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NCT ID: NCT05076175 Recruiting - Colitis, Ulcerative Clinical Trials

A Study Investigating Oral Ozanimod (RPC1063) in Pediatric Participants With Moderate to Severe Active Ulcerative Colitis

Start date: May 30, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to evaluate the effectiveness and safety of ozanimod (RPC1063) in achieving and maintaining clinical remission. Ozanimod will be administered orally to pediatric participants with moderate to severe active ulcerative colitis (UC) who have had an inadequate response to conventional therapy.

NCT ID: NCT05074550 Recruiting - Polycythemia Vera Clinical Trials

Safety Evaluation Study for Patients With Polycythemia Vera

Start date: August 24, 2022
Phase: Phase 1
Study type: Interventional

This is PhaseI Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of PPMX-T003 in Polycythemia Vera

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: NCT05067790 Recruiting - Clinical trials for Spinal Muscular Atrophy

A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam

ASCEND
Start date: January 21, 2022
Phase: Phase 3
Study type: Interventional

The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam. The secondary objective of this study is to evaluate the safety and tolerability of HD nusinersen in participants with SMA previously treated with risdiplam.

NCT ID: NCT05067283 Recruiting - Clinical trials for Advanced Solid Tumors

A Study of MK-1084 in KRAS Mutant Advanced Solid Tumors (MK-1084-001)

Start date: December 17, 2021
Phase: Phase 1
Study type: Interventional

This is a study evaluating the safety, pharmacokinetics, and efficacy of MK-1084 alone, and MK-1084 plus other combination therapies in participants with advanced solid tumors with identified kirsten rat sarcoma viral oncogene homolog G12C (KRAS G12C) mutation.

NCT ID: NCT05064878 Recruiting - Clinical trials for CDKL5 Deficiency Disorder

A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder

Start date: March 8, 2022
Phase: Phase 3
Study type: Interventional

This is a multicenter, double-blind, parallel-group, placebo controlled, 2-part study to evaluate the efficacy and safety of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).

NCT ID: NCT05064618 Recruiting - Pancreatic Cancer Clinical Trials

Investigator-initiated Clinical Trial of MIKE-1

MIKE-1
Start date: August 23, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

To evaluate the safety and tolerability of Am80(Generic name: Tamibarotene, Development code: MIKE-1) in combination with gemcitabine (GEM) and nab-paclitaxel (nab-PTX) in patients with unresectable pancreatic cancer and to determine the recommended dose. Efficacy will also be exploratively investigated.

NCT ID: NCT05063162 Recruiting - Clinical trials for Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease (MOG-AD)

A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody-associated Disease (MOG-AD)

cosMOG
Start date: February 2, 2022
Phase: Phase 3
Study type: Interventional

The purpose of the study is to evalute the efficacy, safety and tolerability of rozanolixizumab for treatment of adult participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD).

NCT ID: NCT05053139 Recruiting - Haemophilia A Clinical Trials

A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors

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

This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). When and how often participants will receive Mim8 is dependent on their previous treatment - but is otherwise decided by chance. Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month. The study will last 54-124 weeks (12-29 months) depending on how long participants will be followed in run-in before they start treatment and if they continue in the follow period or transfer to an open label extension study. Participants will have 12-17 clinic visits.

NCT ID: NCT05052801 Recruiting - Gastric Cancer Clinical Trials

Bemarituzumab or Placebo Plus Chemotherapy in Gastric Cancers With Fibroblast Growth Factor Receptor 2b (FGFR2b) Overexpression

FORTITUDE-101
Start date: March 7, 2022
Phase: Phase 3
Study type: Interventional

The main objective of this study is to compare efficacy of bemarituzumab combined with oxaliplatin, leucovorin, and 5-fluorouracil (5-FU) (mFOLFOX6) to placebo plus mFOLFOX6 as assessed by overall survival (OS) in participants with FGFR2b ≥10% 2+/3+ tumor cell staining (FGFR2b ≥10% 2+/3+TC)