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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 Saturday, September 8, 2029
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, June 2, 2028
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)

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 Study of APG-115 Alone or Combined With Azacitidine in Patients With AML, CMML, or MDS

Status(es): Recruiting
Study Date(s): Friday, April 24, 2020 to Thursday, April 24, 2025
Disease(s): acute myeloid leukemia (AML), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a two-part study in patients with relapsed/refractory acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), or high risk myelodysplastic syndrome (MDS) that will initially evaluate the safety and tolerability of APG-115 as a single agent in Part 1, followed by a combination of APG-115 + azacitidine in Part 2.

A Study of APG-2575 in Combination With Azacitidine in Patients With Acute Myeloid Leukemia (AML)

Status(es): Recruiting
Study Date(s): Friday, July 16, 2021 to Wednesday, July 29, 2026
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is a Phase Ib/II, open-label, multi-center study evaluating the safety, tolerability, efficacy and pharmacokinetics (PK) of APG-2575 in combination with azacitidine in the patients with AML or MDS/CMML. The study consists of dose escalation (Part I) and dose expansion phase (Part II).

A Study of KER-050 to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes

Status(es): Recruiting
Study Date(s): Wednesday, August 19, 2020 to Sunday, November 30, 2025
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
KER-050 is an investigational therapeutic protein designed to increase red blood cell and platelet production by inhibiting the signaling of a subset of the transforming growth factor beta (TGF-ß) family of proteins to promote hematopoiesis. The primary objective of Part 1 of this study is to evaluate the safety and tolerability of ascending doses of KER-050 in participants with very low, low, or intermediate risk myelodysplastic syndromes (MDS) to determine the dose(s) that will be evaluated for safety and tolerability in Part 2 of the study. Secondary and exploratory objectives of this...

CLNP023C12303 (APPULSE)

Status(es): Recruiting
Study Date(s): Saturday, April 1, 2023 to Saturday, June 1, 2024
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: Greater than or equal to 18 yrs
The purpose of the study is to find out if the investigational treatment, iptacopan, is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switch from their current standard of care treatment (eculizumab or ravulizumab) to study treatment, iptacopan/LNP023.   

Low Dose Danazol for the Treatment of Telomere Related Diseases

Status(es): Recruiting
Study Date(s): Thursday, March 1, 2018 to Thursday, April 30, 2026
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...

Optimizing Haploidentical Aplastic Anemia Transplantation (CHAMP)

Status(es): Recruiting
Study Date(s): Friday, May 19, 2017 to Tuesday, February 1, 2022
Disease(s): aplastic anemia
Age Group: up to 76 years
Assess overall survival (OS) at 1 year post-hematopoietic stem cell transplantation (HSCT) from a haploidentical marrow donor in patients with severe aplastic anemia (SAA).

PIONEER: A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status(es): Recruiting
Study Date(s): Thursday, February 4, 2021 to Tuesday, October 1, 2024
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 12 years to 17 years
The purpose of this open-label study is to evaluate the safety, effectiveness, and biologic activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds who have paroxysmal nocturnal hemoglobinuria (PNH). 

The study will consist of a 4-week screening period followed by a 16-week treatment period. Participants switching from a C5 inhibitor will have an additional 4-week run-in period between the screening and treatment periods. At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-...

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any clinical trial listed on this website. Pharmaceutical company sponsored content is highlighted only to give additional information about the trial. All trials are listed on https://clinicaltrials.gov/. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a clinical trial, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Further Reading

Patients should learn as much as possible about a clinical trial before making the decision to participate.

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