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NCT ID: NCT05132569 Terminated - Myasthenia Gravis Clinical Trials

Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia Gravis

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

This was a multicenter, randomized, double-blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of tolebrutinib compared with placebo in adult participants aged 18 to 85 years old with moderate-to-severe generalized myasthenia gravis (gMG), who received Standard of Care (SoC). The double-blind (DB) treatment period of 26 weeks comprised of 7 site visits followed by a 2-year open label extension (OLE) period with quarterly visits. The efficacy of tolebrutinib versus placebo during the DB period was assessed by clinical evaluations, including scales based on physician examination or direct participant feedback i.e., patient reported outcomes (PROs). These evaluations continued during the OLE to measure long term efficacy and safety.

NCT ID: NCT05129280 Terminated - Solid Tumors Clinical Trials

A Study to Evaluate Safety, Pharmacokinetics, and Preliminary Anti-tumor Activity of RO7444973 in Participants With Unresectable and/or Metastatic MAGE-A4-positive Solid Tumors

Start date: January 25, 2022
Phase: Phase 1
Study type: Interventional

This is a first-in-human, open-label, uncontrolled, multi-center, monotherapy dose-escalation and dose expansion study of RO7444973.The aim of this study is to evaluate the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RO7444973 in participants with unresectable and/or metastatic melanoma-associated antigen A4 (MAGE-A4)-positive, solid tumors, carrying the HLA-A*02:01 allele.

NCT ID: NCT05126303 Terminated - Acute Kidney Injury Clinical Trials

Efficacy and Safety of RMC-035 in Subjects at High Risk for Acute Kidney Injury Following Open-Chest Cardiac Surgery

AKITA
Start date: March 31, 2022
Phase: Phase 2
Study type: Interventional

This study evaluates RMC-035 compared to placebo for the prevention of acute kidney injury (AKI) in subjects who are at high risk for AKI following cardiac surgery. Half of the subjects will receive RMC-035 and the other half will receive placebo.

NCT ID: NCT05125068 Terminated - IgA Nephropathy Clinical Trials

Safety and Efficacy of AT-1501 in Patients With IgA Nephropathy (IgAN)

Start date: March 21, 2022
Phase: Phase 2
Study type: Interventional

This is a phase 2a, multicenter, open-label study designed to evaluate the safety and efficacy of AT-1501 in patients with biopsy proven IgAN and at least 0.75 g/24 hours of protein in their urine at the time of screening.

NCT ID: NCT05116774 Terminated - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)

BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy

REDEEM-1
Start date: December 6, 2021
Phase: Phase 2
Study type: Interventional

The purpose of this study is to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH patients with residual anemia despite treatment with a C5 inhibitor.

NCT ID: NCT05116046 Terminated - Achondroplasia Clinical Trials

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Start date: December 24, 2021
Phase: Phase 2
Study type: Interventional

All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll in this open-label extension (OLE) study for up to an additional 24 months of treatment. Approximately 63 participants will be offered to continue at the previously received dose of Recifercept either Low Dose Medium Dose High Dose or at the therapeutic dose once it is identified. Participants will attend the clinic monthly for 24 months. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements & patient/caregiver quality of life questionnaires.

NCT ID: NCT05088460 Terminated - Clinical trials for Familial Partial Lipodystrophy

A Study to Examine the Effects of the Leptin Receptor (LEPR) Agonist Antibody REGN4461 in Adult Patients With Familial Partial Lipodystrophy (FPLD)

LEAP
Start date: February 28, 2022
Phase: Phase 2
Study type: Interventional

Two cohorts are being studied based on leptin levels. Cohort A is composed of patients with baseline leptin <8.0 ng/mL and Cohort B is composed of patients with baseline leptin 8.0 to ≤20.0 ng/mL The primary objectives will be evaluated for patients in Cohort A only: - To evaluate the effect of REGN4461 on fasting triglycerides (TG) in patients with elevated baseline fasting TG - To evaluate the effect of REGN4461 on hyperglycemia in patients with elevated baseline Hemoglobin A1c (HbA1c) The following secondary objectives of the study will be evaluated for Cohort B and for the combined set of Cohorts A plus B: - To evaluate the effect of REGN4461 on fasting TG levels in patients with hypertriglyceridemia - To evaluate the effect of REGN4461 on glycemic control in patients with hyperglycemia The following secondary objectives of the study will be evaluated for Cohorts A and B separately, and for the combined set of Cohorts A plus B: - To evaluate the effect of REGN4461 on liver fat in patients with hepatic steatosis - To evaluate the effect of REGN4461 on hunger - To evaluate safety and tolerability of REGN4461 - To characterize the concentration profile of REGN4461 over time - To assess immunogenicity to REGN4461

NCT ID: NCT05086744 Terminated - Clinical trials for Immune Thrombocytopenia (ITP)

Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

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

The main purpose of this study is to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.

NCT ID: NCT05079230 Terminated - Clinical trials for Acute Myeloid Leukemia

Study of Magrolimab Versus Placebo in Combination With Venetoclax and Azacitidine in Participants With Acute Myeloid Leukemia

ENHANCE-3
Start date: July 7, 2022
Phase: Phase 3
Study type: Interventional

The goal of this clinical study is to compare the study drugs, magrolimab + venetoclax + azacitidine, versus placebo + venetoclax + azacitidine in participants with untreated acute myeloid leukemia (AML) who are not able to have chemotherapy.

NCT ID: NCT05073458 Terminated - Clinical trials for Warm Autoimmune Hemolytic Anemia (wAIHA)

Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia

PATHWAY
Start date: March 15, 2022
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),