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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.

A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant

Status(es): Recruiting
Study Date(s): Tuesday, May 4, 2021 to Monday, April 1, 2024
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), pediatric
Age Group: 4 years and older
The purpose of this study is to see if conditioning regimens that include personalized rabbit ATG (P-rATG) help the immune system recover sooner and decrease the chances of transplant-related side effects. Participants in this study will be children and adults who have acute leukemia or myelodysplastic syndrome (MDS), and they will receive a standard conditioning regimen to prepare the body for an allogeneic hematopoietic cell transplant (allo-HCT). The conditioning regimen will include r-ATG, one of two combinations of chemotherapy, and possibly total body irradiation (TBI).

A Novel TBI Free Conditioning Protocol for Haploidentical Transplant in Acquired Aplastic Anemia: (FluCAB-Prime) (Pakistan only)

Status(es): Recruiting
Study Date(s): Thursday, July 12, 2018 to Wednesday, June 30, 2021
Disease(s): aplastic anemia
Age Group: 2 years to 60 years
Severe and very severe aplastic anemia are life threatening disorders for which allogeneic stem cell transplant is only curative treatment. However, matched sibling donor (MSD) is available in only 25-35% cases. Pakistan has a population of around 203 million but there is no donor registry available so there is no option available for matched unrelated donor (MUD) transplants . Haploidentical transplant represents only curative option for patients lacking MSD. Protocols involving post transplant cyclophosphamide require Total body irradiation (TBI) and utilize peripheral blood stem cell(PBSC...

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-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 III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity Between SB12 (Proposed Eculizumab Biosimilar) and Soliris® in Subjects With Paroxysmal Nocturnal Haemoglobinuria

Status(es): Recruiting
Study Date(s): Wednesday, August 7, 2019 to Thursday, July 1, 2021
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
This is a randomised Phase III, double-blind, multicentre, cross-over study to compare the efficacy, safety, pharmacokinetics, and immunogenicity between SB12 and Soliris® in subjects with PNH.

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. (COMMODORE 2)

Status(es): Recruiting
Study Date(s): Thursday, October 8, 2020 to Friday, October 25, 2024
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH), pediatric
Age Group: 12 years and older
This study will enroll participants aged 12 years or older with a body weight >= 40 kg diagnosed with PNH who have not been previously treated with complement inhibitor therapy. Approximately 200 participants will be randomized in a 2:1 ratio into the following regimens: [1] Crovalimab; [2] Eculizumab.
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