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NCT ID: NCT05348915 Active, not recruiting - Sickle Cell Disease Clinical Trials

A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

Start date: March 29, 2022
Phase: Phase 3
Study type: Interventional

This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.

NCT ID: NCT05089084 Active, not recruiting - Clinical trials for Familial Chylomicronemia

Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)

PALISADE
Start date: January 11, 2022
Phase: Phase 3
Study type: Interventional

The purpose of AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 plozasiran) in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of plozasiran or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive plozasiran.

NCT ID: NCT04948333 Active, not recruiting - Clinical trials for Chronic Myelogenous Leukemia

Asciminib Treatment Optimization in ≥ 3rd Line CML-CP.

Start date: October 13, 2021
Phase: Phase 3
Study type: Interventional

The purpose of the study is to optimize the treatment of asciminib in patients with chronic myelogenous leukemia in chronic phase (CML-CP) previously treated with 2 or more Tyrosine Kinase Inhibitors (TKIs). Patients for this study will be identified based on warning criteria and resistance definition following European Leukemia Network (ELN) 2020 recommendations. In addition, the study will investigate the use of two different posologies. For this, patients will receive asciminib 40 mg (twice-daily) BID or of 80 mg (once daily) once daily (QD).

NCT ID: NCT04935879 Active, not recruiting - Sickle Cell Disease Clinical Trials

A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

Start date: October 26, 2021
Phase: Phase 3
Study type: Interventional

This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD). Participants will be randomized to receive inclacumab or placebo.

NCT ID: NCT04266301 Active, not recruiting - Clinical trials for Myelodysplastic Syndromes

Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)

STIMULUS-MDS2
Start date: April 14, 2013
Phase: Phase 3
Study type: Interventional

This is a Phase III multi-center, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to azacitidine in adult subjects with intermediate, high or very high-risk myelodysplastic syndrome (MDS) as per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) who are not eligible for intensive chemotherapy or hematopoietic stem cell transplantation (HSCT) according to medical judgment by the investigator. The purpose of the current study is to assess clinical effects of MBG453 in combination with azacitidine in adult subjects with IPSS-R intermediate, high, very high risk MDS and CMML-2.

NCT ID: NCT04218084 Active, not recruiting - Sickle Cell Disease Clinical Trials

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

HOPE Kids 2
Start date: November 11, 2020
Phase: Phase 3
Study type: Interventional

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

NCT ID: NCT03938792 Active, not recruiting - Hemophilia A Clinical Trials

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Start date: March 9, 2020
Phase: Phase 3
Study type: Interventional

Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.

NCT ID: NCT03814746 Active, not recruiting - Clinical trials for Sickle Cell Disease (SCD)

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

STAND
Start date: July 26, 2019
Phase: Phase 3
Study type: Interventional

The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit.

NCT ID: NCT03782207 Active, not recruiting - Clinical trials for Non-Small Cell Lung Cancer

A Study Investigating the Outcomes and Safety of Atezolizumab Under Real-World Conditions in Patients Treated in Routine Clinical Practice

IMreal
Start date: February 7, 2019
Phase:
Study type: Observational

This is a non-interventional, multi-country, multi-centre, multiple cohort prospective study, with retrospective collection of prior medical/treatment history data from medical records, designed to assess the real-world outcomes and safety of atezolizumab for indications in the existing label in the real world setting of routine clinical practice.

NCT ID: NCT03573882 Active, not recruiting - Sickle Cell Disease Clinical Trials

Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031

034OLE
Start date: June 6, 2018
Phase: Phase 3
Study type: Interventional

Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials