A RANDOMIZED DOUBLE-BLIND PHASE IIA STUDY EVALUATING THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF CROVALIMAB AS ADJUNCT TREATMENT IN PREVENTION OF VASO-OCCLUSIVE EPISODES (VOE) IN SICKLE CELL DISEASE (SCD)

Conditions

Sickle Cell Disease

Brief summary

Annualized rate of medical facility VOEs (AVR)

Detailed description

Annualized rate of home VOE captured by patient report on a handheld device at home, Annualized rate of uncomplicated medical facility VOE, Annualized rate of acute chest syndrome (ACS), Annualized rate of days hospitalized for medical facility VOE, Annualized rate of days hospitalized for treatment of non-VOE complications of SCD, Change in hematologic measures from baseline to Week 49, Time to first medical facility VOE from randomization, Change in urinary albumin-creatinine ratio from baseline to Week 49, Change from baseline to Week 49 in tricuspid regurgitant jet velocity (TRV), Proportion of patients with TRV > 2.5 m/s at Week 49, Change from baseline to Week 49 in Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue score in adults

Related papers

No related papers found yet.

Interventions

None listed

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Annualized rate of medical facility VOEs (AVR)	—

Secondary

Measure	Time frame
Annualized rate of home VOE captured by patient report on a handheld device at home, Annualized rate of uncomplicated medical facility VOE, Annualized rate of acute chest syndrome (ACS), Annualized rate of days hospitalized for medical facility VOE, Annualized rate of days hospitalized for treatment of non-VOE complications of SCD, Change in hematologic measures from baseline to Week 49, Time to first medical facility VOE from randomization, Change in urinary albumin-creatinine ratio from baseline to Week 49, Change from baseline to Week 49 in tricuspid regurgitant jet velocity (TRV), Proportion of patients with TRV > 2.5 m/s at Week 49, Change from baseline to Week 49 in Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue score in adults	—

Measure

Time frame

Annualized rate of home VOE captured by patient report on a handheld device at home, Annualized rate of uncomplicated medical facility VOE, Annualized rate of acute chest syndrome (ACS), Annualized rate of days hospitalized for medical facility VOE, Annualized rate of days hospitalized for treatment of non-VOE complications of SCD, Change in hematologic measures from baseline to Week 49, Time to first medical facility VOE from randomization, Change in urinary albumin-creatinine ratio from baseline to Week 49, Change from baseline to Week 49 in tricuspid regurgitant jet velocity (TRV), Proportion of patients with TRV > 2.5 m/s at Week 49, Change from baseline to Week 49 in Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue score in adults

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Countries

France, Italy, Netherlands, Spain

Outcome results