Clinical Trials | Page 3 | Aplastic Anemia & MDS International Foundation

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.

A Dose-finding Study of CC-90009 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-risk Myelodysplastic Syndromes

Status(es): Recruiting
Study Date(s): Thursday, July 28, 2016 to Saturday, February 26, 2022
Disease(s):
Age Group: 18 and older
Study CC-90009-AML-001 is an open-label, Phase 1, dose escalation and expansion, first-in-human clinical study of CC-90009 in subjects with relapsed or refractory acute myeloid leukemia (AML) and relapsed or refractory high-risk myelodysplastic syndrome. The dose escalation part (Part A) of the study will evaluate the safety and tolerability of escalating doses of CC-90009 in relapsed and refractory AML. The expansion part, (Part B), will further evaluate the safety and efficacy of CC-90009 administered at or below the maximum tolerated dose (MTD) in selected expansion cohorts of one or more...

A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status(es): Active, not recruiting
Study Date(s): Thursday, June 22, 2017 to Wednesday, December 30, 2020
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of this study is to evaluate long term safety and efficacy of ACH-0144471 in patients with PNH who have demonstrated clinical benefit from ACH-0144471 in Clinical Study ACH471-100. This study is designed to include up to 12 patients.

A Phase 1 Study of AMG 330 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Monday, August 31, 2015 to Sunday, May 16, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
  The purpose of this First-in-Human Phase 1 study is to determine if AMG 330 given as a continuous IV infusion is safe and tolerable in adult subjects that have relapsed/refractory Acute Myeloid Leukemia, and to determine the maximum tolerated dose and/or a biologically active dose. The study will be conducted in multiple sites and test increasing doses of AMG 330. The safety of subjects will be monitored by intensive assessment of vital signs, electrocardiograms, physical examinations, and laboratory tests.

A Phase 1 Trial of CD25/Treg-depleted DLI Plus Ipilimumab for Myeloid Disease Relapse After Matched-HCT

Status(es): Recruiting
Study Date(s): Thursday, April 11, 2019 to Tuesday, April 26, 2022
Disease(s): acute myeloid leukemia (AML), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN)
Age Group: 18 years or older
In this research study, our main goal for the ipilimumab portion of the study is to determine the highest dose of ipilimumab that can be given safely in several courses and to determine what side effects are seen in patients with Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS), Myeloproliferative Neoplasms (MPN), Chronic Myelomonocytic Leukemia (CMML), or Myelofibrosis (MF).

A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status(es): Recruiting
Study Date(s): Thursday, February 22, 2018 to Monday, August 1, 2022
Disease(s):
Age Group: birth to 18 years
The purpose of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).

A Phase I Study of CYC140, a PLK-1 Inhibitor, in Advanced Leukemias or MDS

Status(es): Recruiting
Study Date(s): Thursday, March 21, 2019 to Monday, October 26, 2020
Disease(s): acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), myelodysplastic syndromes (MDS)
Age Group: 18 or older years
A Phase I Pharmacologic Study of CYC140, a polo-like kinase 1 inhibitor, in Patients with Advanced Leukemias or Myelodysplastic Syndromes.  This is a First in Human, open-label, single arm, dose escalation study in patients with advanced leukemias or myelodysplastic syndromes.   

A Phase I-II Study of a Liposomal Formulation of Cytarabine and Daunorubicin (CPX-351) in Patients Treated for Higher-risk Myelodysplastic Syndromes Experiencing Hypomethylating Agent Failure.

Status(es): Not yet recruiting
Study Date(s): Tuesday, October 1, 2019 to Tuesday, October 31, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 78 years
Proposed is a two-phase study. The Phase I portion will confirm the tolerability and safety of CPX-351 chemotherapy. Patients who meet eligibility criteria will receive dose level 1 of CPX-351 (44mg/m2 of daunorubicin and 100mg/m2 of cytarabine) on 2 days (day 1 and day 5) of the cycle. If less than 2 DLTs are observed in the first cohort of 6, we will increase level of exposure to Dose Level 2 by giving (44mg/m2 of daunorubicin and 100mg/m2 of cytarabine) on days 1, 3 and 5 of If less than 2 DLTs are observed Dose Level 2 will become the RP2D If 2 or more DLTs are observed Dose Level 1...

A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.

Status(es): Not yet recruiting
Study Date(s): Saturday, February 1, 2020 to Sunday, March 20, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Acute myeloid leukemia is a malignant disorder characterized by the rapid, uncontrolled proliferation of malignant clonal hematopoietic stem cells that accumulate as immature, undifferentiated cells (blasts) in the bone marrow and circulation. APG-115 is a potent and orally active small-molecule MDM2 inhibitor, it binds to MDM2 protein and shows potent cell growth inhibitory activity in vitro with low nanomolar potencies in a subset of human cancer cell lines. APG-115 has demonstrated its strong antitumor activities with either daily or less frequent dosing-schedules in the acute leukemia...

A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

Status(es): Recruiting
Study Date(s): Thursday, May 11, 2017 to Tuesday, April 1, 2025
Disease(s): aplastic anemia
Age Group: 1-18
This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia. All patients will be treated with eltrombopag for the 26-week Treatment Period, followed by a 52-week Follow-Up Period. Patients who have been previously untreated with immunosuppressive therapy will be treated according to the standard of care, hATG/cyclosporine, in...

A Phase II Trial of CD24Fc for Prevention of Acute Graft-versus-Host Disease Following Myeloablative Allogeneic Hematopoietic Stem Cell Transplant

Status(es): Recruiting
Study Date(s): Monday, February 1, 2016 to Monday, June 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a multicenter prospective randomized phase IIa/IIb clinical trial designed to determine the MTD (Maximum Tolerated Dose) of CD24Fc for acute GVHD (Graft Versus Host Disease) prophylaxis.