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NCT ID: NCT05135559 Recruiting - Clinical trials for Haemophilia A and B With and Without Inhibitors

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

Explorer10
Start date: March 24, 2022
Phase: Phase 3
Study type: Interventional

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use. Participants will have to inject the study medicine every day under the skin with a pen-injector. The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.

NCT ID: NCT05133531 Recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

ACCESS-1
Start date: August 1, 2022
Phase: Phase 3
Study type: Interventional

This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on patients with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for patients with PNH and how the combination compares with 2 existing treatments, one called ravulizumab and the other called eculizumab. The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and eculizumab may also be called the "comparator drug". The study is looking at several research questions, including: - How effective is the pozelimab + cemdisiran combination compared to ravulizumab? - How effective is pozelimab + cemdisiran combination compared to eculizumab? - What side effects may happen from taking the study drugs? - How much study drugs are in the blood at different times? - Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects)

NCT ID: NCT05132582 Recruiting - Clinical trials for HER2 Positive Breast Cancer

A Study of Tucatinib or Placebo With Trastuzumab and Pertuzumab for Metastatic HER2+ Breast Cancer

HER2CLIMB-05
Start date: March 7, 2022
Phase: Phase 3
Study type: Interventional

This study is being done to see if tucatinib works better than placebo when given with other drugs to treat participants with HER2-positive breast cancer. A placebo is a pill that looks the same as tucatinib but has no medicine in it. This study will also test what side effects happen when participants take this combination of drugs. A side effect is anything a drug does to the body besides treating your disease. Participants will have cancer that has spread in the body near where it started (locally advanced) and cannot be removed (unresectable) or has spread through the body (metastatic). In this study, all participants will get either tucatinib or placebo. Participants will be assigned randomly to a group. This is a blinded study, so patients and their doctors will not know which group a participant is in. All participants will also get trastuzumab and pertuzumab. These are 2 drugs used to treat this type of cancer.

NCT ID: NCT05131828 Recruiting - Multiple Sclerosis Clinical Trials

CCMR Two: A Phase IIa, Randomised, Double-blind, Placebo-controlled Trial of the Ability of the Combination of Metformin and Clemastine to Promote Remyelination in People With Relapsing-remitting Multiple Sclerosis Already on Disease-modifying Therapy

Start date: March 8, 2022
Phase: Phase 2
Study type: Interventional

The greatest unmet need for people with multiple sclerosis is an effective therapy for the progressive phase. Current treatments suppress the damage caused by the immune system but there is only a limited window in which these can work. Consequently, much of the research community is turning its attention to the process of repair, called remyelination, in which the lining of nerve cells is reformed. This protects nerves from dying and therefore can delay, prevent, or even reverse, disability progression. It has previously been shown that stem cells are already present in the brain that can carry out this process. Clemastine, an anti-histamine drug, can instruct them to become active and has already shown a beneficial effect in a phase 2 trial. Now, more recent experiments have shown that changes take place within these stem cells as they age, which prevents them responding to drugs like clemastine, but that this can be reversed by treatment with metformin, a commonly used anti-diabetes drug. Our goal is to establish whether the combination of metformin and clemastine can promote remyelination in people with MS. We will focus on people with relapsing MS as they will have a greater proportion of nerves healthy enough to allow remyelination to take place, which will maximise the chance of detecting an effect with a smaller sample size. Participants will also continue treatment with a current disease-modifying MS treatment, to reduce the chance of developing new areas of damage, allowing the trial to focus on the repair process. The treatment duration will be 24 weeks, but given the established safety of the proposed medications, we are able to limit the number of visits to the trial centre to ensure participation is not overly burdensome.

NCT ID: NCT05131022 Recruiting - Clinical trials for Chronic Lymphocytic Leukemia (CLL)

A Study of NX-5948 in Adults With Relapsed/Refractory B-cell Malignancies

Start date: April 13, 2022
Phase: Phase 1
Study type: Interventional

This is a first-in-human Phase 1a/1b multicenter, open-label study designed to evaluate the safety and anti-cancer activity of NX-5948 in patients with advanced B-cell malignancies.

NCT ID: NCT05130437 Recruiting - Propionic Acidemia Clinical Trials

A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927

Start date: November 9, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).

NCT ID: NCT05130320 Recruiting - Clinical trials for Coronavirus Antibody Levels

Severe Asthma Covid Vaccine Response Study

SHINE
Start date: May 6, 2021
Phase:
Study type: Observational

This study aims to employ a simple finger-prick home test to detect post vaccination antibody levels. The investigators will compare antibody responses in patients with severe asthma on varying treatment regimes (biologics, daily steroids, inhalers-only) with healthy, age-matched controls to study if the magnitude and range of responses vary between severe asthmatics and healthy individuals.

NCT ID: NCT05127278 Recruiting - Psychosis Clinical Trials

E-Detection Tool for Emerging Mental Disorders

ENTER
Start date: April 20, 2022
Phase:
Study type: Observational

Psychosis is a severe mental disorder that involves abnormal experiences and altered behaviour. Although the onset of psychosis occurs in young people, most cases will only be identified later in the course of illness. The delayed detection is the main cause of significant negative long-term outcomes. The current proposal will develop an innovative E-mental health detection tool to effectively improve the identification of young people with emerging psychosis. This tool will be implemented on digital platforms including websites, tablets and mobile phones. Young people will be asked to complete an online questionnaire and two short online-exercises and will then be invited for face-to-face assessments to further confirm whether they are experiencing an emerging psychosis. This digital approach leverages previous expertise by the research team and it is expected to provide novel avenues to enable the identification of young people with emerging psychosis.

NCT ID: NCT05126901 Recruiting - Anemia Clinical Trials

Evaluate the Safety and Efficacy of Ferric Maltol Oral Suspension vs. Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, With a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years

FORTIS
Start date: October 4, 2021
Phase: Phase 3
Study type: Interventional

The objective of the study is to compare the safety and gastrointestinal tolerability of ferric maltol oral suspension and ferrous sulfate oral liquid in children and adolescents aged 2 years to 17 years, and assess the safety and tolerability of ferric maltol oral suspension in children 1 month to less than 2 years, in the treatment of iron deficiency anaemia during the 12 weeks treatment period.

NCT ID: NCT05126277 Recruiting - Lupus Nephritis Clinical Trials

Safety, Efficacy and Tolerability of Ianalumab Versus Placebo, Combination With SoC Therapy, in Participants With Active Lupus Nephritis

SIRIUS-LN
Start date: July 14, 2022
Phase: Phase 3
Study type: Interventional

This trial will evaluate efficacy, safety, and tolerability of subcutaneous (s.c.) ianalumab given every 4 weeks (q4w) or every 12 weeks (q12w) compared to placebo, in combination with SoC, in adult participants with active LN