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NCT ID: NCT05168475 Active, not recruiting - Clinical trials for Giant Cell Arteritis

Biologics in Refractory Vasculitis

BIOVAS
Start date: July 14, 2021
Phase: Phase 2
Study type: Interventional

Vasculitis occur when the body's immune system, rather than protecting the body, attacks blood vessels, causing injury to the vessel and the part of the body it supplies with blood. Vasculitis is rare, and there are a number of different types, which can affect both adults and children. We treat vasculitis with steroids and drugs aiming to damp down the activity of the immune system, but they often cause side effects. Some patients do not improve with this treatment, or cannot tolerate it and their vasculitis worsens; this is known as refractory vasculitis. Patients with refractory vasculitis are at high risk of health complications from the disease and its therapy and are in need of newer more effective treatments with fewer side effects. Biologics are drugs which are designed to precisely target parts of the immune system and may have fewer side effects. Biologics have been used for several years to treat vasculitis, particularly anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis or AAV. However, for many of the rarer types of vasculitis, and especially those vasculitis disease types that are not ANCA-associated, there is little information to support use of biologic therapies as effective treatments. The purpose of this trial is to find out whether biologics are effective and represent value for money for participants with refractory vasculitis. The trial will include patients with Non-ANCA-associated vasculitis (NAAV)

NCT ID: NCT05168202 Active, not recruiting - Clinical trials for Myelodysplastic Syndromes

A Study to Assess the Effect of CC-95251 in Participants With Acute Myeloid Leukemia and Myelodysplastic Syndromes

Start date: January 19, 2022
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, and preliminary clinical activity of CC-95251 alone and in combination with antineoplastic agents in participants with relapsed or refractory acute myeloid leukemia and relapsed or refractory and treatment-naive higher risk melodysplastic syndromes.

NCT ID: NCT05167201 Active, not recruiting - Clinical trials for Chronic Obstructive Pulmonary Disease

Domiciliary Nasal High Flow and Patient Outcomes in Chronic Hypercapnic Respiratory Failure in the United Kingdom

Start date: March 28, 2022
Phase: N/A
Study type: Interventional

Chronic hypercapnic respiratory failure (CHRF) in the context of Chronic Obstructive Pulmonary Disease (COPD) and Obesity Hypoventilation Syndrome (OHS) is associated with increased mortality. The availability and effectiveness of domiciliary Non-invasive ventilation (NIV) treatment (when indicated) is key as this treatment can improve quality of life and reduce health-care costs from associated burden of disease. The emerging obesity epidemic means that there is now increased home mechanical ventilation set-ups in patients with obesity related respiratory failure (ORRF), yet there are no alternative treatments for patients struggling with domiciliary NIV. Domiciliary NHF has been shown to improve health related quality of life in stable CHRF in patients with COPD and improve cost effectiveness yet there are no current studies looking at the use of domiciliary NHF and its outcomes in ORRF. The study aims to deliver a pre and post intervention study evaluating patient reported and clinical outcomes in patients using NHF over twelve weeks, who have either COPD or OHS and have been unable to use domiciliary NIV. The study wishes to address key outcomes such as quality of life, clinical effectiveness, compliance and acceptability with the use of domiciliary NHF in both of these patient populations.

NCT ID: NCT05166265 Active, not recruiting - Clinical trials for Hearing Loss, Conductive

Clinical Investigation Study of Safety and Performance of the Sentio System.

Start date: January 19, 2022
Phase: N/A
Study type: Interventional

Prospective, open label, single-arm, multi-centre investigation, designed to follow clinical practice for Sentio bone conduction devices in 50 subjects, during 10 clinical visits, and 24 months follow-up.

NCT ID: NCT05163288 Active, not recruiting - Clinical trials for Niemann-Pick Disease, Type C

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Start date: June 30, 2022
Phase: Phase 3
Study type: Interventional

A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

NCT ID: NCT05162768 Active, not recruiting - Clinical trials for Mitochondrial Diseases

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

NuPower
Start date: April 29, 2022
Phase: Phase 3
Study type: Interventional

SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).

NCT ID: NCT05158296 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Start date: December 8, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

NCT ID: NCT05156320 Active, not recruiting - Clinical trials for Neuromuscular Diseases

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

SAPPHIRE
Start date: February 24, 2022
Phase: Phase 3
Study type: Interventional

This Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and are receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.

NCT ID: NCT05155579 Active, not recruiting - Malaria Clinical Trials

Assessment of Safety and Immunogenicity of a Single Vial Presentation of R21/Matrix-M and Co-Administration With EPI Vaccines

Start date: May 14, 2022
Phase: Phase 1
Study type: Interventional

This is a Phase Ib trial conducted in Bougouni, Mali to evaluate the safety and immunogenicity of R21/Matrix-M in a single and two vial presentation, with different immunisation schedules, and when co-administered with EPI vaccines in African children.

NCT ID: NCT05155553 Active, not recruiting - Clinical trials for Meningitis/Encephalitis

QIAstat-Dx® Meningitis/Encephalitis Panel Performance Evaluation Study

Start date: May 6, 2022
Phase:
Study type: Observational

Study for performance evaluation of the QIAstat-Dx® Meningitis/Encephalitis Panel in comparison with other chosen comparator methods.