Clinical trials: Small Vessel Disease
Fabry Disease
Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease
- Trial name
- NCT00526071
- Testing
- migalastat HCl
- Where
- 8 sites in United States, Australia and 3 more
- Volunteers
- 23 people
- Sponsor
- Amicus Therapeutics
Relapsed or Refractory B-cell Malignancies, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL)
Dose Determining Study of EXS73565 in Participants With Relapsed or Refractory B-Cell Malignancies
The purpose of this study is to characterize the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of EXS73565 administered orally as a single agent in participants with…
- Conditions
- Relapsed or Refractory B-cell Malignancies, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Follicular Lymphoma (FL), Marginal Zone Lymphoma (MZL)
- Trial name
- NCT06980116
- Testing
- EXS73565
- Where
- 17 sites in Georgia, Greece and 5 more
- Volunteers
- 85 people
- Sponsor
- Exscientia AI Ltd., a wholly owned subsidiary of Recursion Pharmaceuticals, Inc.
Solid Tumors, Multiple Myeloma
Phase I, Multicenter, Dose Escalation Study of DCR-MYC in Patients With Solid Tumors, Multiple Myeloma, or Lymphoma
The purpose of this study is to assess the safety and tolerability of the investigational anticancer drug DCR-MYC. DCR-MYC is a novel synthetic double-stranded RNA in a stable lipid particle suspension that targets the oncogene…
- Conditions
- Solid Tumors, Multiple Myeloma, Non-Hodgkins Lymphoma, Pancreatic Neuroendocrine Tumors
- Trial name
- NCT02110563
- Testing
- DCR-MYC
- Where
- 6 sites in United States
- Volunteers
- 50 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Fabry Disease
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
- Trial name
- NCT06270316
- Testing
- AMT-191
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- UniQure Biopharma B.V.
Fabry Disease
Early Detection of Fabry Disease
The main aim of this study is early detection of FD using real-world data for the development of advanced natural language processing methods and to develop a predictive algorithm and to measure the performance of the algorithm…
- Trial name
- NCT05106764
- Testing
- No intervention
- Where
- 3 sites in Germany
- Volunteers
- 50 people
- Sponsor
- Takeda
Relapsed/Refractory Hematological Malignancies, Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Leukemia (CLL/SLL
A Study of APG-3288 in Relapsed/Refractory Blood Cancers
This is a Phase I, multicenter, open-label, two-stage study of APG-3288 monotherapy, aiming to determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of APG-3288 administered orally once daily in patients…
- Conditions
- Relapsed/Refractory Hematological Malignancies, Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Leukemia (CLL/SLL, Relapsed/Refractory Diffuse Large B-cell Lymphoma (DLBCL; Including Richter Transformation), Relapsed/Refractory Mantle Cell Lymphoma (MCL)
- Trial name
- NCT07424833
- Testing
- APG-3288
- Where
- 6 sites in United States, China
- Volunteers
- 180 people
- Sponsor
- Ascentage Pharma Group Inc.
Fabry Disease
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without…
- Trial name
- FIELD (NCT00701415)
- Testing
- Agalsidase beta
- Where
- 12 sites in United States, Argentina and 7 more
- Volunteers
- 31 people
- Sponsor
- Genzyme, a Sanofi Company
Indolent Non-Hodgkin's Lymphomas
Efficacy and Safety of Idelalisib (GS-1101) in Combination With Bendamustine and Rituximab for Previously Treated Indolent Non-Hodgkin Lymphomas
The primary objective of this study is to evaluate the addition of idelalisib to bendamustine/rituximab on progression-free survival (PFS) in adults with previously treated indolent non-Hodgkin lymphoma (iNHL).
- Trial name
- Bridalveil (NCT01732926)
- Testing
- Idelalisib, Rituximab, Bendamustine
- Where
- 144 sites in United States, Australia and 13 more
- Volunteers
- 475 people
- Sponsor
- Gilead Sciences
Fabry Disease
The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease
- Trial name
- NCT06819514
- Testing
- EXG110 Injection
- Where
- 3 sites in China
- Volunteers
- 16 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Fabry Disease
Migalastat Food Effect Study
A 5-period crossover study to evaluate the effect of meal type and timing on migalastat HCl pharmacokinetics in healthy male and female subjects.
- Trial name
- NCT01489995
- Testing
- A (migalastat), B (migalastat), C (migalastat)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Pulmonary Hypertension, Interstitial Lung Disease
A Phase 3 Trial of Inhaled Mosliciguat in PH-ILD (PHrontier)
A Phase 3, Multicenter, Randomized, Double-Blind Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Inhaled Mosliciguat in Participants with Pulmonary Hypertension Associated with Interstitial Lung Disease
- Conditions
- Pulmonary Hypertension, Interstitial Lung Disease, Vascular Diseases, Cardiovascular Diseases
- Trial name
- NCT07816185
- Testing
- Mosliciguat, Dry Powder Inhaler
- Where
- 3 sites in United States
- Volunteers
- 376 people
- Sponsor
- Pulmovant, Inc.
Abdominal Aortic Aneurysm
Nectero EAST System Clinical Study
The purpose of this randomized clinical trial is to treat patients with small to mid-sized abdominal aortic aneurysms (AAA), maximum diameter of 3.5 cm to 5.0 cm, using a locally delivered, single-dose endovascular treatment.
- Trial name
- stAAAble (NCT06001918)
- Testing
- Nectero EAST System
- Where
- 42 sites in United States, Australia and 1 more
- Volunteers
- 400 people
- Sponsor
- Nectero Medical, Inc.
CLL / SLL, CLL (Chronic Lymphocytic Leukemia)
A Study of Lonitoclax (ZE50-0134) in Relapsed or Refractory B-cell Malignancies
This Phase 1, open-label, multicenter study is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of lonitoclax (ZE50-0134) in adults with relapsed or refractory chronic…
- Conditions
- CLL / SLL, CLL (Chronic Lymphocytic Leukemia), SLL (Small Lymphocytic Lymphoma), Marginal Zone Lymphoma(MZL)
- Trial name
- NCT06708897
- Testing
- Lonitoclax (ZE50-0134)
- Where
- 5 sites in United States
- Volunteers
- 84 people
- Sponsor
- Lomond Therapeutics Holdings, Inc.
Follicular Lymphoma, Small Lymphocytic Lymphoma
Efficacy and Safety Study of Idelalisib in Participants With Indolent B-Cell Non-Hodgkin Lymphomas
The primary objective will be to assess the overall response rate and to evaluate the efficacy and safety of idelalisib (IDELA; GS-1101) in participants with previously treated indolent Non-Hodgkin Lymphoma (iNHL) that is…
- Conditions
- Follicular Lymphoma, Small Lymphocytic Lymphoma, Lymphoplasmacytic Lymphoma, Marginal Zone Lymphoma
- Trial name
- DELTA (NCT01282424)
- Testing
- Idelalisib
- Where
- 41 sites in United States, France and 4 more
- Volunteers
- 125 people
- Sponsor
- Gilead Sciences
Fabry Disease
A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2…
- Trial name
- NCT01650779
- Testing
- Agalsidase beta
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Genzyme, a Sanofi Company