibitor to Reduce or Prevent PVR in RRD Eyes at High Risk for PVR

NCT05660447
View directory record Official record
Conditions
Rhegmatogenous Retinal Detachment, Proliferative Vitreoretinopathy
Interventions
Netarsudil 0.02% Ophthalmic Solution [RHOPRESSA], Glycerin 0.2%/Hypromellose 0.2%/Peg 1%/Soln,Oph,Ud
Drug
Lead sponsor
Wills Eye
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 29, 2024 · Synced Jul 23, 2026, 12:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

Sapanisertib and Serabelisib (PIKTOR) in Various Combinations in Patients With HR+/HER2- Advanced/Metastatic Breast Cancer

NCT07558733
View directory record Official record
Conditions
HR+ HER2- Breast Cancer
Interventions
Serabelisib, Sapanisertib, Fulvestrant
Drug
Lead sponsor
Faeth Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
7
States / cities
Los Angeles, California • Lone Tree, Colorado • Springfield, Oregon + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Completed Phase 1 Interventional

A Study To Assess The Tolerability And Clinical Activity Of Gedatolisib In Combination With Palbociclib/Letrozole Or Palbociclib/Fulvestrant In Women With Metastatic Breast Cancer

NCT02684032
View directory record Official record
Conditions
Breast Cancer
Interventions
Gedatolisib, Palbociclib, Letrozole, Fulvestrant
Drug
Lead sponsor
Celcuity Inc
Industry
Eligibility
18 Years and older · Female only
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
41
States / cities
Birmingham, Alabama • Corona, California • Fountain Valley, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 26, 2022 · Synced Jul 23, 2026, 12:52 PM EDT
Recruiting Phase 1 Interventional

9-ING-41 Combined With Retifanlimab, Plus Modified FOLFIRINOX for Patients With Advanced Pancreatic Adenocarcinoma (RiLEY)

NCT06896188
View directory record Official record
Conditions
Pancreatic Adenocarcinoma
Interventions
Retifanlimab, Chemotherapy, 9-ING-41
Drug
Lead sponsor
Anwaar Saeed
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Jul 23, 2026, 12:52 PM EDT
Completed Phase 3 Interventional Results available

Tanezumab in Osteoarthritis of the Knee

NCT00733902
View directory record Official record
Conditions
Arthritis, Osteoarthritis
Interventions
tanezumab, Placebo
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 99 Years
Enrollment
697 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
82
States / cities
Phoenix, Arizona • Danbury, Connecticut • Stamford, Connecticut + 65 more
Source: ClinicalTrials.gov public record
Updated Mar 21, 2021 · Synced Jul 23, 2026, 12:52 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322)

NCT05882734
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
M1774, Cemiplimab
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
6
States / cities
Santa Monica, California • Pittsburgh, Pennsylvania • Knoxville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Terminated Phase 2 Interventional Results available

Study of Safety and Efficacy of Dapagliflozin + Metformin XR Versus Metformin XR in Participants With HR+, HER2-, Advanced Breast Cancer While on Treatment With Alpelisib and Fulvestrant

NCT04899349
View directory record Official record
Conditions
Breast Cancer
Interventions
Alpelisib, Fulvestrant, Metformin XR, Dapagliflozin + metformin XR, Dapagliflozin
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Jul 23, 2026, 12:52 PM EDT
Suspended Phase 1Phase 2 Interventional

Dose Escalation/ Expansion Study of CA-4948 as Monotherapy in Patients With Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)

NCT04278768
View directory record Official record
Conditions
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Interventions
Emavusertib, Venetoclax
Drug
Lead sponsor
Curis, Inc.
Industry
Eligibility
18 Years and older
Enrollment
366 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
12
States / cities
Tampa, Florida • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Recruiting Phase 1 Interventional

Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)

NCT07543991
View directory record Official record
Conditions
Giant Axonal Neuropathy (GAN)
Interventions
scAAV9/JeT-GAN
Genetic
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
10 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Completed Phase 1 Interventional

Phase I Trial of Oral PX-866

NCT00726583
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
PX-866
Drug
Lead sponsor
Cascadian Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
2
States / cities
Aurora, Colorado • Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 16, 2018 · Synced Jul 23, 2026, 12:52 PM EDT
Enrolling by invitation Phase 2 Interventional

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

NCT06148311
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Matching placebo, Dexmedetomidine
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
16 Years to 80 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Jul 23, 2026, 12:52 PM EDT
Completed Phase 2 Interventional

The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia

NCT02276716
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Phosphatidylserine
Dietary Supplement
Lead sponsor
NYU Langone Health
Other
Eligibility
12 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced Jul 23, 2026, 12:52 PM EDT
Completed Phase 3 Interventional Results available

The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia

NCT01987219
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Albuterol-sulphate, Ipratropium-bromide, placebo
Drug · Other
Lead sponsor
NYU Langone Health
Other
Eligibility
12 Years to 80 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 1, 2016 · Synced Jul 23, 2026, 12:52 PM EDT