Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency

NCT ID: NCT03385395

Last Updated: 2018-04-10

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-07-31
• Study Completion Date: 2019-12-31

Brief Summary

This randomized trial is being conducted to show non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state. This will be conducted in individuals with alpha-1-antitrypsin deficiency and clinical evidence of emphysema.

Conditions

Alpha 1-Antitrypsin Deficiency

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

OctaAlpha1

Group Type: EXPERIMENTAL

OctaAlpha1

Intervention Type: DRUG

For OctaAlpha1 the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions

Glassia®

Group Type: ACTIVE_COMPARATOR

Glassia

Intervention Type: DRUG

For Glassia the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions

Interventions

OctaAlpha1

For OctaAlpha1 the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions

Intervention Type: DRUG

Glassia

For Glassia the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Any subject who needs chronic IV augmentation and maintenance therapy with A1PI because of congenital alpha-1-proteinase inhibitor (A1PI) deficiency and clinically diagnosed emphysema
• ≥18 years of age
• Individuals with A1PI serum concentration <11 µM at screening
• Following bronchodilators:

• Initial FEV1(pred) between 25% and 75% or
• If the initial FEV1 was greater than 75% of predicted, a diffusing capacity of the lung for carbon monoxide (DLC O) less than 70% of predicted
• Following bronchodilators: Initial forced expiratory volume/forced vital capacity (FEV1/FVC) ratio less than 70%
• Non-smoking for at least 6 months before study treatment starts
• Able to understand and provide written informed consent
• Women of reproductive age: negative result of pregnancy test (human chorionic gonadotropin [HCG]-based assay) and agreement to use adequate contraception for the duration of the trial

Exclusion Criteria

• Any inflammatory condition or malignant tumor in the 7 days before treatment starts that according to investigator judgment might influence the metabolism of an enzyme inhibitor such as A1PI
• More than one A1PI-deficiency related exacerbation and/or hospitalization during the 3 months before study treatment starts
• Clinically significant liver or kidney disease in the preceding 6 months before study treatment starts
• Severe gas exchange abnormality (i.e., PaCO2 ≥46 mmHg)
• Known IgA deficiency with documented antibodies against IgA
• History of hypersensitivity to blood or plasma derived products, or any component of the product
• Known presence of antibodies against A1PI
• Seropositivity for HBsAg or HCV, HIV-1/2 IgG antibodies
• Administration of A1PI products in the 4 weeks before study treatment starts
• Participating in another clinical study currently or during the 3 months before study treatment starts.
• Live viral vaccination within the last month before study treatment starts
• A current life-threatening malignancy
• Emergency operation within 3 months before study treatment starts
• History of, or suspected, alcohol or drug abuse within 1 year before study treatment starts or currently on drug abuse therapy
• Pregnant and nursing women

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Octapharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Other Identifiers

OctaAlpha1

Identifier Type: -

Identifier Source: org_study_id