We have highlighted some clinical trials currently recruiting patients. Each has a specific purpose related to aplastic anemia, MDS, or PNH. Should you consider a clinical trial? Ask your doctor or contact the study coordinators for further information. Learn more in the Clinical Trials section.
If you only want to see studies that are currently recruiting patients, select that option from the “recruitment” line at the beginning of the advanced search. To locate a clinical trial in your state, use the “advanced search” feature. Under locations, select your state(s) and your country.
Search for aplastic anemia studies actively recruiting patients. The following are a sample of the 83 open studies recruiting aplastic anemia patients in the United States.
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Title/Link to more information |
Study Purpose |
Study Coordinator |
| Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases | This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with CD3+ /CD19+ depleted peripheral stem cell grafts from mismatched donors. There are two conditioning regimens depending upon patient diagnosis and age. |
This study is being conducted at the Children’s Hospital of Philadelphia, PA Contact: Margaret Tartaglione, RN, 215-590-4029 or tartaglione@email.chop.edu Please refer to this study by its ClinicalTrials.gov identifier: NCT02277639 |
Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 310 studies recruiting MDS patients in the United States.
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Title/Link to more information |
Study Purpose |
Study Coordinator |
| The Myelodysplasia Transplantation-Associated Outcomes (MDS-TAO) Study |
The purpose of this observational study is to compare overall survival in older adults with myelodysplastic syndromes (MDS) who receive reduced intensity conditioning hematopoietic stem cell transplant (RIC HSCT) versus those who do not receive HSCT. | This study is being conducted in Boston, MA. Beth Israel Deaconess Medical Center—contact David Avigan, MD 616-667-9920 davigan@bidmc.harvard.edu Dana Farber Cancer Institute—contact Gregory Abel, MD, MPH 717-632-2304 GAABEL@PARTNERS.ORG Massachusetts General Hospital—contact Yi-Ben Chen, MD 617-726-1124 yachen@partners.org Please refer to this study by its ClinicalTrials.gov identifier: NCT02390414 |
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A Phase I/II Study of OPN-305 in Second-line Lower Risk Myelodysplastic Syndrome |
The dose-confirming part of this study, comprising at least 10 patients is designed as a single center, prospective, single arm, open label in patients who have failed or are unresponsive to an Erythropoiesis Stimulating Agent (ESA) and/or Azacitidine (AZA) followed by a dose expansion part with an additional 30 patients; the objective of the whole study being to assess the safety, efficacy, pharmacokinetics and pharmacodynamics of intravenously infused multiple doses of OPN-305 in low and intermediate-1 risk myelodysplastic syndrome (Second line Lower risk MDS). | This study is being conducted in Houston, TX. Contact: Robert M Miller, FRCS, MBBS RMiller@opsona.com Please refer to this study by its ClinicalTrials.gov identifier: NCT02363491 |
| Phase II Decitabine (DAC) Versus Azacitidine (AZA) in Myelodysplastic Syndrome (MDS) | The goal of this clinical research study is to compare how 2 different drugs, decitabine and azacitidine, when given on a shorter than standard dosing schedule, may help to control MDS. The safety of each study drug given on these schedules will also be studied. |
This study is being conducted in FL, MD, MA, NY, OH and TX Contact: Elias Jabbour, MD at MD Anderson Cancer Center, 713-792-4764 Please refer to this study by its ClinicalTrials.gov identifier: NCT02269280 |
| A Dose Escalation and Cohort Expansion Study of TEN-010 in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome |
TEN-010 is a small molecule, bromodomain and extra-terminal domain (BET) bromodomain inhibitor. This study is designed to characterize the safety, tolerability, and pharmacokinetics of TEN-010 in patients with relapsed/refractory acute myeloid leukemia (RR-AML) and hypomethylating agent (HMA)-refractory myelodysplastic syndrome (MDS). In addition, this trial will assess response to treatment using International Working Group (IWG) response criteria. This study will be conducted in two parts: dose escalation and dose expansion. For dose escalation (Part A), a standard "3+3" design will be used in which successive cohorts of three or more patients with RR-AML or HMA-refractory MDS will be treated at escalating doses until a maximum tolerated dose (MTD) is identified. For the dose expansion part of the study (Part B), patients will be treated with TEN-010 at the MTD (or the highest dose tested if the MTD is not defined) to further characterize its safety, pharmacokinetics, and clinical response. |
This study is being conducted in NY. Contact: NY- Tania Curcio 212-746-2571
tjc9003@med.cornell.edu |
Please search for a list of current paroxysmal nocturnal hemoglobinuria studies actively recruiting patients. The following are a sample of the 9 studies recruiting PNH patients in the United States.
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Title/Link to more information |
Study Purpose |
Study Coordinator |
| A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects With PNH | This study will be the initial exploration of APL-2 in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of APL-2 will guide decisions to further develop the drug. | This study is being conducted at the University of Louisville, KY Contact: Candace Depp pnh@apellis.com Please refer to this study by its ClinicalTrials.gov identifier: NCT02264639 |
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This study is being conducted in Houston, TX. Contact: Robert M Miller, FRCS, MBBS RMiller@opsona.com Please refer to this study by its ClinicalTrials.gov identifier: NCT02363491 |