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NCT ID: NCT04285086 Active, not recruiting - Clinical trials for Essential Thrombocythemia

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

SURPASS ET
Start date: August 25, 2020
Phase: Phase 3
Study type: Interventional

This is a Phase 3 open-label, multicenter, randomized, active-controlled study designed to compare the efficacy and safety and tolerability of P1101 compared with ANA after 12 months of treatment as second-line therapy for subjects with ET who have had a suboptimal or failed response to HU.

NCT ID: NCT04281537 Completed - Fabry Disease Clinical Trials

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

Start date: March 1, 2020
Phase:
Study type: Observational

This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study will comprise a prospective time and motion evaluation and a cross-sectional evaluation of patient and caregiver-reported outcomes. The study will evaluate the time associated with the preparation and administration of a single dose of ERT in patients by health care providers as well as the impact on Fabry patients and caregivers time and costs associated with an ERT treatment. The study will also evaluate the patients' quality of life wellbeing, fatigue and work productivity.

NCT ID: NCT04281485 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Start date: November 5, 2020
Phase: Phase 3
Study type: Interventional

The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.

NCT ID: NCT04281472 Completed - Clinical trials for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)

ADHERE
Start date: April 15, 2020
Phase: Phase 2
Study type: Interventional

This is a Phase 2 study to evaluate the safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.

NCT ID: NCT04280718 Active, not recruiting - Clinical trials for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)

ADHERE+
Start date: September 18, 2020
Phase: Phase 2
Study type: Interventional

This is the open-label extension study of phase II ARGX-113-1802 to evaluate the long-term safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP. Patients already stabilized on efgartigimod PH20 SC will also have the opportunity to participate in a sub study to explore less frequent dosing of efgartigimod PH20 SC.

NCT ID: NCT04280705 Completed - COVID-19 Clinical Trials

Adaptive COVID-19 Treatment Trial (ACTT)

Start date: February 21, 2020
Phase: Phase 3
Study type: Interventional

This study is an adaptive, randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of novel therapeutic agents in hospitalized adults diagnosed with COVID-19. The study is a multicenter trial that will be conducted in up to approximately 100 sites globally. The study will compare different investigational therapeutic agents to a control arm. There will be interim monitoring to introduce new arms and allow early stopping for futility, efficacy, or safety. If one therapy proves to be efficacious, then this treatment may become the control arm for comparison(s) with new experimental treatment(s). Any such change would be accompanied by an updated sample size. Because background standards of supportive care may evolve/improve over time as more is learned about successful management of COVID-19, comparisons of safety and efficacy will be based on data from concurrently randomized subjects. An independent Data and Safety Monitoring Board (DSMB) will actively monitor interim data to make recommendations about early study closure or changes to study arms. To evaluate the clinical efficacy, as assessed by time to recovery, of different investigational therapeutics as compared to the control arm.

NCT ID: NCT04279847 Recruiting - Clinical trials for Myelodysplastic Syndrome

Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid Neoplasms

LIMBER
Start date: February 23, 2021
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of INCB057643 as monotherapy or combination with ruxolitinib for participants with myelofibrosis (MF) and other myeloid neoplasms.

NCT ID: NCT04278924 Active, not recruiting - Clinical trials for Primary Immune Thrombocytopenia

A Study of TAK-079 in Adults With Persistent/Chronic Primary Immune Thrombocytopenia

Start date: November 9, 2020
Phase: Phase 2
Study type: Interventional

Primary immune thrombocytopenia (ITP) is a rare disease that results in low levels of platelets - the cells that help blood clot. The main aim of the study is to check for side effects from taking TAK-079 at three different dose levels. Another aim is to learn if TAK-079 can increase the platelet count in people with ITP. In addition to receiving stable background therapy for ITP, participants will receive an injection of either TAK-079 or a placebo once a week for 2 months. A placebo looks like TAK-079 but will not have any medicine in it. After treatment, all participants will be followed-up for another 2 months. Then, participants who received TAK-079 will continue to be followed-up for an extra 4 months. Participants who received the placebo and would like to receive TAK-079 may be able to do this in an extension period in the study.

NCT ID: NCT04278417 Active, not recruiting - Clinical trials for Proliferative Diabetic Retinopathy

Study of Efficacy and Safety of Brolucizumab Versus Panretinal Photocoagulation Laser in Patients With Proliferative Diabetic Retinopathy

CONDOR
Start date: November 19, 2020
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of brolucizumab compared to panretinal photocoagulation laser (PRP) in patients with proliferative diabetic retinopathy (PDR). This evaluation will provide information that brolucizumab is non-inferior to PRP with respect to the change in best corrected visual acuity at Week 54.

NCT ID: NCT04277546 Terminated - Ulcerative Colitis Clinical Trials

Open-label Extension Study of Brazikumab in Ulcerative Colitis

Expedition
Start date: March 3, 2020
Phase: Phase 2
Study type: Interventional

The purpose of this OLE Study D5272C00002 (Legacy #3151-202-008) is to permit participants who previously enrolled in the double-blind Study D5272C00001 (Legacy #3151-201-008) to receive brazikumab, allowing for long-term observation of safety and efficacy in these participants treated with brazikumab. There are no formal hypotheses to be tested. Safety and efficacy data obtained in this study will be included in regulatory product submissions as appropriate.