Last updated: Month X, 2022
Condition
Sickle Cell Disease
Age
18 and Older
Status
Actively Recruiting
The purpose of this research study is to determine how safe, how
well tolerated and how well each dose of the study drug
(GBT021601) works in the treatment of people who are living with SCD.
The
studies are
carefully
monitored to adhere to a
predefined protocol. They are the most important way researchers can confirm that a new drug or
treatment is safe and
effective.
GBT021601 is a hemoglobin S (HbS) polymerization inhibitor and an investigational trial drug for the potential treatment of sickle cell disease.
This is a randomized, double-blind, placebo controlled, once-daily treatment study in healthy participants. It is the first of a three-part multicenter, phase 2/3 study of orally administered GBT021601 in participants with SCD. Part A will evaluate the safety, tolerability, and efficacy of GBT021601 in adult participants with SCD.
Find out if you are eligible to participate in the DREAM trial..
Am
I eligible?
Study Enrollment Begins:
December 9, 2022
Total
Duration per
Participant:
Approximately 90 days
CONNECTICUT
Yale University
New Haven, CT 06519
GBT021601-021
GBT021601-021
WASHINGTON, DC
Children’s National Medical Center Hematology and Oncology
Washington, DC 20010
GBT021601-021
GBT021601-021
ILLINOIS
University of Illinois Medical Center
Chicago, IL 60612
GBT021601-021
GBT021601-021
FLORIDA
Palm Beach Children's Hospital
West Palm Beach, FL 33407
GBT021601-021
GBT021601-021
LOS ANGELES
Our Lady of the Lake
Baton Rouge, LA 70808
GBT021601-021
GBT021601-021
LOS ANGELES
University Medical Center New Orleans
New Orleans, LA 70112
GBT021601-021
GBT021601-021
NORTH CAROLINA
University of North Carolina Chapel Hill
Chapel Hill, NC 27514
GBT021601-021
GBT021601-021
ARKANSAS
Arkansas Primary Care Clinics
Little Rock, AR 72204
GBT021601-021
GBT021601-021
TEXAS
University of Texas Health, Houston
Houston, TX 77030
GBT021601-021
GBT021601-021
VIRGINIA
Inova Fairfax Hospital
Fairfax, VA 22031
GBT021601-021
GBT021601-021
VIRGINIA
Virginia Commonwealth University
Richmond, VA 23298
GBT021601-021
GBT021601-021
CALIFORNIA
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center
Torrance, CA 90502
GBT021601-021
GBT021601-021
Sickle cell disease (SCD) is a group of red blood cell disorders, where the red blood cells can change shape—from healthy, round, bendable cells to stiff, sticky, crescent shaped cells that die early and can easily get stuck and block healthy blood flow in the body. Sickle cell disease is not contagious. It is a genetic condition that is present at birth and is passed down from both parents.
STATS
It is estimated that:
Americans affected
by SCD
Black or African-American babies are born with SCD
Hispanic-American babies are born with SCD
of people living with SCD usually receive their care in an emergency room
SDC is a genetic, hereditary disease
Treatment can help, but this condition cannot be cured
Requires medical diagnosis
Lab tests or imaging always required
Chronic: lifelong
For informational purposes only. Consult your local medical authority for
advice.
Content ources: National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention
SYMPTOMS
Pain areas: in the joints
Pain types: can be sudden
in the
chest,
can include
vaso-occlusive crises (VOCs)
Whole
body: dizziness, fatigue, low oxygen in the body, or malaise
Urinary: inability to
make concentrated or dilute urine or
blood in urine
Also common: abnormal breakdown of red blood cells,
delayed
development, inflamed fingers or toes,
pallor, shortness of breath, or yellow skin and eyes
TREATMENT
Management of sickle cell is usually aimed at avoiding pain episodes, like VOCs, relieving symptoms and preventing complications. Treatments might include medications and blood transfusions. Vaccinations and penicillin can be used to help prevent life-threatening infections.
Would you like to know more?
Contact us at DREAMstudy@gbt.com
