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Enriched Hematopoetic Stem Cell Infusion

Phase 1

Sickle Cell Disease | Monoclonal antibody | Hematology |Tourmaline Bio, Inc.|Last Updated: Oct 14, 2020

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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment9
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00777231Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesPHASE1 COMPLETED 9Jan 1, 2005Aug 1, 2013Oct 14, 20203 United States
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Study Endpoints
Primary Endpoints
Level of Donor Chimerism from Enriched Hematopoietic Stem Cell Engraftment
From one month to three years

Tests are done at key time points to monitor for donor chimerism by evaluating presence of bone marrow-derived hematopoietic stem cells.

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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Sickle Cell DiseaseEXPERIMENTALRecipients treated with an enriched hematopoetic stem cell infusion
Non-Malignant DisordersEXPERIMENTALRecipients treated with an enriched hematopoetic stem cell infusion
Aplastic AnemiaEXPERIMENTALRecipients treated with an enriched hematopoetic stem cell infusion
Sickle Cell Disease : Extended ProtocolEXPERIMENTALRecipients treated with an enriched hematopoetic stem cell infusion and Campath 1H conditioning
Interventions
NameTypeDescription
Enriched Hematopoetic Stem Cell InfusionBIOLOGICALEnriched Hematopoetic Stem Cell Infusion
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Eligibility Criteria
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: The following criteria are established to identify subjects with sickle cell disease (SCD) who have a high predicted morbidity and are at risk for early mortality. Subjects with S/S disease, S/C disease, Hemoglobin H disease, Alpha Thalassemia Major, Thalassemia Major (also know...

Countries:United States
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