The Pfizer DREAM study is now enrolling Phase 3, which is evaluating the efficacy and safety of an investigational medicine called Osivelotor (formerly GBT021601) in people with Sickle Cell Disease. (SCD).
The DREAM study is being conducted in 2 parts:
- Part A is a randomized, open-label, dose-finding study. Enrollment has been completed for Part A.
- Part B is a randomized, placebo-controlled, double-blind study.
About Osivelotor
Osivelotor is a potential HbS polymerization inhibitor which is designed to bind directly to HbS, to potentially reduce polymerization and red blood cell sickling.
Ongoing and future clinical trials will determine whether the investigational medication is safe and/or effective.
Osivelotor has the potential to achieve targeted percent Hb occupancy, while potentially attaining the desired hematological effect and reduction of vaso-occlusive crises (VOCs) at a low dose.
Osivelotor could reduce patient ‘pill-burden’, improving clinical outcomes with an improved side effect profile.
Key inclusion criteria
Documentation of sickle cell disease genotype HbSS or HbSB. May be based on history of laboratory testing or confirmed by laboratory testing during screening.
Aged:
- Part B: 12 years and older at Screening
For participants taking HU and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator and no sign of hematological toxicity.
If applicable, must agree to use a highly effective method of contraception or maintain sexual abstinence up to 84 days after the last dose of study drug.
Must have had ≥2 and ≤10 VOCs, within 12 months of Screening
Additional eligibility criteria will be assessed by the study site team during screening.
Key exclusion criteria
Must not be receiving regularly scheduled RBC transfusion therapy (i.e. chronic, prophylactic, or preventive transfusion) or have received an RBC or exchange transfusion for any reason within 90 days of starting the study treatment.
Must not have been hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF.
Received EPO or other hematopoietic growth factor treatment within 28 days of enrolling (or anticipated to require such agents during the study).
Current or recent use of:
-Voxelotor
-Crizanlizumab
-Strong or moderate inducers or inhibitors of CYP3A4/CYP3A5.
-P-glycoprotein substrates digoxin or dabigatran
Must not have had more than 10 VOCs within 12 months of Screening.
Female participants must not be breastfeeding or pregnant.
Additional eligibility criteria will be assessed by the study site team during screening.
Watch this video to learn more about Osivelotor
Osivelotor is investigational and not approved by any health authority.
People are often introduced to the world of clinical trials through conversations with their healthcare providers. As with anything new, there may be a number of questions, concerns, or misconceptions.
Being their trusted healthcare provider, we appreciate you taking the time to have an open discussion with patients and their loved ones who may be interested in participating.
If you would like more study-specific information or to connect with a Pfizer Field Medical Director please click here to email us.
If any of your patients take part in the DREAM study, rest assured that they will remain under your medical care for all non-study–related needs and will continue to receive any benefits to which they are entitled.