Clinical Trials | Aplastic Anemia & MDS International Foundation
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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.

(COMMANDS) - A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alpha for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Due to Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Wednesday, January 2, 2019 to Friday, September 8, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is an interventional active-controlled, open-label, randomized Phase 3 study to compare the efficacy and safety of luspatercept (ACE-536) versus epoetin alfa for the treatment of anemia due to IPSS-R very low, low or intermediate risk MDS in ESA naïve subjects who require RBC transfusions. The study is divided into the Screening Period, a Treatment Period and a Post-Treatment Follow-up Period.

A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, Umbilical Cord Blood-derived, Stem and Progenitor Cells, versus Unmanipulated Umbilical Cord Blood for Patients with Hematological Malignancies

Status(es): Recruiting
Study Date(s): Tuesday, November 1, 2016 to Tuesday, December 1, 2020
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 12 years to 65 years
This study is an open-label, controlled, multicenter, international, Phase III, randomized study comparing transplantation of Omidubicel/ NiCord® to transplantation of one or two unmanipulated, unrelated cord blood units in patients with hematological malignancies for whom allogenic SCT is currently a recommended and potentially lifesaving treatment (all with required disease features rendering them eligible for allogeneic transplantation).

A phase I/II Study of REGN7257 (Anti-interleukin 2 receptor subunit gamma [IL2RG] monoclonal antibody) in patients with severe aplastic anemia that is refractory to or relapsed on immunosuppressive therapy

Status(es): Not yet recruiting
Study Date(s): Friday, August 21, 2020 to Thursday, July 20, 2023
Disease(s): aplastic anemia
Age Group: 18 years and older
The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with severe aplastic anemia (SAA) that is refractory to or has relapsed while on standard of care immunosuppressive therapy (IST). An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in IST-refractory/relapsed patients. The secondary objectives of this study are to assess the following for REGN7257: Clinical response over time Maintenance of response Impact on transfusion requirements Effect on blood counts and cell populations Pharmacokinetics...

A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment

Status(es): Recruiting
Study Date(s): Thursday, August 8, 2019 to Monday, May 15, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
IMerge is a two-part Phase 2/3 clinical trial of imetelstat in transfusion dependent patients with lower risk MDS who are relapsed after or refractory to erythroid stimulating agents (ESAs). The Phase 3 portion is planned to enroll approximately 170 patients in a randomized (2:1 ratio of imetelstat:placebo), double-blind, placebo-controlled clinical trial to test the hypothesis that imetelstat improves the rate of red blood cell transfusion independence (TI). The trial is planned to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia. The...

APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)

Status(es): Active, not recruiting
Study Date(s): Friday, January 11, 2019 to Sunday, November 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
A Phase III, multicenter, randomized study to compare the rate of complete response (CR) and duration of CR, in patients with TP53-mutated MDS who will receive APR-246 and azacitidine or azacitidine alone. The p53, or P53, protein is coded by the TP53 gene which is mutated in a portion of patients with MDS. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndromes (MDS) with a susceptible TP53 mutation. For more information, please see: https://ir.aprea.com/news-releases/...

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 Monday, February 28, 2022
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): Active, not recruiting
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): 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...

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any clinical trial listed on this website. Pharmaceutical company sponsored content is highlighted only to give additional information about the trial. All trials are listed on https://clinicaltrials.gov/. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a clinical trial, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Further Reading

Patients should learn as much as possible about a clinical trial before making the decision to participate.