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

Octapharma

Status and phase

Withdrawn

Phase 2

Conditions

Alpha 1-Antitrypsin Deficiency

Treatments

Drug: Glassia

Drug: OctaAlpha1

Study type

Interventional

Funder types

Industry

Identifiers

NCT03385395

OctaAlpha1

Details and patient eligibility

About

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.

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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