131 matching studies

Sponsor Condition of Interest
Global Patient Registry of Inherited Retinal Diseases
Janssen Research & Development, LLC Inherited Retinal Diseases
The purpose of this study is to better understand the natural history of Inherited Retinal Disease (IRD) and help inform patient management. expand

The purpose of this study is to better understand the natural history of Inherited Retinal Disease (IRD) and help inform patient management.

Type: Observational

Start Date: Apr 1992

open study
A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extreme1
OHB Neonatology Ltd. Bronchopulmonary Dysplasia Chronic Lung Disease of Prematurity Intraventricular Hemorrhage Retinopathy of Prematurity (ROP)
The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants receiving standard neonatal care alone. expand

The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants receiving standard neonatal care alone.

Type: Interventional

Start Date: May 2019

open study
A Trial of Lu AF82422 in Participants With Multiple System Atrophy (MSA)
H. Lundbeck A/S Multiple System Atrophy
The main goal of this trial is to evaluate the efficacy and safety of Lu AF82422 for the treatment of participants with Multiple System Atrophy (MSA). expand

The main goal of this trial is to evaluate the efficacy and safety of Lu AF82422 for the treatment of participants with Multiple System Atrophy (MSA).

Type: Interventional

Start Date: Dec 2024

open study
Extracellular Vesicle Treatment for Acute Respiratory Distress Syndrome (ARDS) (EXTINGUISH ARDS)
Direct Biologics, LLC Acute Respiratory Distress Syndrome ARDS
To evaluate the safety and efficacy of intravenous (IV) administration of bone marrow mesenchymal stem cell derived extracellular vesicles (EVs), ExoFlo, versus placebo for the treatment of hospitalized patients with moderate-to-severe Acute Respiratory Distress Syndrome (ARDS). expand

To evaluate the safety and efficacy of intravenous (IV) administration of bone marrow mesenchymal stem cell derived extracellular vesicles (EVs), ExoFlo, versus placebo for the treatment of hospitalized patients with moderate-to-severe Acute Respiratory Distress Syndrome (ARDS).

Type: Interventional

Start Date: Jul 2022

open study
Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
Arcturus Therapeutics, Inc. Cystic Fibrosis CFTR Gene Mutation
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators. expand

ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.

Type: Interventional

Start Date: Dec 2024

open study
High Dose Ascorbic Acid (HDAA) in Patients With Plasma Cell Disorders
University of Arkansas Plasma Cell Disorder
The purpose of this research is to evaluate whether HDAA in combination with a single dose of 100 mg/m2 IV melphalan followed by autologous stem cell transplantation (ASCT) is safe and effective for subjects with relapsed refractory multiple myeloma. The proposed melphalan dose is 50% of the curren1 expand

The purpose of this research is to evaluate whether HDAA in combination with a single dose of 100 mg/m2 IV melphalan followed by autologous stem cell transplantation (ASCT) is safe and effective for subjects with relapsed refractory multiple myeloma. The proposed melphalan dose is 50% of the current standard myeloablative dose (200 mg/m2). Based on our preclinical data, the investigator hypothesize that the combination of reduced dose melphalan with IV HDAA will have high efficacy and tolerability Primary Objective To determine tumor response using International Myeloma Working Group (IMWG) criteria (see Appendix B). Secondary Objectives Objectives: 1. Determine the safety and tolerability of HDAA in combination with reduced dose melphalan conditioning and autologous stem cell transplantation (ASCT) in relapsed refractory multiple myeloma subjects. 2. Determine the rate of Minimal Residual Disease (MRD) negativity at time point of response assessment using 8 color flow cytometry on BM sample. Functional imaging, such as positron emission tomography (PET) scan and magnetic resonance imaging (MRI), will also be performed to assess the disease status. 3. Categorize and quantify adverse events compared to historical control. 4. Determine quality of life parameters using standardized health-related quality of life measures 5. Determine oxidative stress parameters in plasma during treatment.

Type: Interventional

Start Date: Jul 2024

open study