Sickle Cell Disease Research and Clinical Trials in Ontario

This section is intended solely to inform on the various research studies and patient recruitment currently underway. The inclusion is not an endorsement of a particular product, company, or study.

We have provided clinical trials currently recruiting in Ontario with links to more information at is a registry and results database of federally and privately supported clinical trials conducted in the United States and around the world. gives you information about a trial’s purpose, who may participate, locations, and phone numbers for more details. This information should be used in conjunction with advice from health care professionals.

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 have learned to manage their SCD well, transitioned to adult care and can support youth participants emotionally and socially. Participants will be randomly assigned 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, 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