Clinical trials: Hereditary Sensory and Autonomic Neuropathies
- Condition Hereditary Sensory and Autonomic Neuropathies
- Funder type Industry (pharma, biotech, devices)
PIK3CA-Related Overgrowth Spectrum (PROS)
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the…
- Trial name
- EPIK-P1 (NCT04285723)
- Testing
- alpelisib
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Endometrial Cancer
Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel and a Substudy With an Insulin-Suppressing Diet in Patients With Advanced/Recurrent Endometrial Cancer
This is a Phase 2, multicenter, open-label, single-arm study to evaluate the efficacy and safety of sapanisertib and serabelisib (PIKTOR) with paclitaxel in participants with advanced or recurrent endometrial cancer.
- Trial name
- NCT06463028
- Testing
- Sapanisertib, Serabelisib, Paclitaxel
- Where
- 18 sites in United States
- Volunteers
- 40 people
- Sponsor
- Faeth Therapeutics
Advanced Solid Tumor
A Study to Evaluate ARV-6723 Alone and With Pembrolizumab in Participants With Advanced Solid Tumors
This is a study to evaluate the safety and potential anti-tumor activity of an investigational drug called ARV-6723, in participants with advanced solid tumors.
- Trial name
- NCT07749586
- Testing
- ARV-6723, Pembrolizumab, SOC
- Where
- 4 sites in United States
- Volunteers
- 499 people
- Sponsor
- Arvinas Inc.
Solid Tumors
Dose Escalation of RMC-5552 Monotherapy in Relapsed/Refractory Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of escalating doses of RMC-5552 monotherapy in adult participants with relapsed/refractory solid tumors…
- Trial name
- NCT04774952
- Testing
- RMC-5552
- Where
- 8 sites in United States
- Volunteers
- 58 people
- Sponsor
- Revolution Medicines, Inc.
Advanced Solid Tumor
Berzosertib Human Mass Balance Study (DDRiver Solid Tumors 208)
The study was conducted in two periods, Period 1 (mass balance) and Period 2 (extension). The purpose of Period 1 of this study was to provide a quantitative characterization of the mass balance, rates and routes of elimination…
- Trial name
- NCT05246111
- Testing
- [14C]Berzosertib, Berzosertib, Topotecan
- Where
- 2 sites in Hungary
- Volunteers
- 6 people
- Sponsor
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Recurrent Head and Neck Squamous Cell Carcinoma, Metastatic Head and Neck Squamous Cell Carcinoma
A Phase Ib/II Study of BYL719 and Cetuximab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma
This was a multi-center, open-label, Phase Ib dose escalation /Phase II study in recurrent or metastatic head and neck squamous cell carcinoma (RM HNSCC) patients considered to be resistant, ineligible or intolerant to…
- Trial name
- NCT01602315
- Testing
- BYL719 as film-coated (FC) whole tablets, BYL719 as dispersible tablets (DT), cetuximab
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 179 people
- Sponsor
- Novartis Pharmaceuticals
Breast Cancer
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
This study was designed to assess the safety and efficacy of the combination of dapagliflozin plus metformin extended release (XR) compared with metformin XR during treatment with alpelisib plus fulvestrant in participants with…
- Trial name
- EPIK-B4 (NCT04899349)
- Testing
- Alpelisib, Fulvestrant, Metformin XR
- Where
- 2 sites in United States, Malaysia
- Volunteers
- 2 people
- Sponsor
- Novartis Pharmaceuticals
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
- Trial name
- EXIST-2 (NCT00790400)
- Testing
- Everolimus (RAD001)
- Where
- 25 sites in United States, Canada and 9 more
- Volunteers
- 118 people
- Sponsor
- Novartis Pharmaceuticals
PIK3CA-Related Overgrowth Spectrum
A Study of the Epidemiology and Hospital Management of Patients With PROS in France
The purpose of this study was to estimate the incidence and prevalence of a group of genetic disorders known as PIK3CA-Related Overgrowth Spectrum (PROS) in France.
- Trial name
- EpiPROS (NCT07222423)
- Where
- United States
- Volunteers
- 3,605 people
- Sponsor
- Novartis Pharmaceuticals
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)
This is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA)-related Overgrowth…
- Trial name
- MOSAIC (NCT03094832)
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 50 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Recurrent Glioblastoma Multiforme, Primary Glioblastoma Multiforme
A Study to Assess the Safety and Tolerability of AZD1390 Given With Radiation Therapy in Patients With Brain Cancer
This study will test an investigational drug called AZD1390 in combination with radiation therapy for the treatment of brain tumors. This is the first time AZD1390 is being given to patients.
- Conditions
- Recurrent Glioblastoma Multiforme, Primary Glioblastoma Multiforme, Brain Neoplasms, Malignant, Leptomeningeal Disease (LMD)
- Trial name
- NCT03423628
- Testing
- Radiation Therapy, AZD1390
- Where
- 11 sites in United States, Japan and 1 more
- Volunteers
- 159 people
- Sponsor
- AstraZeneca
Alzheimer's Disease
Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease
The purpose of this study is to evaluate the potential benefits of CERE-110 in the treatment of Alzheimer's disease. CERE-110 is an experimental drug that is designed to help nerve cells in the brain function better.
- Trial name
- NCT00876863
- Testing
- CERE-110: Adeno-Associated Virus injection Surgery
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Sangamo Therapeutics
Breast Cancer
Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole, Based on Prior Endocrine Therapy, in Patients With PIK3CA Mutant, HR+, HER2- Advanced Breast Cancer Who Have Progressed on or After Prior Treatments
This was a Phase II, multicenter, open-label, three-cohort, non-comparative study of alpelisib plus endocrine therapy (either fulvestrant or letrozole) in subjects (pre- and post-menopausal women and men) with HR-positive…
- Trial name
- BYLieve (NCT03056755)
- Testing
- Alpelisib, Fulvestrant, Letrozole
- Where
- 94 sites in United States, Argentina and 17 more
- Volunteers
- 383 people
- Sponsor
- Novartis Pharmaceuticals
Covid19
A Phase 2 Study of RTB101 as COVID-19 Post-Exposure Prophylaxis in Older Adults
The proposed trial will obtain preliminary data on the feasibility of studying RTB101 as compared to placebo for COVID-19 post-exposure prophylaxis in adults age ≥ 65 years to inform the design of a subsequent pivotal trial.
- Trial name
- NCT04584710
- Testing
- RTB101
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- Restorbio Inc.
Specific Advanced Solid Tumors
Study of ACR-2316 in Specific Advanced Solid Tumors
This is a first in-human, Open-label Phase 1/2 study to assess the safety and activity of ACR-2316 for the treatment of subjects with specific, histologically confirmed, locally advanced, recurrent or metastatic solid tumors.
- Trial name
- NCT06667141
- Testing
- ACR-2316
- Where
- 15 sites in United States
- Volunteers
- 100 people
- Sponsor
- Acrivon Therapeutics
Breast Cancer
Study of the Molecular Features of Postmenopausal Women With HR+ HER2-negative aBC on First-line Treatment With Ribociclib and Letrozole and, in Patients With a PIK3CA Mutation, on Second-line Treatment With Alpelisib Plus Fulvestrant
The purpose of this clinical trial is to study of the molecular features of postmenopausal women with hormone receptor-positive (HR+) HER2-negative advanced breast cancer on first-line treatment with ribociclib and letrozole and…
- Trial name
- BioItaLEE (NCT03439046)
- Testing
- Ribociclib, Letrozole, Alpelisib
- Where
- 41 sites in Italy
- Volunteers
- 287 people
- Sponsor
- Novartis Pharmaceuticals
PI3K and P110delta Hyperactivation Syndrome
CVL237 Tablets for APDS/PASLI
This study was designed to evaluate the efficacy and safety of CVL237 tablets in patients with APDS/PASLI (activated phosphoinositol 3-kinase δ syndrome /p110 delta-activated mutation leading to senescent T cells…
- Trial name
- NCT06293716
- Testing
- CVL237 tablets
- Where
- China
- Volunteers
- 35 people
- Sponsor
- Convalife (Shanghai) Co., Ltd.
Haematologic Neoplasms
A Study of AZD2962, an IRAK4 Inhibitor (IRAK4 [a Body Protein] Blocker), in Participants With Haematologic Neoplasms (Blood Cancers)
The purpose of the study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary efficacy of AZD2962, an Interleukin-1 Receptor-Associated Kinase 4 (IRAK4) inhibitor, as monotherapy and in…
- Trial name
- NCT07064122
- Testing
- AZD2962
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 72 people
- Sponsor
- AstraZeneca
Osteoarthritis, Arthritis
Long-Term Analgesic Efficacy And Safety Of Tanezumab Alone Or In Combination With Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) Versus NSAIDs Alone In Patients With Osteoarthritis Of The Knee Or Hip
The purpose of this study is to investigate the long-term analgesic efficacy and safety of tanezumab for patients with osteoarthritis (OA) of the knee or hip currently experiencing partial benefit from, and are tolerating…
- Trial name
- NCT00809354
- Testing
- NSAID, tanezumab
- Where
- 333 sites in United States, Canada and 10 more
- Volunteers
- 2,720 people
- Sponsor
- Pfizer
Rheumatoid Arthritis
A Study Investigating the Efficacy and Safety of BGB-45035 Versus Placebo in Adults With Moderate to Severe Active Rheumatoid Arthritis
This study aims to evaluate the efficacy of BGB-45035 in adults with moderate to severe active rheumatoid arthritis (RA) who have had an inadequate response to conventional synthetic disease-modifying antirheumatic drugs…
- Trial name
- NCT07100938
- Testing
- BGB-45035
- Where
- 29 sites in China
- Volunteers
- 49 people
- Sponsor
- BeiGene