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

April 6, 2018 updated by: Octapharma

A Randomized, Double-blind, Parallel-group, Multicenter, Pharmacokinetic Study Comparing Weekly Intravenous Administration of OctaAlpha1 (Octapharma) With a Marketed Preparation Glassia® (Kamada Ltd.) in Subjects With Alpha-1-antitrypsin Deficiency

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: OctaAlpha1
Drug: OctaAlpha1
For OctaAlpha1 the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions
Active Comparator: Glassia®
Drug: Glassia
For Glassia the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state
Time Frame: 26 weeks
Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state
26 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (AUC)
Time Frame: Time period including days 1 to 14 after first infusion in study
Compare PK parameters following a single dose between the two treatment groups calculating area under the plasma concentration-time curve (AUC)-ratio: 90% confidence interval (CI) should lie within 80%-125%.
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 Frame: Time period including days 1 to 14 after first infusion in study
Compare PK parameters following a single dose between the two treatment groups calculating maximum plasma concentration (Cmax)-ratio: 90% CI should lie within 80%-125%
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 Frame: Time period including days 1 to 14 after first infusion in study
Compare PK parameters following a single dose between the two treatment groups calculating tmax (time to reach maximum serum concentration)
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 Frame: Time period including days 1 to 14 after first infusion in study
Compare PK parameters following a single dose between the two treatment groups calculating t1/2 (apparent terminal half-life)
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 Frame: Time period including days 1 to 14 after first infusion in study
Compare PK parameters following a single dose between the two treatment groups calculating λZ (apparent terminal elimination rate constant determined by log-linear regression analysis)
Time period including days 1 to 14 after first infusion in study
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on occurrence of adverse events
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on occurrence of adverse events
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN)
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN) via lab test
26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19)
Time Frame: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19)
26 weeks
Trough Levels of A1PI
Time Frame: 26 weeks
Investigate descriptively the trough levels of A1PI and anti-NE capacity of OctaAlpha1 compared to Glassia®
26 weeks
Pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests
Time Frame: 26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests (done according to the American Thoracic Society/European Respiratory Society Taskforce Standardisation of Lung Function Testing guideline)
26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total A1PI in the airway lining fluid of the lung.
Time Frame: 26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total A1PI in the airway lining fluid of the lung.
26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total LTB4 in the airway lining fluid of the lung.
Time Frame: 26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total LTB4 in the airway lining fluid of the lung.
26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory
Time Frame: 26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory
26 weeks
Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1
Time Frame: 26 weeks
Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1
26 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Octapharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2018

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2019

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 1, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 20, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 28, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 10, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 6, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • OctaAlpha1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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