Sickle Cell Disease Research and Clinical Trials in Ontario

iPeer2Peer Program for Youth with Sickle Cell Disease
The iPeer2Peer Sickle Cell Disease (SCD) study matches youth (12-18 years of age) with SCD to a mentor (trained young adult) who has learned to manage their SCD well, transition to adult care, and can support youth participants emotionally and socially. Participants will be randomly assigned to one of two groups, either (1) The intervention group: Study group participants are matched with a mentor for 15 weeks, and are expected to have up to ten calls with one another; (2) The control group: This study group will be on a 15-week waitlist to receive a mentor. This study will first assess the feasibility of conducting this research with youth with SCD. Also, this study will assess the preliminary effectiveness of peer mentorship by comparing various health outcomes of the two study groups post-intervention. For more on this study, click here

Measures of Respiratory Health (MRH)
The Lung Clearance Index, measured by multiple breath washout, is a measure of lung function that is considered a research tool in Canada as the device used to measure it is not approved by Health Canada. The study will assess lung function in patients undergoing routine lung function testing for clinical indications (Cystic Fibrosis and Other Respiratory Diseases). In addition, healthy controls of different ages will be asked to perform this lung function test to gain reference data that can be used to interpret LCI in patients with lung disease. For more on this study, click here

Dose-Ranging Study of Benserazide In Thalassemia Intermedia (Pb04-001) (Sickle Cell Disease in Phase 11)
Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body. The diseases are characterized by hemolytic anemia, organ damage, and early mortality. Increases in another type of (normal) hemoglobin, fetal globin (HbF), which is normally silenced in infancy, reduce the severity of both conditions. Even incremental augmentation of fetal globin is established to reduce red blood cell pathology, anemia, and certain complications, and to improve survival.

This trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice. The study drug acts by suppressing 4 repressors of the fetal globin gene promoter in progenitor cells from thalassemia patients. The drug has been used for many years in a combination product for different actions – to enhance half-life and reduce side effects of an active drug- and is considered safe for chronic use.

This trial will first evaluate 3 dose levels in small cohorts of non-transfused patients with beta-thalassemia intermedia. The most active dose will then be evaluated in larger subject groups with beta-thalassemia and other hemoglobinopathies.

For more on this study, click here

ICANCOPE with Sickle Cell Pain
The project will test a tailored web and smartphone-based application (iCanCope with SCD) to improve pain self-management and functioning in youth (aged 12-18) with sickle cell disease. The program will include goal setting, peer-based social support, and pain self-management training. The investigators will determine initial program effectiveness through a pilot three-site randomized controlled trial in 160 youth randomized to treatment compared to attention control. For more on this study, click here

Long-Term Safety and Efficacy of Ferriprox® In Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.
For more on this study, click here

Gene Transfer for Patients with Sickle Cell Disease
The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and the success of gene correction in subjects with sickle cell disease
For more on this study, click here

A Safety and Efficacy Study Evaluating CTX001 in Subjects with Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
For more on this study, click here

Efficacy and Safety of Ferriprox® in Patients with Sickle Cell Disease or Other Anemias (FIRST)
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body. We will be comparing deferiprone with deferoxamine, another drug that removes iron from the body.
For more on this study, click here

RUBY Short Trial Description for Patient Advocacy Organizations
A Study Evaluating the Safety and Efficacy of EDIT-301 in People with Severe Sickle Cell Disease (RUBY)
The RUBY study is evaluating the safety and efficacy of a single dose of EDIT-301, a new investigational medicine for adults 18 to 50 years old with severe sickle cell disease (SCD). The goal is to provide long-lasting relief from the symptoms of SCD. EDIT-301 is an investigational gene editing medicine that uses CRISPR-Cas12a gene editing technology to edit your stem cells outside your body. When your edited cells are returned to you, your body can potentially create its own healthy, non-sickled blood cells containing increased levels of fetal hemoglobin. You may be eligible for the study if you have the following genotypes: HbSS, HbS-beta-zero thalassemia, or HbS-beta-plus thalassemia. Participation in this study is at no cost to you, including travel, meals, and any required accommodation for you and a companion. To learn more about the clinical study, please visit www.RubySCDstudy.com.

If you have any questions, please call or email us at 617-401-9007, patients@editasmed.com.

The University Health Network-Red Blood Cell Disorders clinic in Toronto, Canada is accepting referrals for participation in sickle cell disease, thalassemia, and membranopathy clinical trials as listed on the downloadable document on this page. Please review the document for major inclusion criteria and brief study schema.
To enroll, kindly ask your treating physician to refer you or speak to the RBCD clinic promptly.