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

NCT03385395 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: OctaAlpha1
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (1)

• Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state

  Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state

  26 weeks

Secondary Outcomes (25)

• Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (AUC)

  Time period including days 1 to 14 after first infusion in study

• Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (Cmax)

  Time period including days 1 to 14 after first infusion in study

• Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (tmax)

  Time period including days 1 to 14 after first infusion in study

• Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (t1/2)

  Time period including days 1 to 14 after first infusion in study

• Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (λZ)

  Time period including days 1 to 14 after first infusion in study

Study Arms (2)

OctaAlpha1

EXPERIMENTAL

Drug: OctaAlpha1

Glassia®

ACTIVE COMPARATOR

Drug: Glassia

Interventions

OctaAlpha1 DRUG

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

OctaAlpha1

Glassia DRUG

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

Glassia®

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Octapharma: lead

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Interventions

alpha 1-Antitrypsin

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Serpins; Peptides; Amino Acids, Peptides, and Proteins; Acute-Phase Proteins; Blood Proteins; Proteins; Alpha-Globulins; Serum Globulins; Globulins

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2017
• First Posted: December 28, 2017
• Study Start: July 1, 2018
• Primary Completion: December 1, 2019
• Study Completion: December 1, 2019
• Last Updated: April 10, 2018

Record last verified: 2018-04