A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
NCT05861999
Recruiting
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
Gender:
ALL
Ages:
Between 3 months and 24 months
Trial Updated:
07/28/2025
Locations: University of Arkansas for Medical Sciences, Little Rock, Arkansas +18 locations
Conditions: Muscular Atrophy, Spinal
Register for Updates
Trial of Therapeutic Cancer Vaccine OSE2101 in Patients With Non-Small Cell Lung Cancer and Secondary Resistance to Immune Checkpoint Inhibitor
NCT06472245
Recruiting
Multicenter, randomized (2:1), open-label phase 3 study in HLA-A2 positive patients with squamous and non-squamous metastatic NSCLC with ICI secondary resistance. Patients will be randomized into 2 arms (randomization 2:1): experimental Arm A with OSE2101 monotherapy or control Arm B SoC with docetaxel monotherapy. Stratification factors will be histology (squamous versus non squamous) and ECOG Performance Status (0 versus 1).
Gender:
ALL
Ages:
18 years and above
Trial Updated:
07/28/2025
Locations: Clinical Research Advisors, LLC, Beverly Hills, California +204 locations
Conditions: Patients With Non-Small Cell Lung Cancer
Register for Updates
Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
NCT06669754
Recruiting
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to prevent angioedema attacks in participants aged ≥ 12 years with hereditary angioedema.
Gender:
ALL
Ages:
12 years and above
Trial Updated:
07/28/2025
Locations: Study Site, Little Rock, Arkansas +51 locations
Conditions: Hereditary Angioedema (HAE)
Register for Updates
A Study Evaluating Efruxifermin in Subjects With Compensated Cirrhosis Due to NASH/MASH
NCT06528314
Recruiting
This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis due to NASH/MASH.
Gender:
ALL
Ages:
Between 18 years and 80 years
Trial Updated:
07/28/2025
Locations: Akero Clinical Study Site, Birmingham, Alabama +261 locations
Conditions: NASH - Nonalcoholic Steatohepatitis, MASH - Metabolic Dysfunction-Associated Steatohepatitis
Register for Updates
A Study to Assess the Efficacy and Safety of Induction Therapy With Afimkibart (Also Known as RO7790121) in Participants With Moderately to Severely Active Ulcerative Colitis
NCT06588855
Recruiting
This Phase III, multicenter, double-blind, placebo-controlled study will evaluate the efficacy and safety of induction therapy with Afimkibart (RO7790121) compared with placebo in participants with moderately to severely active ulcerative colitis (UC).
Gender:
ALL
Ages:
Between 16 years and 80 years
Trial Updated:
07/28/2025
Locations: Mayo Clinic Hospital, Scottsdale, Arizona +144 locations
Conditions: Moderately to Severely Active Ulcerative Colitis
Register for Updates
Nitrous Oxide and EMOtional Cognition
NCT06557642
Recruiting
The goal of this study is to uncover the underlying neuropsychological mechanisms of nitrous oxide, which may be relevant to its reported antidepressant effects. In particular, the study is designed to determine whether nitrous oxide changes the way in which healthy volunteers aged 18-40 years remember negative autobiographical memories. The main question the study aims to answer is: • Does nitrous oxide interfere with the emotional aspects of negative autobiographical memories? Other neurops... Read More
Gender:
ALL
Ages:
Between 18 years and 40 years
Trial Updated:
07/28/2025
Locations: Psychiatry Department, Oxford, Not set
Conditions: Depression
Register for Updates
A Study to Assess the Efficacy and Safety of Afimkibart (Also Known as RO7790121) for Induction and Maintenance Therapy in Participants With Moderately to Severely Active Ulcerative Colitis
NCT06589986
Recruiting
This Phase III, multicenter, double-blind, placebo-controlled, treat-through study will evaluate the efficacy and safety of Afimkibart (RO7790121) compared with placebo in participants with moderately to severely active ulcerative colitis (UC).
Gender:
ALL
Ages:
Between 16 years and 80 years
Trial Updated:
07/28/2025
Locations: Digestive Health Specialists of the Southeast (Gastroenterology Associates of Dothan) - Dothan, Dothan, Alabama +278 locations
Conditions: Moderately to Severely Active Ulcerative Colitis
Register for Updates
The Causal Role of Ketone Bodies in Obesity-associated Disease Prevention - Combining Genetic Epidemiology With a Randomised Trial to Infer Causality
NCT06668168
Recruiting
Excess weight increases the risk of several diseases including cardiovascular disease, type 2 diabetes, kidney disease and various cancers. There is a need for preventative strategies for obesity-associated disease, especially for people in the overweight and moderately obese ranges where pharmacological intervention may not be suitable. Low-carbohydrate (ketogenic) diets are popular for weight control. Ketogenic diets increase circulating ketones, which can have favourable effects on cardiomet... Read More
Gender:
ALL
Ages:
Between 18 years and 65 years
Trial Updated:
07/28/2025
Locations: University of Bath, Bath, Not set
Conditions: Nutrition, Obesity and Overweight
Register for Updates
Phase 3 Study to Evaluate Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 2)
NCT05624749
Recruiting
The trial will evaluate efficacy, safety and tolerability of ianalumab compared to placebo, given as monthly subcutaneous (s.c.) injection on top of standard-of-care (SoC) treatment in participants with active systemic lupus erythematosus (SLE).
Gender:
ALL
Ages:
Between 12 years and 100 years
Trial Updated:
07/28/2025
Locations: Pinnacle Research Group LLC, Anniston, Alabama +107 locations
Conditions: Systemic Lupus Erythematosus
Register for Updates
A Study to Evaluate the Effect of Budesonide, Glycopyrronium, Formoterol Fumarate (BGF) Metered Dose Inhaler (MDI), Budesonide and Formoterol Fumarate (BFF) MDI and Placebo MDI on Exercise Parameters in Participants With Chronic Obstructive Pulmonary Disease (COPD).
NCT06067828
Recruiting
This study will investigate the effect of Budesonide, Glycopyrronium, and Formoterol Fumarate (BGF) metered dose inhaler (MDI) compared with Placebo MDI, and Budesonide and Formoterol Fumarate (BFF) MDI on isotime inspiratory capacity (IC) and exercise endurance time.
Gender:
ALL
Ages:
Between 40 years and 80 years
Trial Updated:
07/28/2025
Locations: Research Site, Los Angeles, California +36 locations
Conditions: Chronic Obstructive Pulmonary Disease
Register for Updates
A Study to Evaluate the Efficacy and Safety of GDC-8264 in Preventing Cardiac Surgery-Associated Acute Kidney Injury (AKI) and Major Adverse Kidney Events (MAKE)
NCT06602453
Recruiting
The aim of this study is to evaluate the efficacy and safety of GDC-8264 compared with placebo in participants undergoing cardiac surgery who are determined to be at moderate to high risk of developing AKI and subsequent MAKE at 90 days after surgery (MAKE90). The study will be performed in two parts- Part 1 and Part 2.
Gender:
ALL
Ages:
40 years and above
Trial Updated:
07/28/2025
Locations: Ronald Reagan UCLA Medical Center, Los Angeles, California +30 locations
Conditions: Acute Kidney Injury
Register for Updates
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
NCT06128629
Recruiting
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Gender:
ALL
Ages:
Between 18 years and 90 years
Trial Updated:
07/28/2025
Locations: University of Arizona, Tucson, Arizona +126 locations
Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Register for Updates