Clinical Trials | Aplastic Anemia & MDS International Foundation
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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.

Clinical Trials

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 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

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 »

Study of PTC299 in Relapsed/Refractory Acute Leukemias Spotlight

Status(es): Recruiting
Study Date(s): Monday, October 29, 2018 to Wednesday, November 25, 2020
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is an open-label, non-randomized, Phase 1b study to evaluate the safety, pharmacokinetics (PK) profiles, and preliminary evidence of antitumor activity of PTC299 and the metabolite, O-desmethyl PTC299, in participants with relapsed/refractory acute myeloid leukemia (AML) who have exhausted standard available therapies known to provide clinical benefit. The study is designed as a series of cohort-based dose escalations. For each cohort, a minimum of 3 evaluable participants with PK and safety data will be assessed. Additional participants will be recruited if additional PK data are...

The National Myelodysplastic Syndromes Natural History Study Spotlight

Status(es): Recruiting
Study Date(s): Friday, April 1, 2016 to Wednesday, September 1, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS).  Participants will be followed long term.  Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.

Trial of the Combination of Bortezomib and Clofarabine in Adults With Relapsed Solid Tumors Spotlight

Status(es): Recruiting
Study Date(s): Wednesday, August 6, 2014 to Friday, August 31, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is an open-label Phase I trial. The starting dose of clofarabine will be 1 mg/m2 administered intravenously on days 1 through 5 of a 21-day cycle; bortezomib will be administered at 0.8 mg/m2 subcutaneously on days 1 and 4 of a 21-day cycle. Dose escalation will follow a 3+3 design, with dose limiting toxicities defined during cycle 1. Dose escalation will proceed in cohorts comprised of two separate groups of patients (one group of patients with solid tumor/lymphoma and one group of patients with MDS), with at least 1 from each group, until hematologic DLT or the second grade 2...

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.