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Alpha1-Proteinase Inhibitor

Phase 3

Alpha 1-Antitrypsin Deficiency | Small molecule | Other |Grifols, S.A.|Last Updated: Aug 21, 2014

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_CONTROLLEDBiomarker
Total Trials2
Total Enrollment115
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00301366The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) DeficiencyPHASE3 COMPLETED 38Jun 1, 2006Mar 1, 2007Aug 19, 201410 United States, United Kingdom
NCT00263887Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)PHASE2 COMPLETED 77Dec 1, 2003Jan 1, 2007Aug 21, 20143 Denmark, Sweden +1
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Study Endpoints
Primary Endpoints
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.
24 weeks

An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.

The Progression Rate of Emphysema Determined by Change in 15th Percentile of Lung Density Measured by Annual CT Scan of the Whole Lung
24 or 30 months
Secondary Endpoints
Change in Lung Density at Each Visit as Measured by Computed Tomography
24 or 30 months
The Frequency of Exacerbations as Determined by Patient Diary.
24 or 30 months
The Deterioration of the Lung Function Will be Assessed by Measurement of the Change in Forced Expiratory Volume at One Second (FEV1) and Transfer Factor of Carbon Monoxide (KCO)
24 or 30 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Alpha-1 Proteinase Inhibitor (Human), modified processEXPERIMENTALStudy the safety and tolerability of weekly infusions of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP, 60 mg/kg) over 20 weeks of therapy in adult Alpha-1 antitrypsin deficient subjects.
Group 1EXPERIMENTALProlastin
Group 2PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
alpha-1 proteinase inhibitor (human)DRUG60 mg/kg weekly for 20 weeks
Alpha1-Proteinase Inhibitor (Human)DRUGWeekly infusion of 60 mg/kg body weight for 2 years
Albumin (Human) 20%, United States Pharmacopeia (USP)DRUGWeekly infusion for 2 years. Albumin (Human) 20% will be diluted with 5% glucose to a final concentration of 2.0%.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Documented diagnosis of congenital Alpha1-antitrypsin deficiency * Documented forced expiratory volume in 1 second (FEV1 ) between 20% - 80% of predicted value within last 6 months. * Signed written informed consent prior to initiation of any study related procedures. Exclusi...

Countries:United StatesUnited KingdomDenmarkSweden
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