Recent Updates
Recently added Catalysts

Aerosolized, Recombinant Alpha 1-Antitrypsin

Phase 1

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

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedUNCONTROLLED
Total Trials2
Total Enrollment55

FDA Designations

No designations recorded

Clinical trial landscape

Aerosolized, Recombinant Alpha 1-Antitrypsin · 2 trials · 1 indication

Phase 1 2
NCT00157092Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin DeficiencyAlpha1-antitrypsin Deficiency
COMPLETED15 Analytics
NCT00161707Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin DeficiencyAlpha1-antitrypsin Deficiency
COMPLETED40 Analytics
PHASE1COMPLETED
Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
Alpha1-antitrypsin DeficiencyUnlock trial analytics
PHASE1COMPLETED
Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency
Alpha1-antitrypsin DeficiencyUnlock trial analytics

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 to 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

Frequently asked questions about Aerosolized, Recombinant Alpha 1-Antitrypsin

What is Aerosolized, Recombinant Alpha 1-Antitrypsin?

Aerosolized, Recombinant Alpha 1-Antitrypsin is an investigational inhaled therapy being developed by Takeda Pharmaceutical Company Limited for Alpha1-antitrypsin Deficiency. It is designed to be delivered directly to the lungs as an aerosol. The drug is in Phase 1 clinical development and has been studied in two completed trials.

What is Aerosolized, Recombinant Alpha 1-Antitrypsin used for in Alpha1-antitrypsin Deficiency?

Aerosolized, Recombinant Alpha 1-Antitrypsin is being studied as a treatment for Alpha1-antitrypsin Deficiency, a rare genetic condition. The clinical program has evaluated its safety and its effect on epithelial lining fluid analytes in adults with the condition. It remains investigational and has not been approved for commercial use.

Who is developing Aerosolized, Recombinant Alpha 1-Antitrypsin?

Aerosolized, Recombinant Alpha 1-Antitrypsin is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the sponsor of the clinical program for this inhaled recombinant therapy in Alpha1-antitrypsin Deficiency.

What phase is Aerosolized, Recombinant Alpha 1-Antitrypsin in?

Aerosolized, Recombinant Alpha 1-Antitrypsin is in Phase 1 clinical development. Both of its completed trials were Phase 1 studies. The drug is investigational and has not been approved by the FDA or any regulatory agency for the treatment of Alpha1-antitrypsin Deficiency.

What clinical trials is Aerosolized, Recombinant Alpha 1-Antitrypsin in?

Aerosolized, Recombinant Alpha 1-Antitrypsin has been evaluated in two completed Phase 1 trials: NCT00161707, a safety study in 40 subjects with Alpha1-antitrypsin Deficiency, and NCT00157092, which studied the effect on epithelial lining fluid analytes in 15 subjects. Both trials were conducted in the United States and are now completed.