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Намерени 3,079 изпитвания Стр. 1 от 154

By invitation only Phase 2

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Hypochondroplasia

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

Начало: 21.04.2026 Край: 31.05.2036 Възраст: 3–18 г. Treatment

Australia, Canada, France +7 QED Therapeutics, a BridgeBio company NCT07393373

By invitation only Phase 3

An Extension Study to Investigate the Long-term Safety and Tolerability of Itepekimab in Adult Participants With Inadequately Controlled Chronic Rhinosinusitis With Nasal Polyps

Chronic Rhinosinusitis With Nasal Polyps

This is a double-blind, parallel-group, Phase 3, 2-arm study that is designed to provide additional safety information, assess the durability of treatment response, and provide additional PK and immunogenicity assessments. The primary purpose of this study is to evaluate safety and tolerability of both new or continued treatment with itepekimab 300 mg SC high dose or itepekimab 300 mg SC low dose in participants with CRSwNP having completed the intervention period of the clinical studies EFC18418 or EFC18419. A secondary purpose of this study is to provide efficacy outcomes beyond the intervention period of the parent trials EFC18418 and EFC18419. Study details include: * The study duration will be up to 72 weeks. * The intervention duration will be 52 weeks. * A follow-up period of 20 weeks will be conducted. * The number of visits will be 8 and the number of phone contacts will be 4.

Начало: 12.03.2026 Край: 12.12.2028 Възраст: от 18 г. Treatment

Australia, China, United Kingdom +1 Sanofi NCT07424144

By invitation only Phase 1/2

Study of TN-001 Topical Eyedrops for Keratoconus

Keratoconus

The goal of this clinical trial is to learn if TN-001, a new eye-drop therapy, is safe and well-tolerated in adult patients with progressive keratoconus. It will also provide data on whether this therapy is effective in treating keratoconus. The main questions it aims to answer are: What medical problems do participants have when using TN-001 eyedrops? Does TN-001 eyedrop therapy stop keratoconus from progressing further? Participants will: Take TN-001 eyedrops twice a day, every day, for 3-6 weeks. Visit the clinic once every week for checkups and tests. Keep a diary of their symptoms.

Начало: 23.12.2025 Край: 01.07.2027 Възраст: 18–40 г. Treatment

Australia TheiaNova Ltd. NCT07388069

By invitation only

Implementation Research on the LILAC Method to Educate Management of Elevated Lipoprotein(a).

Lipoprotein(a) Lipoprotein(a), Hyper-Lp(a)-Emia

The goal of this observational study is to assess the if short educational videos about #LILAC-for-Lpa are feasible and accepted in improving the confidence, testing, and management on Lipoprotein(a).

Начало: 09.09.2025 Край: 01.12.2028 Възраст: от 16 г.

Australia, Malaysia, Singapore Changi General Hospital NCT07177612

By invitation only Phase 2

An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203. TIS6463-204 is evaluating zagociguat in an open-label extension study at the daily dose of 15mg. The study medication is a once daily oral table and will be provided at the clinic and/or shipped to the participant's home. Study assessments will be conducted during clinic visits which will occur at three months and then at six months. Thereafter, clinic visits will occur every six months.

Начало: 18.08.2025 Край: 01.07.2028 Treatment

Australia, Germany, Italy +2 Tisento Therapeutics NCT06961344

By invitation only

TANDem-2: Closing the Gap to Interventions for TAND

Adult Caregivers of Individuals With TSC Tuberous Sclerosis Complex (TSC)

The goal of this pilot study is to determine the feasibility, acceptability and impact of a brief behavioral intervention - the "Well-Beans for Caregivers" program, for adult caregivers of individuals with tuberous sclerosis complex (TSC) of any age. The main questions of the study are: 1. Can this innovative, ultra-brief caregiver well-being intervention be delivered feasibly to caregivers via an online modality? 2. How do the caregivers experience the program (acceptability)? 3. Does limited efficacy testing suggest the program has an impact on caregiver's well-being?

Начало: 15.07.2025 Край: 15.07.2027 Възраст: от 18 г. Treatment

Australia, United States Vrije Universiteit Brussel NCT06879665

By invitation only Phase 1

A Study of KITE-363 in Participants With Refractory Autoimmune Diseases

Systemic Lupus Erythematosus Lupus Nephritis Systemic Sclerosis Idiopathic Inflammatory Myopathy

This study will have two Phases: Phase 1a and Phase 1b. The goal of this clinical study is to learn more about the study drug KITE-363, to establish dosing, tolerability, safety, and preliminary efficacy of KITE-363 in participants with refractory autoimmune diseases. The primary objectives of this study are: Phase 1a: To evaluate the safety and tolerability of KITE-363 in participants with autoimmune disease. To determine the recommended dose for Phase 1b. Phase 1b: To evaluate the safety and efficacy of KITE-363 in participants with autoimmune disease.

Начало: 02.07.2025 Край: 01.07.2029 Възраст: от 18 г. Treatment

Australia, Canada, United States Kite, A Gilead Company NCT07038447

By invitation only Phase 3

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Hypochondroplasia

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

Начало: 20.06.2025 Край: 01.12.2040 Treatment

Australia, Canada, France +6 BioMarin Pharmaceutical NCT07073014

By invitation only

Cities for Better Health Childhood Obesity Prevention Initiative

Obesity

This research project is part of the 'Cities for Better Health Childhood Obesity Prevention Initiative' (CBH COPI). The objective of the CBH COPI initiative is to develop, implement, and evaluate the effectiveness of a package of prevention interventions in five cities to promote healthy eating, physical activity, a positive health-related quality of life, and a reduction in rates living with overweight or obesity among children aged 6-13 years in six cities across Australia, Brazil, Canada, Japan, South Africa and Spain. The primary research objective of CBH COPI is to estimate the impact of the intervention packages on the average BMI of the target population of children across the participating countries, at one-year and two-year follow-ups. The secondary research objective is to estimate the impact of the intervention packages on average health-related quality of life (HRQoL) at the same time points. Exploratory aims include estimating the impact of the packages on behaviours related to physical activity and diet.

Начало: 30.04.2025 Край: 31.12.2027 Възраст: 6–13 г. Prevention

Australia, Brazil, Canada +3 Novo Nordisk A/S NCT06855563

By invitation only Phase 2

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

Myotonic Dystrophy Type 1 (DM1)

The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).

Начало: 28.04.2025 Край: 30.01.2029 Възраст: от 18 г. Treatment

Australia, Belgium, Canada +2 Vertex Pharmaceuticals Incorporated NCT06926621

By invitation only Phase 2/3

An Interventional Study of Infigratinib in Children With Hypochondroplasia

Hypochondroplasia

ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety, of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib, at one of two doses, of who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).

Начало: 22.04.2025 Край: 01.01.2027 Възраст: 3–18 г. Treatment

Australia, Canada, France +7 QED Therapeutics, a BridgeBio company NCT06873035

By invitation only

Assessment of Worldwide Acute Kidney Injury Epidemiology in Neonates 2.0

Acute Kidney Injury Fluid Overload Prematurity Complications Risk Factors +3

There is a growing focus on short- and long-term kidney health in neonates, including those with acute kidney injury (AKI). AKI occurs commonly in the Neonatal Intensive Care Unit (NICU) and is associated with adverse outcomes. In addition to poor outcomes during the hospitalization, infants discharged from the NICU may have an increased burden of kidney disease during childhood. Studies of long-term kidney function in children born prematurely show a fourfold increase in chronic kidney disease (CKD) by adolescence and into adulthood. Despite the landmark findings of the Assessment of Worldwide Acute Kidney Injury Epidemiology in Neonates (AWAKEN) study, the limitations of this study are notable. First, the AWAKEN study enrolled infants admitted in 2014, making the data now over 10 years old. Much has changed in neonatal practice (e.g. increased AKI awareness, treatment strategies). Secondly, the findings of the AWAKEN study were geographically limited. While the AWAKEN study was multi-national and multi-center, it represented only 24 centers (22 from North America, 1 from India and 1 from Australia). Finally, information collected from AWAKEN ended at hospital discharge. The investigators seek to leverage the strength of the Neonatal Kidney Collaborative along with other organizations and collaboratives interested in neonatal kidney health to address these gaps. Therefore, the investigators are conducting a second, modified iteration of this study entitled "AWAKEN 2.0". AWAKEN 2.0 will be a multi-center multi-national retrospective analysis utilizing similar methodology to the AWAKEN study.

Начало: 01.03.2025 Край: 01.12.2026 Възраст: до 2 г.

Australia, Brazil, Canada +16 Indiana University NCT06823115

By invitation only Phase 3

A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)

Developmental and Epileptic Encephalopathy

This (DEEp OLE Study) is a multicentre, open-label study to investigate the long-term safety, efficacy, tolerability, and pharmacokinetics (PK) of LP352 in the treatment of seizures in children and adults with DEE who completed Study LP352-301 or LP352-302. The study consists of 3 main phases: Screening, Titration period and Maintenance period, followed by a Taper period and Follow-Up. The total duration of the study will be approximately 14 months.

Начало: 12.02.2025 Край: 17.12.2027 Възраст: 2–66 г. Treatment

Australia, France, Latvia +3 Longboard Pharmaceuticals NCT06908226

By invitation only

GENomic Predictors in A Multi-Ethnic Population With Kidney Disease Study

Kidney Disease

To establish a prospective, longitudinal cohort of participants who can provide blood, tissue (including kidney histology), urine samples to establish a core biobank for kidney disease research. Results from this biobank will be matched to clinical outcomes to facilitate the discovery (and/or validation) of novel prognostic, predictive or diagnostic biomarkers important for kidney disease.

Начало: 10.02.2025 Край: 01.01.2050 Възраст: от 18 г.

Australia Western Sydney Local Health District NCT06828562

By invitation only Phase 1

Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)

Antiphospholipid Syndrome (APS) Bullous Pemphigoid (BP) Behçet's Syndrome (BS) Dermatomyositis (DM) +2

This is a long-term extension trial of RAY121 in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM) and immune thrombocytopenia (ITP).

Начало: 09.12.2024 Край: 30.04.2027 Възраст: 18–85 г. Treatment

Australia, Bulgaria, Canada +14 Chugai Pharmaceutical NCT06723106

By invitation only

PIECES - Towards Large-Scale Adaptation and Tailored Implementation of Primary Cancer Prevention Programs

Focus of Study: Tailored Implementation

The PIECES project aims to evaluate the Primary Cancer Prevention Implementation Toolkit (PCP-IT), an online implementation support toolkit that aids implementation teams in improving the implementation of primary cancer prevention programs (PCPs). The primary aim of the PIECES study is to support and learn from the process of selecting, adapting, and implementing existing evidence-based programs to improve implementation outcomes and by that, improve the reach and effectiveness of primary cancer prevention programmes in real-world settings. The study objectives are: 1. To assess whether the PCP-IT is usable and if implementation teams are satisfied with the tool; 2. To assess what works for whom in using the PCP-IT for selecting, adapting, and implementing PCP programs; 3. To assess whether the PCP-IT is feasible and effective in improving acceptability, adoption, penetration, and sustainable implementation of the PCP programs. The PCP-IT will be used by various implementation settings across 9 countries (8 European countries and Australia). A multi-site case comparison design is used to generate in-depth knowledge about the performance and working mechanisms of the PCP-IT. Similarities, differences, and patterns across the different implementation sites will be investigated using a Realist Evaluation approach.

Начало: 01.12.2024 Край: 01.12.2026 Възраст: от 18 г. Other

Albania, Australia, Germany +6 Hidde van der Ploeg NCT06718322

By invitation only Phase 3

Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

Начало: 31.07.2024 Край: 01.02.2029 Treatment

Australia, Canada, France +5 Ultragenyx Pharmaceutical Inc NCT06415344

By invitation only Phase 2

Long-term Safety and Tolerability of BHV-7000

Focal Epilepsy

A study to determine if BHV- 7000 is safe and tolerable in adults with refractory focal onset epilepsy

Начало: 30.07.2024 Край: 01.01.2027 Възраст: 18–75 г. Treatment

Argentina, Australia, Belgium +20 Biohaven Therapeutics Ltd. NCT06443463

By invitation only

Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease

Atherosclerotic Cardiovascular Disease Heterozygous Familial Hypercholesterolemia Hypercholesterolemia

LTF-001 is a long-term follow-up study of participants who received an investigational gene-editing therapy developed by the sponsor to evaluate the long-term effects of the investigational therapy. Participants will be followed for a total of 15 years after the first administration of the gene-editing therapy.

Начало: 09.06.2024 Край: 01.10.2039 Възраст: от 18 г.

New Zealand, United Kingdom Verve Therapeutics, Inc. NCT06112327

By invitation only

Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002

Hereditary Angioedema

This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.

Начало: 12.04.2024 Край: 01.04.2040

Australia, France, Germany +3 Intellia Therapeutics NCT06262399

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