Clinical Trials | Aplastic Anemia and 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 Phase III, International, Randomized, Controlled Study of Rigosertib versus Physician’s Choice of Treatment in Patients with Myelodysplastic Syndrome after Failure of a Hypomethylating Agent Spotlight

Status(es): Recruiting
Study Date(s): Thursday, October 1, 2015 to Tuesday, December 31, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 - 81 years
Patients with higher-risk MDS failing HMA treatment (targeted patient population)  have no approved medical therapy and a short life expectancy (approximately 4-6 months; Bello 2009, Jabbour 2008, Jabbour 2010, Prebet 2011). Once treatment with an HMA has failed prognosis is dismal and thus there is a great unmet medical need to identify  novel therapies for these patients. We propose to enroll patients with MDS who have excess blasts (5% to 30% BM blasts), and have progressed during, failed to respond to, are intolerant of, or relapsed after AZA or DAC treatment. Patients’ MDS must be...

A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects with Advanced Hematologic Malignancies with an IDH1 Mutation Spotlight

Status(es): Recruiting
Study Date(s): Monday, March 17, 2014 to Friday, June 30, 2017
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation.

A Phase 2 Safety and Efficacy Study of RA101495 to Treat PNH Patients Who Have an Inadequate Response to Eculizumab Spotlight

Status(es): Recruiting
Study Date(s): Wednesday, February 1, 2017 to Thursday, February 1, 2018
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks. Note: Patients on the study will receive RA101495 at the dose of 0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC

A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects with PNH Spotlight

Status(es): Recruiting
Study Date(s): Monday, November 24, 2014
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of the study is to assess the safety, tolerability and pharmacokinetics of APL-2 in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who are still anemic after treatment with eculizumab (Soliris®).

AK579 (COBALT) Spotlight

Status(es): Recruiting
Study Date(s): Monday, August 1, 2016 to Friday, June 30, 2017
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
Coversin Global Study:  An Open-Label, Safety and Efficacy Trial in PNH Patients

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms Spotlight

Status(es): Recruiting
Study Date(s): Monday, February 8, 2016 to Sunday, January 1, 2017
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
Coversin in paroxysmal nocturnal haemoglobinuria (PNH) in patients with resistance to eculizumab due to complement C5 polymorphisms

Low Dose Danazol for the Treatment of Telomere Related Diseases Spotlight

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...

MEDALIST Trial Spotlight

Status(es): Recruiting
Study Date(s): Tuesday, December 1, 2015 to Tuesday, June 25, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 and older
The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in subjects with anemia due to International Prognostic Scoring System-Revised (IPSS-R) very low, low, or intermediate Myelodysplastic syndrome (MDS) with ring sideroblasts (≥ 15%) who require Red...

Optimizing Cord Blood and Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) (CHAMP) Spotlight

Status(es): Recruiting
Study Date(s): Monday, May 22, 2017 to Thursday, February 1, 2018
Disease(s): aplastic anemia
Age Group: up to 75 years
This study is a prospective, simultaneous, parallel phase II study with one arm receiving unrelated cord blood transplantation and the other arm receiving haploidentical transplantation for Severe Aplastic Anemia (SAA) patients. The primary objective is to assess overall survival (OS) separately in the 2 arms at 1 year post-hematopoietic stem cell transplantation (HSCT).

PEGASUS: A Phase III Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH Spotlight

Status(es): 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 »