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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.

STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS

Status(es): Recruiting
Study Date(s): Thursday, March 31, 2022 to Monday, March 31, 2025
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a single-arm, nonrandomized, open-label, Phase II multicenter study of intravenous MBG453 (sabatolimab) added to hypomethylating agents (HMA) of investigator's choice (IV/SC/ Oral) in adult participants with intermediate, high or very high risk myelodysplastic syndrome (MDS) as per IPSS-R criteria. The study will assess the safety and efficacy of sabatolimab (investigational drug) in combination with an HMA. The study will enroll approximately 90 participants in the United States. MBG453/sabatolimab is an investigational compound. Efficacy and safety have not been established. There...

Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment (APPLY-PNH)

Status(es): Active, not recruiting
Study Date(s): Monday, January 25, 2021 to Tuesday, January 31, 2023
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
This study will include participants 18 years old or older who have PNH and are taking anti-C5 antibody treatment and still have anemia (residual anemia). The study is testing how safe and effective the investigational treatment iptacopan/LNP023 is compared to anti-C5 antibody treatment. Participants will be randomized to 1 of the 2 treatment arms in a 8:5 ratio (which means that for every 8 participants receiving iptacopan/LNP023, 5 participants will receive anti-C5 antibody treatment) to either iptacopan/LNP023 monotherapy at a dose of 200 mg orally twice a day or intravenous anti-C5...

Study of PTC299 in Relapsed/Refractory Acute Leukemias

Status(es): Recruiting
Study Date(s): Monday, October 29, 2018 to Wednesday, November 25, 2020
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is an open-label, non-randomized, Phase 1b study to evaluate the safety, pharmacokinetics (PK) profiles, and preliminary evidence of antitumor activity of PTC299 and the metabolite, O-desmethyl PTC299, in participants with relapsed/refractory acute myeloid leukemia (AML) who have exhausted standard available therapies known to provide clinical benefit. The study is designed as a series of cohort-based dose escalations. For each cohort, a minimum of 3 evaluable participants with PK and safety data will be assessed. Additional participants will be recruited if additional PK data are...

The National Myelodysplastic Syndromes Natural History Study

Status(es): Recruiting
Study Date(s): Friday, April 1, 2016 to Wednesday, September 1, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS).  Participants will be followed long term.  Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.
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