Recent Updates
Recently added Catalysts

Aerosolized, Recombinant Alpha 1-Antitrypsin

Phase 1

Alpha1-antitrypsin Deficiency | Small molecule | Other |Takeda Pharmaceutical Company Limited|Last Updated: May 5, 2021

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedUNCONTROLLEDBiomarker
Total Trials2
Total Enrollment55
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00157092Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin DeficiencyPHASE1 COMPLETED 15Mar 1, 2004Oct 14, 2004May 5, 20211 United States
NCT00161707Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin DeficiencyPHASE1 COMPLETED 40Jan 7, 2003Oct 1, 2003May 5, 20214 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Alpha 1-antitrypsin levels assessed from epithelial lining fluid (ELF) samples
Baseline (minimum of 3 to a maximum of 4 weeks prior to the first administration of study product).
Number of participants who develop antibodies to Recombinant Alpha 1-Antitrypsin (rAAT)
6 weeks after the first inhalation of study drug
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Interventions
NameTypeDescription
Aerosolized, Recombinant Alpha 1-AntitrypsinDRUG -
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Signed Informed Consent * Male or female 18 years of age or older * Documented, endogenous plasma AAT levels \< 11 µM (\< 80 mg/dL), either obtained from the medical history or, if not documented, plasma AAT measured after 28 day washout of any prior replacement therapy * Forc...

Countries:United States
Unlock Eligibility Criteria