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NCT ID: NCT05345223 Recruiting - Clinical trials for Triglyceride Deposit Cardiomyovasculopathy

A Registry Study on Primary/Idiopathic Triglyceride Deposit Cardiomyovasculopathy

Start date: March 31, 2022
Phase:
Study type: Observational

The objective of this study is to construct a registry; in order to understand the onset status of triglyceride deposit cardiomyovasculopathy (TGCV) in Japan with patients' background and natural history of the disease, and to explore disease-specific prognostic factors.

NCT ID: NCT05345171 Recruiting - OTC Deficiency Clinical Trials

Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

Start date: October 18, 2022
Phase: Phase 3
Study type: Interventional

The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

NCT ID: NCT05342493 Recruiting - Migraine Disorders Clinical Trials

Specified Drug Use Survey on AJOVY Subcutaneous Injection for Long-term Use

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

To evaluate the long-term safety of AJOVY in patients under actual use conditions and to specifically evaluate cardiovascular events. In addition, information on efficacy will be collected.

NCT ID: NCT05337137 Recruiting - Clinical trials for Carcinoma, Hepatocellular

A Study of Nivolumab and Relatlimab in Combination With Bevacizumab in Advanced Liver Cancer

RELATIVITY-106
Start date: May 5, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety and effectiveness of triplet therapy of nivolumab, relatlimab and bevacizumab versus nivolumab and bevacizumab in participants with untreated advanced/metastatic hepatocellular carcinoma (HCC).

NCT ID: NCT05335876 Recruiting - Clinical trials for Spinal Muscular Atrophy (SMA)

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

SPECTRUM
Start date: December 19, 2022
Phase: Phase 3
Study type: Interventional

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 15 years from the date of OAV101 administration in the previous clinical trial.

NCT ID: NCT05335499 Recruiting - Clinical trials for Chronic Spontaneous Urticaria

A Phase 2a Study of TAS5315 in Patients With Chronic Spontaneous Urticaria

Start date: June 28, 2022
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of TAS5315 in chronic spontaneous urticaria patients with an inadequate response to H1-antihistamines

NCT ID: NCT05334368 Recruiting - Clinical trials for Hypereosinophilic Syndrome

Depemokimab in Participants With Hypereosinophilic Syndrome, Efficacy, and Safety Trial

DESTINY
Start date: September 6, 2022
Phase: Phase 3
Study type: Interventional

This is a 52-week, randomized, placebo-controlled, double-blind, parallel group, multicenter study of depemokimab in adults with uncontrolled HES receiving standard of care (SoC) therapy. The study will recruit patients with a confirmed diagnosis of HES and who are on stable HES therapy for at least 4 weeks prior to randomization (Visit 2). Eligible participants must have uncontrolled HES with a history of repeated flare (≥2 flares in the previous 12 months) and blood eosinophil count of ≥1,000 cells/ microliter (μL) during Screening. Historical HES flares are defined as documented HES-related worsening of clinical symptoms or blood eosinophil counts requiring an escalation in therapy. Participants who meet the inclusion and exclusion criteria will be randomized in a 2:1 ratio to receive either depemokimab or placebo while continuing their SoC HES therapy.

NCT ID: NCT05330325 Recruiting - Clinical trials for SGA, Turner Syndrome, Noonan Syndrome, ISS

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow

REAL 8
Start date: August 10, 2022
Phase: Phase 3
Study type: Interventional

The study compares two medicines for treatment of children born small and who stay small, or with Turner Syndrome, Noonan Syndrome, or idiopathic short stature. The purpose of the study is to see how well treatment with somapacitan works compared to treatment with Norditropin®. Somapacitan is a new medicine, and Norditropin® is a medicine doctors can already prescribe in some countries. The study will last for about 3 years. The participants will either get somapacitan once a week for 3 years or Norditropin® once a day for 1 year followed by somapacitan once a week for 2 years. Which treatment the participants get is decided by chance.

NCT ID: NCT05327946 Recruiting - Solid Tumors Clinical Trials

A Study in People With Advanced Cancer to Test How Well Different Doses of BI 770371 Alone or in Combination With Ezabenlimab Are Tolerated

Start date: May 9, 2022
Phase: Phase 1
Study type: Interventional

This study is open to adults with advanced cancer (solid tumours). People for whom previous treatment was not successful and for whom no other treatment options exist can join the study. The purpose of this study is to find the highest dose of BI 770371 that people with advanced cancer can tolerate when taken alone or together with a medicine called ezabenlimab. BI 770371 and ezabenlimab are antibodies that may help the immune system fight cancer (checkpoint inhibitors). In this study, BI 770371 is given to people for the first time. Participants get BI 770371 alone or together with ezabenlimab as an infusion every 3 weeks. It is planned that participants can stay in the study for up to 2 years, if they benefit from treatment and can tolerate it. During this time, they visit the study site regularly. The doctors collect information on any health problems of the participants. The doctors also regularly monitor the size of the tumour.

NCT ID: NCT05327894 Recruiting - Clinical trials for Acute Lymphoblastic Leukemia

Interfant-21 Treatment Protocol for Infants Under 1 Year With KMT2A-rearranged ALL or Mixed Phenotype Acute Leukemia

Interfant-21
Start date: December 15, 2022
Phase: Phase 3
Study type: Interventional

This study is a treatment protocol with blinatumomab for infants under 1 year old who are diagnosed with acute lymphoblastic leukemia with a specific unfavorable genetic alteration. The purpose of the study is to improve the outcome of this disease in infants.