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Clinical Trials

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.

(COMMODORE 1) A Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

Status(es): Recruiting
Study Date(s): Wednesday, September 30, 2020 to Friday, October 25, 2024
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 12 years and older
The purpose of the study is to evaluate the efficacy and safety of crovalimab compared with eculizumab in adult and adolescent participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) treated with complement inhibitors. This clinical trial is recruiting people with a diagnosis of PNH, a blood disorder that involves the breakdown of red blood cells, and that receive treatment with eculizumab or ravulizumab before starting the clinical trial. This study will enroll approximately 250 participants around the globe and will assign participants into two different groups receiving Crovalimab or...

(COMMODORE 2) A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors

Status(es): Recruiting
Study Date(s): Thursday, October 8, 2020 to Friday, October 25, 2024
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 12 years and older
The purpose of the study is to evaluate the efficacy and safety of Crovalimab versus Eculizumab in adult and adolescent participants With Paroxysmal Nocturnal Hemoglobinuria (PNH), a blood disorder that involves the breakdown of red blood cells, not previously treated with complement inhibitors. The study will enroll approximately 200 participants around the globe and will assign participants in different groups receiving Eculizumab or Crovalimab in a ratio of 2:1 (which means that for each participant receiving Crovalimab, two participants will receive Eculizumab)

(Verona) A Randomized, Double-Blind, Phase 3 Study Evaluating the Safety and Efficacy of Venetoclax in Combination with Azacitidine in Patients Newly Diagnosed with Higher-Risk Myelodysplastic Syndromes (Higher-Risk MDS)

Status(es): Recruiting
Study Date(s): Thursday, September 10, 2020 to Friday, February 14, 2025
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Myelodysplastic Syndromes (MDS) are a group of serious, rare bone marrow disorders where the body no longer makes enough healthy, normal blood cells in the bone marrow. MDS may progress to acute myeloid leukemia (AML). AML is a type of cancer that affects the blood and bone marrow. Currently, there are very few treatment options for Higher-Risk MDS patients. Azacitidine is one of the approved treatments. The Verona study is a Phase 3, global, randomized, double-blind study to assess the safety and efficacy of venetoclax (an investigational drug) in combination with azacitidine, compared to...

A phase I/II Study of REGN7257 (Anti-interleukin 2 receptor subunit gamma [IL2RG] monoclonal antibody) in patients with severe aplastic anemia that is refractory to or relapsed on immunosuppressive therapy

Status(es): Recruiting
Study Date(s): Friday, August 21, 2020 to Thursday, July 20, 2023
Disease(s): aplastic anemia
Age Group: 18 years and older
The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with severe aplastic anemia (SAA) that is refractory to or has relapsed while on standard of care immunosuppressive therapy (IST). An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in IST-refractory/relapsed patients. The secondary objectives of this study are to assess the following for REGN7257: Clinical response over time Maintenance of response Impact on transfusion requirements Effect on blood counts and cell populations Pharmacokinetics...

A Randomized, Double-blind, Placebo-controlled Phase 3 Study of SY-1425 Plus Azacitidine Versus Placebo Plus Azacitidine in Newly Diagnosed, RARA-positive Adult Patients With Higher-risk Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Monday, February 8, 2021 to Monday, July 10, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This study compares the efficacy of SY-1425 in combination with azacitidine to azacitidine in combination with placebo in participants who are Retinoic Acid Receptor Alpha (RARA) positive, and newly diagnosed with higher-risk myelodysplastic syndrome (MDS), and who have not received treatment for this diagnosis. The primary goal of the study is to compare the complete remission rate between the two treatment arms. A subset of participants have MDS characterized by an overexpression of the RARA gene. A blood test will be used to identify participants with RARA-positive MDS. Assessment of the...

A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment

Status(es): Recruiting
Study Date(s): Thursday, August 8, 2019 to Monday, May 15, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
IMerge is a two-part Phase 2/3 clinical trial of imetelstat in transfusion dependent patients with lower risk MDS who are relapsed after or refractory to erythroid stimulating agents (ESAs). The Phase 3 portion is planned to enroll approximately 170 patients in a randomized (2:1 ratio of imetelstat:placebo), double-blind, placebo-controlled clinical trial to test the hypothesis that imetelstat improves the rate of red blood cell transfusion independence (TI). The trial is planned to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia. The...

ALPHA Study: Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)

Status(es): Recruiting
Study Date(s): Wednesday, January 6, 2021 to Saturday, October 21, 2023
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH. Participants will be assigned to different groups receiving danicopan on top of their C5 inhibitor (eculizumab or ravulizumab) or placebo (a sugar pill) in a ratio of 2:1 (which means that for every participant receiving placebo, two participants will receive danicopan ). At Week 12, participants who received placebo in the first 12 weeks will be switched to receive danicopan in...

APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)

Status(es): Active, not recruiting
Study Date(s): Friday, January 11, 2019 to Sunday, November 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
A Phase III, multicenter, randomized study to compare the rate of complete response (CR) and duration of CR, in patients with TP53-mutated MDS who will receive APR-246 and azacitidine or azacitidine alone. The p53, or P53, protein is coded by the TP53 gene which is mutated in a portion of patients with MDS. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndromes (MDS) with a susceptible TP53 mutation. For more information, please see: https://ir.aprea.com/news-releases/...

Ivosidenib (AG-120) in Patients with IDH1-mutant Relapsed/Refractory Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Friday, September 6, 2019 to Thursday, December 31, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The study is a sub-study of a Phase 1, dose escalation and expansion study of ivosidenib in patients with mIDH1 advanced hematologic malignancies (NCT02074839) and will be evaluating ivosidenib in subjects with mIDH1 relapsed/refractory (R/R) myelodysplastic syndrome (MDS). Patients must have R/R disease after treatment with standard agents indicated for MDS and high disease burden. The objective of the study is to evaluate the safety, clinical activity, tolerability, pharmacokinetics, and pharmacodynamics of treatment with ivosidenib in patients with mIDH1 R/R MDS.

Low Dose Danazol for the Treatment of Telomere Related Diseases

Status(es): Recruiting
Study Date(s): Thursday, March 1, 2018 to Monday, February 28, 2022
Disease(s): aplastic anemia
Age Group: 3 years and older
DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers at the National Institutes of Health (NIH) are studying if the medication danazol can be used to treat people with short telomere disease who also have bone marrow failure, liver, or lung disease.In recent studies, danazol, at high doses, showed a positive influence on telomere length. Additional research is needed to learn more...
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