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

CLINICAL TRIALS of medications…phases…the pros and cons…the rights of “subjects”…are trials necessary?
You will find below links to web sites which provide answers to frequently asked questions about clinical research.
CanadaTrials.com 's Clinical Research FAQ page

ClinicalTrials.gov is a registry and results database of federally and privately supported clinical trials conducted in the United States and around the world. ClinicalTrials.gov 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.

Patient and Families Engagement/Oriented Research- Current opportunities
1. A Survey of Dizziness/Imbalance and Hearing Loss in Adults Living with Sickle Cell Disease:Evaluating the correlation between Sickle Cell Disease and
dizziness/balance and hearing problems. To participate, go to: https://www.surveymonkey.com/r/GZH8FFP
Background: Dr. M. Dawn Nelson, Ph.D., a professor at Central Michigan University is currently researching the association between dizziness/balance and hearing problems and Sickle Cell Disease. Dr. Nelson recently attended a Sickle Cell conference at the FDA (February 2014) where many individuals expressed interest in research in this area. Since then, she has been contacted by the FDA who reported many individuals from the conference had been contacting them interested in being involved in a research study.

Invitation to patients, family members,caregivers and health care providers to participate in chronic pain research.
Chronic Pain Strategy for Patient Oriented Research Network
Given that sickle cell disease comes with chronic pain we are pleased to share with you the research project from the Chronic Pain Network and Drs. Patricia Poulin and Jennifer Stinson who hold a CIHR SPOR Patient Engagement Collaboration Grant (“Improving Chronic Pain Care for all Canadians: A Patient Engagement Project to Identify Research Priorities”).

To ensure the sickle cell community's issues with pain is clearly identified;
we ask that you complete the survey in the provided link.http://fluidsurveys.com/s/Chronicpainmatters/ 

RESEARCH ADVISORY COMMITTEE (RAC):

The Research Advisory Committee will establish the research priorities of the SCDAC, its strategic research partnership and run the research program in the most effective way possible. SCDAC’s research agenda will not be limited to fellowships, basic research, clinical research, research by young investigators but also include social and psychosocial research initiatives. Hence RAC should include psychologists, social workers, and nurses in addition to academic clinicians. The research agenda designed by RAC must be best suited to the persons and processes served by the organization

TERMS OF REFERENCE- RESEARCH ADVISORY COMMITTEE
PURPOSE: The purpose of this committee is to advise the SCDAC Board of Directors on its research programs so as to maximize its impact.

OBJECTIVES:

  • Help the SCDAC establish research priorities and policies.
    • Help the SCDAC run its research programs in the most effective way possible.
    • Help the SCDAC establish strategic research partnerships
  • DUTIES AND RESPONSIBILITIES:

    The Duties and Responsibilities of the Committee shall be to:

  • Advise the SCDAC on research priorities within the field of hemoglobinopathy that will allow the SCDAC research programs to have maximal impact.
    • Advise the SCDAC on the types of research programs (e.g. fellowships, basic research, clinical research, research by young investigators…) that are best suited to the persons and processes served by the organization.
    • Establish and regularly review the terms of reference and operating parameters for the Peer Review Committee
    • Advise the SCDAC about the membership of the Peer Review Committee (which is not exclusive of the Research Advisory Committee)
    • Advise the SCDAC on ways to improve the application and granting process
    • Advise the SCDAC on ways to better publicize the existence of its research programs and the results of the research conducted.
    • Guide the SCDAC in exploring and establishing strategic partnerships that will be beneficial to both parties.
    • Provide any other advice that will benefit the SCDAC research programs, including ways to resolve conflicts that may arise from the Peer Review Committee, its operation or recommendations.
    • Be proactive in raising issues of importance, and conflict of interest concerning research.
    • Report annually to the national Board of Directors on the status state of SCDAC research programs.
  • MEMBERSHIP AND TERMS OF OFFICE:
    The Committee shall consist of not fewer than five and not more than twelve members and shall be composed as follows:
    a) Chairperson (or co-Chairpersons): The Chairperson shall be appointed annually by the SCDAC Board, and shall be eligible to hold the position of Chairperson for a maximum of 6 years. Chairpersons shall ideally be individuals with a strong research background in the field of hematology.
    b) Membership: The members of the committee will be appointed by the Chairperson and will consist of members who have relevant research interests, skills or experience. In choosing members, consideration will also be given to varied representation from different disciplines (medical, nursing, physiotherapy, psycho-social).
    An individual representing the patient population perspective will also be named as a member of the committee.
    The term of appointment for membership shall be one year and members shall be eligible for re-appointment.
    c) Ex-officio: President and Executive Director

    FREQUENCY AND NOTICE OF MEETINGS:
    The Committee shall meet in person or by teleconference not less than once yearly and shall meet at such other times as the Chairperson of the Committee may require.
    Unless otherwise agreed, notice of each meeting confirming the venue or mode of meeting, time and date, together with an agenda of items to be discussed, shall be forwarded to each member of the committee no fewer than five days prior to the date of the meeting.

    AUTHORITY:
    The Research Advisory Committee is a standing committee established by the Board of Directors of the Sickle Cell Disease Association of Canada. It reports through its Chairperson to the Executive Director and the Board of Directors. 

    RESEARCH PEER REVIEW COMMITTEE- TERMS OF REFERENCE

    PURPOSE:
    The Research Peer Review Committee is established to review submissions made in response to calls for competitive research proposals offered by the Sickle Cell Disease Association of Canada (SCDAC), and to advise the SCDAC with respect to continuous quality improvement in the review process. The principles inherent in the review process are identification of excellence, provision of equity of opportunity, and due diligence in the allocation of SCDAC research funds.

    OBJECTIVES:
    a. Evaluate applications submitted to the research grant competitions and for traineeships, and make recommendations for funding to the SCDAC;

    b. Monitor the application processes and protocols governing evaluation of applications to SCDAC grant competitions;

    c. Make recommendations concerning pertinent aspects of the research grant competition process to the Research Advisory Committee.

    MEMBERSHIP AND TERMS OF OFFICE

    The Committee shall consist of a Chair and not fewer than five members and shall be composed as follows:

    a. Chair (or co-Chairs): The Chair shall be appointed by the SCDAC Board of Directors based on a recommendation from the Research Advisory Committee and shall be eligible to hold the position of Chair for a term of three years with possible renewal for a second term. If the Chair resigns during the term, a successor will be appointed for the time remaining in that term. Chairs shall ideally be individuals with a strong research background in the field of hematology.

    b. Membership: The members of the committee will be appointed by the Research Advisory Committee in consultation with the Chair of the Peer Review Committee and will consist of individuals with relevant research interests, skills or experience. In choosing members, consideration will also be given to varied representation from different disciplines (medical, nursing, physiotherapy, psychosocial). One member representing the patient perspective will also be named. The term of appointment for members shall be two years and members shall be eligible for re-appointments. To ensure continuity, efforts must be made to ensure that the majority of the Peer Review Committee members do not rotate at one time.

    c. Ex-officio: The SCDAC Management lead/Executive Director will be a non-voting member of the Peer Review Committee to provide support, documentation and linkage with the SCDAC Management and Board of Directors.

    DUTIES AND RESPONSIBILITIES: 

    Members will:
    a. undertake timely, transparent, fair, objective and thorough review of eligible applications;
    b. submit a written summary of their evaluations, including scores and critique of each application to the Chairperson of the Peer Review Committee;
    c. complete a conflict of interest statement for each grant application;
    d. participate in meetings/teleconferences to reach consensus on the award selection;
    e. submit feedback on the review process.
    The Chairperson will:
    a. chair the meetings and delegate responsibilities;
    b. review the agenda and minutes for each meeting;
    c. provide written feedback and final ranking to each applicant, whether successful or not, based on written summaries provided by the Peer Review Committee members; summarized comments sent back to applicant would not indicate from whom a point of criticism came;
    d. report to the Research Advisory Committee on completion of each annual SCDAC research grant competition cycle providing an appreciation of the review process itself, number and types of applications reviewed, profile of applicants, number of applications recommended, general strength and weaknesses of the applicants and the proposed projects and their alignment with SCDAC research priorities; e. make funding recommendations to the Board of Directors of the SCDAC based on the review process so that funding offers can be made to the best ranked candidates.
    The Committee will:
    a. solicit opinions from external reviewers, as required;
    b. provide a consensus evaluation of each proposal;
    c. make funding recommendations by ranking best candidates for each research program;
    d. review as needed the terms of reference and operating parameters of the Peer Review Committee;
    e. recommend membership in the Peer Review Committee;
    f. advise on the application and granting process;

    CONFIDENTIALITY
    The Peer Review Committee and its external reviewers maintain responsibility to the SCDAC, the sponsors, investigators, and applicants that its deliberations and the information to which it becomes privy be treated with the utmost confidentiality by all its members
    All materials viewed by members of the committee must be kept in the strictest confidence. These items cannot be circulated outside of the Peer Review Committee unless otherwise decided by the committee. Failure to honor confidentiality will be grounds for dismissal from the Peer Review Committee. Each Peer Review Committee member and external reviewer will sign a Confidentiality Agreement with the SCDAC.

    TIMING, NOTICE OF MEETINGS & QUORUM:
    The Committee shall meet in person or by teleconference and members will participate in at least one annual meeting/teleconference to review applications and make recommendations on awards. The Committee shall meet in person or by teleconference at such other times as the Chair may require. Unless otherwise agreed, notice of each meeting confirming the venue or mode of meeting, time and date, together with an agenda of items to be discussed, shall be forwarded to each member of the committee no fewer than five days prior to the date of the meeting. At least half of the members of Peer Review Committee must be present at a meeting to constitute a quorum.

    AUTHORITY: The Peer Review Committee is a standing committee of the SCDAC. It reports through its Chairperson to the Executive Director and the Board of Directors; the Chairperson also keeps the Research Advisory Committee informed of the activities of the Peer Review Committee. The Research Advisory Committee is a separate entity, which also reports to the Executive Director and the National Board of Directors.

    The Medical Advisory Board of the SCDAC will be a three member team led by a hematologist. Other members of the MAB will include a hematology nurse and an allied health professional. The MAB will provide and advise SCDAC on medical matters.

    MEDICAL ADVISORY BOARD- TERMS OF REFERENCE
    Purpose:
    The purpose of the MAB is to advise SCDAC on medical and scientific matters pertaining to the management and health-care of those affected with of people with sickle cell disease.
    Duties and Responsibilities:
    • To co-sign all documents containing medical facts including letters to the Ministry of Health and Long Term Care in Ontario and Health Ministers in all other Provinces
    • To provide information to the SCDAC concerning the comprehensive management of people with sickle cell disease
    • To assist the SCDAC in ensuring equal access to care and treatment for all persons with sickle cell disease
    • To inform the SCDAC and RAC of research initiatives considered to be of benefit and interest to all persons with sickle cell disease.
    • To represent the SCDAC, from time to time, on ad hoc committees developing policies on medical affairs issues
    Nomination and Term of Office:
    The MAB shall be chaired by a hematologist appointed by the SCDAC Board of Directors
    The term of appointment shall be one year, and shall be renewable for up to four consecutive years

    This board designs the research agenda for SCDAC/AAFC to include among other responsibilities attracting more researchers and fellows to the field of hemoglobinopathy and encourage data collection to support optimal evidence-based care and treatment

    The clinical trials provided herein are Toronto General Hospital (TGH) focused. This page will be updated with more clinical trials as are provided by various institutions across the nation.

    List of current clinical trials :

    1. Human Mesenchymal Stromal Cells (MSCs) for the treatment of early stage osteonecrosis of the hip; a phase I dose-escalation study; PI Dr. Armand Keating, UHN
      This pilot study is assessing whether it is feasible to use stem cells to treat avascular necrosis in patients with sickle cell disease
    2. Conversion of human fibroblasts from patientswith hemoglobinopathies to erythropoietic progenitors; PI Dr Armand Keating, UHN
      This pilot study is studying whether it is possible to turn skin cells into normal red blood cells as a potential future cure for sickle cell disease
    3. Sickle Hemoglobin Recovery, Erythropoiesis and Hemolysis Kinetics in Sickle Cell Disease Patients Undergoing Red Blood Cell Exchange; PI Dr David Barth, UHN
      Study looking into factors that can affect how fast red blood cells are being made and destroyed for sickle cell patients receiving regular exchange transfusion
    4. HQP 1001-SCD-007: A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease; PI Dr Richard Ward, UHN
      This new oral drug is being tested as a treatment for sickle cell disease by increasing the amount of fetal (baby) hemoglobin produced by the body
    5. Pulmonary Hypertension Prevalency in Sickle Cell Disease, PI Dr Richard Ward; UHN
      This is a review looking to find a better way to diagnose pulmonary hypertension in sickle cell disease
    6. Improving General Internal Medicine Trainees’ Knowledge of Sickle Cell Disease with the Use of an Online Educational Module; PI Dr Richard Ward, UHN
      This study is aimed at improving healthcare professionals’ knowledge of sickle cell disease
    7. Prevalence and Morbidity of HbSC at UHN; PI Dr Richard Ward
      This is a review of patients with HbSC disease, a type of sickle cell that is less well understood and appreciated
    8. Audit of the Use and Adherence to Sickle Cell Acute Pain Pre-Order Sets in Emergency Department at UHN; PI Dr Richard Ward, UHN
      This study evaluates the effectiveness of ED guidance for managing acute sickle cell problems
    9. Vitamin D and Zinc Deficiency Associated with Increased Pain Episodes in Children with Sickle Cell Disease; PI Dr Rob Klaassen, Children’s Hospital of Eastern Ontario
    10. Quality of life in Sickle Cell Disease (SCD) patients and their families, and treatment with hydroxyurea: A pilot study; PI Dr Rob Klaassen, Children’s Hospital of Eastern Ontario
    11. Validation of a Pictogram-Based Sickle Cell Action Plan and Prescription for Children with Sickle Cell Disease; PI Dr Rob Klaassen, Children’s Hospital of Eastern Ontario                  

    Promoting and funding research to improve treatment and ultimately to find a cure

    To fulfill one of its research objectives which is to attract more researchers and fellows to the field of hemoglobinopathy; SCDAC/AAFC is pleased to collaborate with the University Health Network on its Canadian Adult Comprehensive Hemoglobinopathy (CAtCH ) Fellowship program. It will continue to seek more opportunities as this across the country.

    The CAtCH
    There has been an improvement in survival of patients with Sickle Cell Disease over the past several decades, whereas in 1970s the life expectancy was teenage years, it has slowly improved to the 6th decade of life. This improvement in lifespan has impacted on the number of patients requiring expert ongoing medical care. To date, clinical training and research in Sickle Cell Disease has focused on the pediatric population. However, with improved life expectancy and survival into adulthood, there are new challenges in managing the complications unique to adults.

    The Canadian Adult Comprehensive Hemoglobinopathy (CAtCH ) Fellowship is a combined clinical/research fellowship created in response to the needs of trainees who have shown interest in Sickle Cell Disease, and recognizes the lack of any substantive training opportunities. The Fellowship in Adult Hemoglobinopathies focusses on comprehensive care, multidisciplinary (inter-professional) collaboration, education outreach, clinical and translational research development.

    The Fellowship aims to meet the need for clinicians and clinical / translational researchers in this field. It shall help fulfill current and future anticipated workforce needs across the country. This is an innovative, inter-disciplinary, and first of its kind program in Canada, focussing on comprehensive care and research in adult Sickle Cell Disease, and it has received a prestigious training award from the American Society of Hematology.

    The first year encompasses the fundamentals of Sickle Cell as a chronic disorder, from prenatal through adolescence and into adulthood. Time is spent in the regional reference laboratory learning about newborn screening and diagnostic techniques, and in pediatrics gaining an appreciation for the effect of SCD on children’s physical, social and psychological development. The unique transfusion challenges faced in the Sickle Cell population such as availability of suitable donors and the role of exchange transfusion will be examined at the TGH hospital blood bank and also the Canadian Blood Service. The majority of clinical time will be at Toronto General, exposed to a large volume of patients with a diverse array of Sickle Cell related medical issues. The fellow is strongly encouraged to take a relevant MSc program, e.g clinical research, hemoglobinopathies, or public health.

    In the second year, the fellow will further develop their academic interest in Sickle Cell Disease, gain additional clinical experience, and spend 2 months in the UK, to gain an appreciation of different models of care provision and building partnerships. If the Fellow is from outside of Toronto, it is expected they will return to their home institution and implement further Hemoglobinopathy training locally.

    How can I be tested?

    A simple blood test called the hemoglobin electrophoresis can be done at your local laboratory. This test will tell if you are a carrier of the sickle cell trait or if you have the disease.

    What is Sickle Cell Disease?

    Sickle Cell Disease is a disease that affects a protein called hemoglobin in the red blood cells. Hemoglobin carries oxygen around the body. When a person has Sickle Cell Disease, their hemoglobin does not work properly. This can cause pain, a higher chance of serious infections and organ damage or stroke. A person with Sickle Cell Disease needs lifelong treatment.

    What does it mean to be a sickle cell carrier, or to have the sickle cell trait?

    A carrier of Sickle Cell Disease is also said to have the sickle cell trait. This person has inherited a sickle cell gene from a parent, but does not have sickle cell disease and is not more likely to get sick than any other person. They do not need special medical care and will not develop sickle cell disease at any time in their life.
    Why is it helpful to know that I am a carrier of Sickle Cell Disease, or whether my child is a carrier?
    Knowing whether you or your child has the sickle cell trait is important for several reasons. When you are ready to have a family, your partner can have carrier testing so that you know, as a couple, if there is a chance of having a child with Sickle Cell Disease.
    By having this information, you can let your child know if he or she is a carrier of Sickle Cell Disease in the future.

    What does my child’s carrier result mean?
    When a child is a carrier of Sickle Cell Disease, it is very likely that one of the parents is also a carrier of Sickle Cell Disease. More rarely, when a child is a carrier of Sickle Cell Disease:

    • Both parents are carriers
    • One parent actually has Sickle Cell Disease
    • One parent is a carrier and the other has Sickle Cell Disease

    If I have more children, could they have Sickle Cell Disease?
    Most often when a child is a carrier of Sickle Cell Disease, only one parent is a carrier and the chance to have a baby with Sickle Cell Disease is very low

    When both parents are carriers, each pregnancy they have has:

    • 1 in 4 (25%) chance of having Sickle Cell Disease
    • 1 in 2 (50%) chance of being a carrier (but not having Sickle Cell Disease)
    • 1 in 4 (25%) chance of not having Sickle Cell Disease or being a carrier

    rt

    You can have a blood test to find out if you are a carrier of Sickle Cell Disease. If you want to have this test, talk to your health care provider.
    A carrier does not have, and will not develop, Sickle Cell Disease.

    Sickle cell disease is an inherited condition. People who are at high risk of having a child with sickle cell anemia and are planning to have children may want to consider genetic counseling. A counselor can explain the risk (likelihood) of having a child who has the disease. He or she also can help explain the choices that are available. If a person is born with sickle cell disease, steps should be taken to reduce complications. Genetic counseling is most available at major health centres, hospitals, and clinics that care for people who have sickle cell anemia.

    How can I meet with a genetic counsellor?

    If you and your partner are carriers of Sickle Cell Disease, a genetic counsellor can help you learn more about the genetics of Sickle Cell Disease and your options of testing in a future pregnancy.

    There is no treatment for Sickle Cell Disease in pregnancy; however testing is available to couples who want to learn if their baby has Sickle Cell Disease as early as possible. You can ask your health care provider to refer you to your local Genetics clinic. If you do not have a health care provider, contact your local Genetics Clinic directly for more information.

    Should I test my other children to see if they are carriers of Sickle Cell Disease?

    If only one parent is a carrier of Sickle Cell Disease, there is a 1 in 2 (50%) chance your other children are carriers. Carrier testing for Sickle Cell Disease is not usually done in childhood because being a carrier does not cause health problems in childhood. Carrier testing can be done when your other children are old enough to understand the information and decide on their own if they want to be tested. Some parents have their other children tested at a young age and share this information with their child at a time they feel is right both for their child and their family. If you have more questions about testing your other children, talk to your health care provider.

    If both parents are carriers, your other children should be tested for Sickle Cell Disease.

    Should my relatives be tested?

    Other family members (for example, cousins, aunts, uncles) might be carriers of Sickle Cell Disease. It is important to let family members know about this chance. They can then decide if they want to have carrier testing for Sickle Cell Disease. This is especially important if they are planning to have children. Rarely, family members might have Sickle Cell Disease.

    NEW BORN SCREENING
    The provinces of Ontario, British Columbia, Nova Scotia, New Brunswick, Prince Edward Island, Yukon and progressively Quebec have included sickle cell disease in their newborn screening programs. Only four provinces and two territories remain until the goal of universal newborn screening for SCD throughout Canada is achieved. It will continue to push for other provinces and provinces to screen all newborns for sickle cell disorder.

    Screening involves a simple blood test performed at birth that detects the presence of sickle cell disease or sickle cell trait.

    Other types of traits that may be discovered include:
    Hemoglobin C trait
    Hemoglobin E trait
    Hemoglobin Barts – which indicates an alpha thalassemia trait
    Beta thalassemia trait

    What is Sickle Cell Trait?
    Sickle cell trait is a person who carries one sickle hemoglobin producing gene inherited from their parents and one normal hemoglobin gene. Normal hemoglobin is called type A. Sickle hemoglobin called S. Sickle cell trait is the presence of hemoglobin AS on the hemoglobin electrophoresis. This will NOT cause sickle cell disease.

    Are there different types of sickle cell disease?

    There are three commonly diagnosed types of sickle cell disease:

    1. Hemoglobin SS or sickle cell anemia
    2. Hemoglobin SC disease
    3. Hemoglobin sickle beta-thalassemia

    Each of these can cause sickle pain episodes and complications, but some are more common than others. All of these may also have an increase in fetal hemoglobin which can protect the red cell from sickling and help prevent complications. The medication hydroxyurea also increases fetal hemoglobin.

    There are three commonly diagnosed types of sickle cell disease:
    Hemoglobin SS or sickle cell anemia
    Hemoglobin SC disease
    Hemoglobin sickle beta-thalassemia
    Each of these can cause sickle pain episodes and complications, but some are more common than others. All of these may also have an increase in fetal hemoglobin which can protect the red cell from sickling and help prevent complications. The medication hydroxyurea also increases fetal hemoglobin.
    What are the complications?
    Complications from the sickle cells blocking blood flow and early breaking apart include:

    1. Pain episodes
    2. Strokes
    3. Increased infections
    4. Leg ulcers
    5. Bone damage
    6. Yellow eyes or jaundice
    7. Early gallstones
    8. Lung blockage
    9. Kidney damage and loss of body water in urine
    10. Painful erections in men (priapism)
    11. Blood blockage in the spleen or liver (sequestration)
    12. Eye damage
    13. Low red blood cell counts (anemia)
    14. Delayed growth

    What can be done to help prevent these complications?
    Sickle cell patients should be under the care of a medical team that understands sickle cell disease. All newborn babies detected with sickle cell disease should be placed on daily penicillin and monitored closely to prevent serious infections and maintain their well-being. All of the childhood immunizations should be given plus the pneumococcal vaccine. Parents should know how to check for a fever because this signals the need for a quick medical checkup for serious infection.

    The following are general guidelines to keep the sickle cell patient healthy:

    1. Taking the vitamin folic acid (folate) daily to help make new red cells
    2. Daily penicillin until age six to prevent serious infection
    3. Drinking plenty of water daily (8-10 glasses for adults)
    4. Avoiding too hot or too cold temperatures
    5. Avoiding over exertion and stress
    6. Getting plenty of rest
    7. Getting regular check-ups from knowledgeable health care providers

    Patients and families should watch for the following conditions that need an urgent medical evaluation:

    1. Fever
    2. Chest pain
    3. Shortness of Breath
    4. Increasing tiredness
    5. Abdominal swelling
    6. Unusual headache
    7. Any sudden weakness or loss of feeling
    8. Pain that will not go away with home treatment
    9. Priapism (painful erection that will not go down)
    10. Sudden vision change

    Acknowledgement: Some of the information on this page was a courtesy of:

    1. Toronto General Hospital website and Dr. Richard Ward (TGH)
    2. Ontario Newborn Screening website and Dr. Robert Klaassen (CHEO)
    3. Grady Memorial Hospital’s Sickle Cell Information Centre and National Heart, Lung, and Blood Institute websites