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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.

BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy (REDEEM-2 Trial)

Status(es): Active, not recruiting
Study Date(s): Tuesday, October 26, 2021 to Thursday, August 31, 2023
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of this study is to evaluate the efficacy and safety of BCX9930 as a treatment for PNH in adult patients who are not currently being treated with a complement inhibitor. BCX9930 is a Factor D inhibitor administered orally (by mouth). The study will recruit approximately 57 patients worldwide and each individual’s participation will last approximately 1 year. The study will be run in two parts. In part 1, participants will be randomly assigned in a 2:1 ratio to either BCX9930 or placebo for 12 weeks. Participants will not know which treatment they are receiving in part 1. In part...

BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy (REDEEM-1 Trial)

Status(es): Active, not recruiting
Study Date(s): Tuesday, November 30, 2021 to Tuesday, October 31, 2023
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of this study is to evaluate the efficacy and safety of BCX9930 as a treatment for PNH in adult patients with ongoing anemia despite treatment with a C5 inhibitor (either eculizumab or ravulizumab). BCX9930 is a Factor D inhibitor administered orally (by mouth). The study will recruit approximately 81 patients worldwide and each individual’s participation will last approximately 1 year. The study will be run in two parts. In part 1, participants will be randomly assigned in a 2:1 ratio to either (a) stop their C5 inhibitor and start oral BCX9930 or (b) remain on the C5 inhibitor...

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 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).

PEGASUS: A Phase III Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH

Status(es): Completed
Study Date(s): Thursday, June 14, 2018
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of this study is to establish the efficacy and safety of APL-2 compared to eculizumab in patients with PNH who continue to have Hb levels <10.5 g/dL despite treatment with eculizumab (Soliris®). This study will enroll approximately 70 subjects around the globe to compare APL-2 to eculizumab treatment. All subjects who qualify will receive APL-2. For more information visit the Pegasus Study Website »

Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)

Status(es): Active, not recruiting
Study Date(s): Thursday, February 1, 2018 to Tuesday, December 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Patients with higher risk myelodysplastic syndromes (MDS) who fail current approved therapy or become refractory, have few therapeutic options other than bone marrow transplant, which may not be an option due to age, infirmity or lack of a donor. This is a Phase 1 open label, dose escalation clinical trial of Personalized, Adoptive immunotherapy by Cytotoxic T cells that are targeted to patient-specific cancer cell Neoantigens (PACTN) for such patients. It’s primary goal is to learn the safety and tolerability of PACTN infusion and secondarily, if PACTN show signs of being effective. The...

Randomized, Open-Label, Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment (ACCESS-1)

Status(es): Recruiting
Study Date(s): Sunday, July 31, 2022 to Friday, July 16, 2027
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The primary objective of the study is to evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 24-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with active Paroxysmal Nocturnal Hemoglobinuria (PNH) who are complement inhibitor treatment-naive or have not recently received complement inhibitor therapy.

SELECT-MDS-1, A Randomized, Double-blind, Placebo-controlled Study of Tamibarotene 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 tamibarotene (formerly 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...

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

Status(es): Recruiting
Study Date(s): Monday, December 19, 2016 to Thursday, August 31, 2023
Disease(s): aplastic anemia
Age Group: 2 years and older
People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may return in 1 in 3 people. The drug sirolimus may help by suppressing the immune system.
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