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Infusion of MSC

Phase 1

Discordant Immunological Response in HIV Infected Subjects | Small molecule | Infectious Disease |Design Therapeutics, Inc.|Last Updated: Mar 4, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment5
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02290041Treatment With MSC in HIV-infected Patients With Controlled Viremia and Immunological Discordant ResponsePHASE1 COMPLETED 5Feb 8, 2017Jul 30, 2019Mar 4, 20201 Spain
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Study Endpoints
Primary Endpoints
Incidence of adverse reactions
24 months

Incidence of adverse reactions grade 3 and 4 according to DAIDs scale

Incidence of opportunist diseases
24 months
Changes in CD4+ cell count and CD4+/CD8+ ratio
28 days after the 4th infusion MSCs/placebo

Changes in CD4+ cell count and CD4+/CD8+ ratio as measured by flow citometry

Secondary Endpoints
T CD4 +/µl count evolution and CD4 +/CD8 + ratio throughout 48 weeks
48 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Mesenchymal stem cellsEXPERIMENTALIntravenous infusion of 4 doses of allogenic adult mesenchymal stem cells from adipose tissue
PlaceboPLACEBO_COMPARATORIntravenous infusion of 4 doses of Placebo
Interventions
NameTypeDescription
Infusion of MSCDRUGIntravenous infusion of 4 doses of adipose tissue derived allogeneic adult mesenchymal stem cells (1 million MSCs/Kg, weeks 0-4-8-20).
Infusion of placeboDRUGInfusion of placebo (weeks 0-4-8-20)
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Confirmed HIV infection * Age\> 18 years, both sexes * In treatment with antiretroviral therapy (ART) * Sustained HIV viral load \<50 copies / ml for ≥ 1 years prior to study entry * CD4 + cell count \< 350/mL * Immunological discordant response defined as: an increase \<75 or...

Countries:Spain
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