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Immune Globulin

Phase 1

Sickle Cell Disease | Small molecule | Hematology |Grifols, S.A.|Last Updated: Mar 31, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment300
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01757418Intravenous Gammaglobulin for Sickle Cell Pain CrisesPHASE1 COMPLETED 300Nov 1, 2008Dec 18, 2024Mar 31, 20261 United States
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Study Endpoints
Primary Endpoints
Length of vaso-occlusive crisis (VOC)
Number of days from time of presentation to emergency room to end of crisis, average 4 days and maximum 30 days

Length (duration) of vaso-occlusive crisis as measured from the time of presentation to the emergency room to end of VOC defined as 12 hours from the last dose of parenteral opioid analgesia for the treatment of VOC prior to hospital discharge. Group results will be summarized in number of days using univariate statistics.

Secondary Endpoints
Total Opioid Use
From study drug infusion to end of crisis, average 4 days and maximum 30 days
Time to end of vaso-occlusive crisis
Number of days from start of study drug infusion to end of crisis, average 4 days and maximum 30 days
Length of Hospitalization
From admission to discharge, average 4 days and maximum 30 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Intravenous Immune Globulin (IVIG)EXPERIMENTALIVIG used in the trial is the GAMUNEX brand, at doses up through 800 mg/kg in Phase 1 and at 400mg/kg in Phase 2.
Normal salinePLACEBO_COMPARATORAn equivalent volume (weight-based) of normal saline
Interventions
NameTypeDescription
Immune Globulin Intravenous (IVIG)DRUGA single dose of intravenous immune globulin administered within 24 hours of hospital presentation. The maximum dose in Phase I was 800 mg/kg. The dose for Phase II is 400mg/kg.
Normal salineOTHERA single dose of normal saline administered within 24 hours of hospital admission for uncomplicated pain crisis.
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Eligibility Criteria
Age Range6 Years — 13 Years
SexALL
Healthy VolunteersNo
Study Sites1

Each subject must fulfill each of the following Inclusion/Exclusion criteria at screening and continue to fulfill these criteria prior to dosing: Inclusion Criteria: * Documented Sickle Cell Disease (SS or S-β thalassemia genotype) * Age 12-65 years for Phase 1 (Completed), 6-13.99 years for Phase...

Countries:United States
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