Alpha 1-Antitrypsin DeficiencyPhase 2NCT03385395
Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency
A Phase 2 study testing OctaAlpha1 and Glassia in people with Alpha 1-Antitrypsin Deficiency, run by Octapharma.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- What is being tested?
- OctaAlpha1 and Glassia
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jul 2018to Dec 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
About this study
Official title: 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.
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- OctaAlpha1, Glassia
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OctaAlpha1Drug
For OctaAlpha1 the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions
GlassiaDrug
For Glassia the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions
| Study group | Role | What they receive |
|---|---|---|
| OctaAlpha1 | Experimental | Not described Receives: Drug: OctaAlpha1 |
| Glassia® | Active comparator | Not described Receives: Drug: Glassia |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 26 weeks
Secondary outcomes (25)
Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (AUC)
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%.
Measured: 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)
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%
Measured: 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)
Compare PK parameters following a single dose between the two treatment groups calculating tmax (time to reach maximum serum concentration)
Measured: 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)
Compare PK parameters following a single dose between the two treatment groups calculating t1/2 (apparent terminal half-life)
Measured: 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)
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)
Measured: 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
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on occurrence of adverse events
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN)
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN) via lab test
Measured: 26 weeks
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19)
Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19)
Measured: 26 weeks
Trough Levels of A1PI
Investigate descriptively the trough levels of A1PI and anti-NE capacity of OctaAlpha1 compared to Glassia®
Measured: 26 weeks
Pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests
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)
Measured: 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.
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.
Measured: 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.
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.
Measured: 26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory
Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory
Measured: 26 weeks
Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1
Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2017
- Study startJul 2018 (estimated)
- Primary completionDec 2019 (estimated)
- Study completionDec 2019 (estimated)
Why it stopped: IND has been closed.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha 1-Antitrypsin DeficiencyStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03385395
- Sponsor's protocol ID
- OctaAlpha1
- Status last verified
- Apr 2018
- First submitted
- 1 Dec 2017
- Last updated
- 10 Apr 2018
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT03385395
- Acronym / sponsor ID
- OctaAlpha1
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: IND has been closed.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- Jul 2018 (estimated)
- Primary completion
- Dec 2019 (estimated)
- Study completion
- Dec 2019 (estimated)
- First posted
- 28 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| OctaAlpha1 | Experimental | Drug: OctaAlpha1 |
| Glassia® | Active comparator | Drug: Glassia |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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:
- Following bronchodilators: Initial forced expiratory volume/forced vital capacity (FEV1/FVC) ratio less than 70%
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OctaAlpha1For OctaAlpha1 the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions | Drug | OctaAlpha1 |
| GlassiaFor Glassia the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions | Drug | Glassia® |
Primary outcome
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
25 secondary outcomes
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 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 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 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 following principles of bio-equivalence testing (λZ)
Time frame: 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 blood pressure
Time frame: 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 body temperature
Time frame: 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 monitoring hematological parameter hematocrit
Time frame: 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 white blood cells
Time frame: 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 hepatic parameter alanine aminotransferase
Time frame: 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 renal parameter creatinine
Time frame: 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 viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19)
Time frame: 26 weeks
Trough Levels of A1PI
Time frame: 26 weeks
Pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests
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.
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.
Time frame: 26 weeks
Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory
Time frame: 26 weeks
Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1
Time frame: 26 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2018.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing OctaAlpha1 and Glassia in people with Alpha 1-Antitrypsin Deficiency, run by Octapharma.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 10 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state (time frame: 26 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Apr 2018.