Clinical trials: Efficacy
Sarcoma
Safety and Efficacy Study Using Rexin-G for Sarcoma
Rexin-G is a tumor-targeted (pathotropic or disease-seeking) nanoparticle that when injected intravenously, seeks out and accumulates in cancerous lesions, thus enhancing local drug concentration within tumors.
- Trial name
- NCT00505713
- Testing
- Rexin-G
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Epeius Biotechnologies
Spinal Muscular Atrophy
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS…
- Trial name
- EMBRACE (NCT02462759)
- Testing
- Nusinersen
- Where
- 7 sites in United States, Germany
- Volunteers
- 21 people
- Sponsor
- Biogen
Asthma
Study on BI 54903 (Inhaled Corticosteroid) Administered Once Daily or Twice Daily Via Respimat Inhaler in Patients With Asthma Inadequately Controlled on Short Acting Beta-2 Agonist (SABA) Therapy Alone
The aim of this study is to assess and compare efficacy and safety of BI 54903 at doses of very low dose twice daily (b.i.d) and low dose once daily (evening dosing) and placebo over an 12-week treatment period in asthmatic…
- Trial name
- NCT01458886
- Testing
- BI 54903 MD, BI 54903
- Sponsor
- Boehringer Ingelheim
Netherton Syndrome
Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome
To assess the safety, tolerability, and efficacy of QRX003 lotion (4%) when added to standard of care treatment regimen, including systemic therapy in subjects with Netherton syndrome
- Trial name
- NCT05789056
- Testing
- QRX003, 4% Lotion
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- Quoin Pharmaceuticals
Hemophilia A
China ADVATE PTP Study
The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)
- Trial name
- NCT02170402
- Testing
- Octocog alfa (recombinant human coagulation factor VIII)
- Where
- 11 sites in China
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
SARS-CoV Infection, Covid19
A Study to Evaluate the Efficacy, Immune Response, and Safety of a COVID-19 Vaccine in Adults ≥ 18 Years With a Pediatric Expansion in Adolescents (12 to<18 Years) at Risk for SARS-CoV-2
This is a phase 3, randomized, observer-blinded, placebo-controlled study to evaluate the efficacy, safety, and immunogenicity of SARS-CoV-2 rS with Matrix-M1 adjuvant in adult participants and adolescent participants.
- Trial name
- NCT04611802
- Testing
- SARS-CoV-2 rS/Matrix-M1 Adjuvant (Initial Vaccination Period), SARS-CoV-2 rS/Matrix-M1 Adjuvant (Crossover Vaccination period), SARS-CoV-2 rS/Matrix-M1 Adjuvant (Booster Vaccination)
- Where
- 154 sites in United States, Mexico and 1 more
- Volunteers
- 33,000 people, healthy volunteers welcome
- Sponsor
- Novavax
Locally Advanced Solid Tumor
Phase III Clinical Study of VC004 in Patients With Localized Advanced/ Metastatic Solid Tumors
This Study is a single arm, open label, multicenter phase III clinical trial to evaluate the safety and efficacy of VC004 in patients with locally advanced/metastatic solid tumors.
- Trial name
- NCT06658353
- Testing
- VC004 Capsules
- Where
- China
- Volunteers
- 54 people
- Sponsor
- Jiangsu vcare pharmaceutical technology co., LTD
Acute Lymphoblastic Leukemia
A Real-world Study of the Effectiveness of Tisagenlecleucel in Acute Lymphoblastic Leukemia Patients
This was a retrospective, cross-sectional, center-based chart review study that collected real-world data for relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL) patients receiving tisagenlecleucel (tisa-cel).
- Trial name
- KareALL (NCT07039383)
- Where
- United States
- Volunteers
- 79 people
- Sponsor
- Novartis Pharmaceuticals
Angelman Syndrome
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.
- Trial name
- NCT02996305
- Testing
- OV101 Regimen 1, OV101 regimen 2
- Where
- 13 sites in United States, Israel
- Volunteers
- 88 people
- Sponsor
- Healx AI
Asthma
Dose Finding Trial to Assess the Efficacy and Safety of SOTB07 in Persistent Asthma
This study is a dose finding trial to assess the efficacy and safety of SOTB07 in persistent asthma.
- Trial name
- NCT00936624
- Testing
- SOTB07, montelukast
- Where
- South Korea
- Volunteers
- 245 people
- Sponsor
- SK Chemicals Co., Ltd.
Primary Sjögren's Syndrome
Study of Pharmacodynamic Effects of VAY736 in Patients With Primary Sjögren's Syndrome
This study consists of three consecutive parts. Part 1 in primary Sjögren's syndrome (pSS) patients (n=2-6) and Part 2 in healthy voluteers (n=3) are feasibility studies to assess if the selected [Zr-89]-rituximab PET/CT method…
- Trial name
- NCT02495129
- Testing
- VAY736 lower dose, VAY736 higher dose
- Sponsor
- Novartis Pharmaceuticals
COVID-19
Trial to Evaluate Efficacy and Safety of Nitazoxanide in the Treatment of Mild or Moderate COVID-19
Trial to Evaluate Efficacy and Safety of Nitazoxanide in the Treatment of Mild or Moderate COVID-19
- Trial name
- NCT04486313
- Testing
- Nitazoxanide, Vitamin Super B-Complex
- Where
- 2 sites in United States
- Volunteers
- 935 people
- Sponsor
- Romark Laboratories L.C.
Aortic Valve Insufficiency, Aortic Valve Stenosis
ALLIANCE AVIV: Safety and Effectiveness of the SAPIEN X4 Transcatheter Heart Valve in Failing Aortic Bioprosthetic Valves
The objective of this study is to establish the safety and effectiveness of the Edwards SAPIEN X4/X4S Transcatheter Heart Valve (THV) in subjects who are at high or greater risk with a failing aortic bioprosthetic valve.
- Trial name
- NCT05172973
- Testing
- SAPIEN X4/X4S THV
- Where
- 37 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- Edwards Lifesciences
Neuroblastoma, CNS Metastases
131I-omburtamab Radioimmunotherapy for Neuroblastoma Central Nervous System/Leptomeningeal Metastases
Children with a neuroblastoma diagnose and central nervous system (CNS)/leptomeningeal metastases will be given up to 2 rounds of intracerebroventricular treatment with a radiolabelled monoclonal antibody, 131I-omburtamab to…
- Conditions
- Neuroblastoma, CNS Metastases, Leptomeningeal Metastases
- Trial name
- NCT03275402
- Testing
- 131I-omburtamab
- Where
- 8 sites in United States, Denmark and 2 more
- Volunteers
- 52 people
- Sponsor
- Y-mAbs Therapeutics
Systemic Lupus Erythematosus, Lupus Nephritis
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
- Trial name
- NCT06121297
- Testing
- CABA-201
- Where
- 23 sites in United States, Canada and 1 more
- Volunteers
- 28 people
- Sponsor
- Cabaletta Bio
Influenza
Study to Assess the Efficacy and Safety of GP681 Versus Placebo for Postexposure Prophylaxis Against Influenza
Index patients who are infected with influenza virus (Q-PCR positive) can be treated with anti-influenza drugs if their influenza symptoms onset was within 48 hours of screening.
- Trial name
- NCT06574503
- Testing
- GP681 40mg, GP681 Simulant
- Where
- China
- Volunteers
- 748 people, healthy volunteers welcome
- Sponsor
- Jiangxi Qingfeng Pharmaceutical Co. Ltd.
Upper Respiratory Tract Infections
A Randomized, Double-blind, Placebo-controlled Study on the Efficacy and Safety of CVT-E002 in the Treatment of Upper Respiratory Tract Infections in a Pediatric (3-11 Years) Population
Parents with children aged 3-11 will participate in this study. About 500 children will take part in the study. We expect about half of those children will develop symptoms of a respiratory infection.
- Trial name
- NCT00965822
- Testing
- North American ginseng
- Where
- 4 sites in Canada
- Volunteers
- 293 people, healthy volunteers welcome
- Sponsor
- Afexa Life Sciences Inc
Acute Pain
A Study to Look at Tapentadol Oral Solution in Children and Adolescents in Pain
The purpose of the study was to evaluate the efficacy of tapentadol oral solution, based on the total amount of supplemental opioid analgesic used over 12 hours or 24 hours after initiation of investigational medicinal product…
- Trial name
- NCT02081391
- Testing
- Tapentadol oral solution 4 mg/mL, Tapentadol oral solution 20 mg/mL
- Where
- 43 sites in United States, Bulgaria and 8 more
- Volunteers
- 216 people
- Sponsor
- Grünenthal GmbH
X-Linked Retinitis Pigmentosa
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants…
- Trial name
- NCT04850118
- Testing
- rAAV2tYF-GRK1-hRPGRco, Control
- Where
- 25 sites in United States, Australia and 2 more
- Volunteers
- 85 people
- Sponsor
- Beacon Therapeutics
Achondroplasia
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
- Trial name
- NCT03424018
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 119 people
- Sponsor
- BioMarin Pharmaceutical
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