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Alpha1-Proteinase Inhibitor

Phase 3

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment115

FDA Designations

No designations recorded

Clinical trial landscape

Alpha1-Proteinase Inhibitor · 3 trials · 2 indications

Phase 3 1Phase 2 2
NCT00301366The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) DeficiencyAlpha 1-Antitrypsin Deficiency
COMPLETED38 Analytics
PHASE3COMPLETED
The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
Alpha 1-Antitrypsin DeficiencyUnlock trial analytics

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
Change in Free Elastase in Induced Sputum From Baseline to Week 4
Baseline vs Week 4

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 to 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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Frequently asked questions about Alpha1-Proteinase Inhibitor

What is Alpha1-Proteinase Inhibitor used for?

Alpha1-Proteinase Inhibitor is being studied for the treatment of Cystic Fibrosis and Alpha 1-Antitrypsin Deficiency. It is an investigational therapy in clinical development, and it is not yet approved for these indications.

Who makes Alpha1-Proteinase Inhibitor?

Alpha1-Proteinase Inhibitor is being developed by Grifols, S.A., a biopharmaceutical company. Grifols is publicly traded under the ticker symbol GRFS.

What phase is Alpha1-Proteinase Inhibitor in?

Alpha1-Proteinase Inhibitor is in Phase 3 clinical development. It has completed two trials, including a Phase 3 study, and is still being evaluated for safety and efficacy in patients with Alpha 1-Antitrypsin Deficiency.

What clinical trials has Alpha1-Proteinase Inhibitor been in?

Alpha1-Proteinase Inhibitor has been studied in three completed clinical trials. These include NCT00263887, a Phase 2 study in Alpha 1-Antitrypsin Deficiency; NCT00301366, a Phase 3 safety and tolerability study in the same condition; and NCT00486837, a Phase 2 study in Cystic Fibrosis.

Is Alpha1-Proteinase Inhibitor the same as Alpha-1-Antitrypsin?

Alpha1-Proteinase Inhibitor is also known as Alpha-1-Antitrypsin. Clinical trials for this drug, such as NCT00263887 and NCT00301366, refer to it as Alpha-1-Antitrypsin (AAT) therapy.