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NCT ID: NCT05109234 Enrolling by invitation - Clinical trials for Childhood Absence Epilepsy

A Study to Test the Long-term Safety, Tolerability and Efficacy of Brivaracetam in Study Participants 2 to 26 Years of Age With Childhood Absence Epilepsy or Juvenile Absence Epilepsy

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

The purpose of the study is to investigate the long-term safety, tolerability and efficacy of brivaracetam in pediatric study participants with childhood absence epilepsy (CAE) or juvenile absence epilepsy (JAE).

NCT ID: NCT05100862 Recruiting - Clinical trials for Marginal Zone Lymphoma

A Study of Zanubrutinib Plus Anti-CD20 Versus Lenalidomide Plus Rituximab in Participants With Relapsed/Refractory Follicular or Marginal Zone Lymphoma

MAHOGANY
Start date: March 10, 2022
Phase: Phase 3
Study type: Interventional

The purpose of the study is to compare the efficacy of zanubrutinib plus obinutuzumab versus lenalidomide plus rituximab (R^2) in participants with relapsed/refractory (R/R) follicular lymphoma (FL), as measured by progression-free survival as determined by an independent review committee in accordance with the 2014 modification of the International Working Group on non-Hodgkin lymphoma (NHL) Criteria based on n positron emission tomography and computed tomography (PET/CT), and to compare the efficacy of zanubrutinib plus rituximab versus R^2 in participants with R/R marginal zone lymphoma (MZL), as measured by progression free survival (PFS) assessed by IRC in accordance with CT-based Lugano 2014 Criteria.

NCT ID: NCT05099640 Completed - Phenylketonuria Clinical Trials

A Study of PTC923 in Participants With Phenylketonuria

Start date: September 30, 2021
Phase: Phase 3
Study type: Interventional

The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (that is, the average of each respective treatment dose 2-week period of double-blind treatment).

NCT ID: NCT05096403 Suspended - Clinical trials for Cold Agglutinin Disease

A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)

Start date: October 20, 2022
Phase: Phase 3
Study type: Interventional

The purpose of the study is to determine the efficacy of pegcetacoplan administration compared to placebo in increasing hemoglobin (Hgb) level from baseline and avoiding transfusion in participants with primary cold agglutinin disease (CAD).

NCT ID: NCT05092776 Recruiting - Clinical trials for Familial Mediterranean Fever

Efficacy and Safety of RPH-104 for Resolution and Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine

Start date: April 29, 2021
Phase: Phase 2
Study type: Interventional

The primary purpose of this study is to assess the efficacy and safety profiles of investigational product RPH-104 (R-Pharm Overseas, Inc., USA) for treatment of Familial Mediterranean Fever (FMF) in adult patients resistant/intolerant to colchicine (crFMF). Pharmacokinetic and pharmacodynamic parameters of RPH-104 single or multiple doses in this patient population will be assessed as well.

NCT ID: NCT05085002 Terminated - Clinical trials for Advanced Breast Cancer

A Study of Lerociclib in Participants With Advanced Breast Cancer

Start date: December 21, 2021
Phase: Phase 2
Study type: Interventional

This is a multicenter, single-arm, open-label study to evaluate the safety and efficacy of lerociclib in combination with standard endocrine therapy in female or male participants with HR+/HER2- MBC. The study population will consist of either newly diagnosed, treatment naïve participants with HR+/HER2- MBC (1L population) and participants with HR+/HER2- MBC who have already progressed on first line endocrine therapy such as tamoxifen, anastrozole, or letrozole (2L population). All premenopausal or perimenopausal female participants, and all male participants, must be receiving goserelin for at least 28 days prior to entering the study and will remain on goserelin throughout the study, in accordance with the prescribing information and according to the study site's standard practice.

NCT ID: NCT05084261 Completed - Ulcerative Colitis Clinical Trials

An Investigation of Oral BT051 in Subjects With Moderately to Severely Active Ulcerative Colitis (UC)

SCOUT
Start date: November 30, 2021
Phase: Phase 1
Study type: Interventional

This is a randomised, double-blind, placebo-controlled study to assess the safety and tolerability of multiple ascending doses of BT051 in subjects with moderately to severely active ulcerative colitis. Subjects will be randomised using a 3 active:1 placebo ratio to 3 ascending dose cohorts of 8 subjects and will be dosed daily for 28 days. The 3 initial dose levels will be 200 mg, 800 mg and 3200 mg per day. Progression to the next cohort will be based on the safety and tolerability of the previous cohort.

NCT ID: NCT05083364 Recruiting - IgA Nephropathy Clinical Trials

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease

Start date: February 2, 2022
Phase: Phase 1
Study type: Interventional

The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with complement-mediated renal disease (C3 Glomerulopathy [C3G] and IgA Nephropathy [IgAN]). In Part 1 of the study, HVs will receive either one or two doses of ARO-C3 or placebo. In Part 2 of the study, adult patients with C3G/IgAN will receive 3 open-label doses of ARO-C3. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

NCT ID: NCT05070858 Recruiting - Clinical trials for Generalized Myasthenia Gravis

A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis

NIMBLE
Start date: December 14, 2021
Phase: Phase 3
Study type: Interventional

The primary objective is: To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG) The secondary objectives of the study are: - To evaluate the effect of pozelimab + cemdisiran (ie, combination) and cemdisiran monotherapy on: - Clinician-assessed signs of myasthenia gravis (MG) and muscle strength - Daily functioning that is impacted by signs and symptoms in patients with symptomatic gMG (cemdisiran monotherapy only). - Proportion of patients with improvements in daily function that is impacted by signs and symptoms of MG - Proportion of patients that have improvements in clinician-assessed signs of MG and muscle strength - Health related quality of life - Proportion of patients with minimal MG symptoms - Patient- and clinician-reported signs and symptoms of MG - To evaluate the safety and tolerability of pozelimab + cemdisiran and cemdisiran monotherapy - To assess the concentration of total pozelimab in serum - To assess the concentrations of cemdisiran and its metabolites in plasma - To assess the immunogenicity of pozelimab - To assess the concentration of total C5 in plasma - To assess the immunogenicity of cemdisiran - To study the effect of pozelimab + cemdisiran and cemdisiran monotherapy on complement activation

NCT ID: NCT05070364 Active, not recruiting - Clinical trials for Hepatitis Delta Virus

Phase 3 Study to Evaluate the Efficacy and Safety of Peginterferon Lambda for 48 Weeks in Patients With Chronic HDV

LIMT-2
Start date: December 21, 2021
Phase: Phase 3
Study type: Interventional

The Phase 3 LIMT-2 study will evaluate the safety and efficacy of Peginterferon Lambda treatment for 48 weeks with 24 weeks follow-up compared to no treatment for 12 weeks in patients chronically infected with HDV. The primary analysis will compare the proportion of patients with HDV RNA < LLOQ at the 24-week post-treatment visit in the Peginterferon Lambda treatment group vs the proportion of patients with HDV RNA < LLOQ at the Week 12 visit in the no-treatment comparator group.