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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

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Market & Valuation

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Trial Design

RandomizedUNCONTROLLEDBiomarker
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
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Interventions

NameTypeDescription
Aerosolized, Recombinant Alpha 1-AntitrypsinDRUG -
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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
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Frequently asked questions about Aerosolized, Recombinant Alpha 1-Antitrypsin

What is Aerosolized, Recombinant Alpha 1-Antitrypsin used for?

Aerosolized, Recombinant Alpha 1-Antitrypsin is an investigational drug being developed for the treatment of Alpha1-antitrypsin Deficiency, a rare genetic condition. It is administered via aerosolization and is currently in Phase 1 clinical development. The drug is not yet approved and remains under investigation for this indication.

Who makes Aerosolized, Recombinant Alpha 1-Antitrypsin?

Aerosolized, Recombinant Alpha 1-Antitrypsin is being developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company. Takeda's stock is listed under the ticker symbol TAK. The company is conducting clinical trials to evaluate the safety and effects of this investigational drug.

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

Aerosolized, Recombinant Alpha 1-Antitrypsin is in Phase 1 clinical development. Two Phase 1 trials have been completed, with a total of 55 participants enrolled. The drug is investigational and has not received regulatory approval. It is being studied for the treatment of Alpha1-antitrypsin Deficiency.

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

Aerosolized, Recombinant Alpha 1-Antitrypsin has been studied in two completed Phase 1 clinical trials. NCT00157092 evaluated its effect on epithelial lining fluid analytes in 15 subjects, and NCT00161707 assessed its safety in 40 subjects. Both trials were conducted in the United States and enrolled adults with Alpha1-antitrypsin Deficiency.

Is Aerosolized, Recombinant Alpha 1-Antitrypsin FDA approved?

Aerosolized, Recombinant Alpha 1-Antitrypsin is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for Alpha1-antitrypsin Deficiency. The completed trials have focused on safety and biomarker effects, but the drug has not yet received regulatory approval for any use.