A 52-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group, group sequential, Phase 3 study to evaluate the efficacy and safety of rilzabrutinib in patients aged 10 to 65 years with sickle-cell disease

Conditions

Blood and lymphatic diseases

Brief summary

Annualized rate of clinical VOC

Detailed description

Time to first clinical VOC incidence, Annualized rate of visits due to SCD-related complications as assessed by the Investigator, Annualized rate of home-managed VOCs as reported in the Sickle Cell Pain Crisis (SCPC) eDiary, Change in fatigue as measured by the PROMIS SF v1.0 Fatigue 13a total score (adults), Change in Hb levels, Change in fatigue as measured by the PedsQL Multidimensional Fatigue Scale total score (pediatric participants), Incidence of treatment emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) and adverse events leading to discontinuation., Incidence of potentially clinically significant laboratory, vital signs, and ECG abnormalities, Absolute number of simple and exchange blood transfusion, Number of days requiring acetaminophen, NSAID and/or short-acting opioid usage

Related papers

No related papers found yet.

Papers published before 2007-09-01 may not appear yet. Historical indexing is in progress.

Interventions

DRUGSar444671 placebo - matched placebo for test

DRUGRilzabrutinib

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Annualized rate of clinical VOC	—

Secondary

Measure	Time frame
Time to first clinical VOC incidence, Annualized rate of visits due to SCD-related complications as assessed by the Investigator, Annualized rate of home-managed VOCs as reported in the Sickle Cell Pain Crisis (SCPC) eDiary, Change in fatigue as measured by the PROMIS SF v1.0 Fatigue 13a total score (adults), Change in Hb levels, Change in fatigue as measured by the PedsQL Multidimensional Fatigue Scale total score (pediatric participants), Incidence of treatment emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) and adverse events leading to discontinuation., Incidence of potentially clinically significant laboratory, vital signs, and ECG abnormalities, Absolute number of simple and exchange blood transfusion, Number of days requiring acetaminophen, NSAID and/or short-acting opioid usage	—

Measure

Time frame

Time to first clinical VOC incidence, Annualized rate of visits due to SCD-related complications as assessed by the Investigator, Annualized rate of home-managed VOCs as reported in the Sickle Cell Pain Crisis (SCPC) eDiary, Change in fatigue as measured by the PROMIS SF v1.0 Fatigue 13a total score (adults), Change in Hb levels, Change in fatigue as measured by the PedsQL Multidimensional Fatigue Scale total score (pediatric participants), Incidence of treatment emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) and adverse events leading to discontinuation., Incidence of potentially clinically significant laboratory, vital signs, and ECG abnormalities, Absolute number of simple and exchange blood transfusion, Number of days requiring acetaminophen, NSAID and/or short-acting opioid usage

—

Countries

Belgium, France, Germany, Greece, Italy, Netherlands, Spain

Outcome results

None listed