The Pfizer DREAM study is evaluating the efficacy and safety of an investigational medicine called Osivelotor (formerly GBT021601) in people with Sickle Cell Disease (SCD).
This Phase 3 study is being conducted in 2 parts. Part A has completed enrollment. Part B, which will be enrolling, is a 48-week randomized, placebo-controlled, double-blind study, followed by an open-label extension phase for eligible participants.
About Osivelotor
Osivelotor is an HbS polymerization inhibitor designed to bind directly to HbS, to potentially reduce polymerization and red blood cell sickling.
Osivelotor has the potential to increase hemoglobin levels and reduce rates of vaso-occlusive crises (VOCs) at a low dose.
Ongoing and future clinical trials will determine whether the investigational medication is safe and/or effective.
Watch this video to learn more about Osivelotor
Osivelotor is investigational and not approved by any health authority.
Key Inclusion Criteria
- Documentation of sickle cell disease genotype HbSS or HbSβ0 thalassemia. May be based on history of laboratory testing or confirmed by laboratory testing during screening.
- Participants aged 12 years and older at Screening
- More than or equal to 2 and ≤ 10 VOCs, within 12 months of Screening. A VOC is defined as an acute episode of pain that:
- Has no medically determined cause other than a vaso-occlusive event, and
- Results in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), and
- Requires parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics.
- Hb ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable by the Investigator.
- 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.
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 or any time during the screening period.
- 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
- Mitapivat
- Strong or moderate inducers of CYP3A4/CYP3A5
- P-glycoprotein substrates digoxin or dabigatran
- Female participants must not be breastfeeding or pregnant.
Additional eligibility criteria will be assessed by the study site team during screening.
A Patient’s Story: Insights into Sickle Cell Disease and clinical trial participation
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 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 treatment.