A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCD
ConditionSickle Cell Disease
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age12+
SponsorPfizer
About this trial
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female with SCD, HbSS and HbSB-zero
Participants with Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable by the Investigator.
For participants taking hydroxyurea 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.
Participants with SCD ages 12 years and older, inclusive at screening.
Disqualifiers
Participants who had more than 10 VOC within 12 months of screening
Female participant who is breastfeeding or pregnant
Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF or anytime during the screening period.
Trial design
Design model
Parallel
Treatments tested in this trial
Osivelotor
DrugTablets which contain drug substance
Treatment groups
389 Participants
are divided into 3 treatment groupsGroup A: Part BPlacebo comparator 1 intervention
Group B: OLEExperimental treatment 1 intervention
Group C: Part AActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
Part A
Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 as measured by change in osivelotor concentrations from baseline or percentage change from baseline of clinical measures of anemia Hb and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).
Time frame
Through week 122
Part B
Co-primary endpoints: Hb response (increase from baseline of \>1 g/dL) at Week 48 (based on average of Hb levels at Week 40 and Week 48) and the Annualized rate of VOC through end of Week 48. 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. Complicated VOCs of acute chest syndrome (ACS), hepatic sequestration, splenic sequestration, priapism, and dactylitis that meet the requirements listed above will be included in this co-primary endpoint.
Time frame
Through week 483
OLE
Incidence of Treatment Emergent Adverse Events: * Incidence of SAEs * Incidence of AEs leading to discontinuation * Change from baseline in laboratory parameters.
Time frame
Approximately 24 months after last patient enrolled Source ClinicalTrials.gov