Trial Outcomes & Findings for Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency (NCT NCT02870309)
NCT ID: NCT02870309
Last Updated: 2021-10-28
Results Overview
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether considered related to the medicinal product. TEAEs were defined as any AE occurring after or on the first Alpha-1 MP infusion until the final visit of study.
COMPLETED
PHASE1/PHASE2
4 participants
Up to Week 12
2021-10-28
Participant Flow
The trial was conducted at 2 medical institutions in Japan from 29 July 2016 to 15 March 2017.
Participant milestones
| Measure |
Alpha-1 MP
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Overall Study
STARTED
|
4
|
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Overall Study
COMPLETED
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4
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency
Baseline characteristics by cohort
| Measure |
Alpha-1 MP
n=4 Participants
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Age, Continuous
|
60.8 years
STANDARD_DEVIATION 5.56 • n=93 Participants
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|
Sex: Female, Male
Female
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1 Participants
n=93 Participants
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Sex: Female, Male
Male
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3 Participants
n=93 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
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4 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=93 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=93 Participants
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Race (NIH/OMB)
White
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0 Participants
n=93 Participants
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|
Race (NIH/OMB)
More than one race
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0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
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PRIMARY outcome
Timeframe: Up to Week 12Population: Safety population included all the participants who received any amount of Alpha-1 MP.
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether considered related to the medicinal product. TEAEs were defined as any AE occurring after or on the first Alpha-1 MP infusion until the final visit of study.
Outcome measures
| Measure |
Alpha-1 MP
n=4 Participants
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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3 Participants
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PRIMARY outcome
Timeframe: Up to Week 12Population: Safety population included all the participants who received any amount of Alpha-1 MP.
ADRs were defined as adverse events (AEs) which were in the investigator's opinion of causal relationship to the study treatment. AE was defined as any unfavorable and unintended signs, symptoms or diseases temporally associated with the use of a medicinal product.
Outcome measures
| Measure |
Alpha-1 MP
n=4 Participants
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Number of Participants With Adverse Drug Reaction (ADRs)
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1 Participants
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PRIMARY outcome
Timeframe: Up to Week 12Population: Safety population included all the participants who received any amount of Alpha-1 MP.
A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
Outcome measures
| Measure |
Alpha-1 MP
n=4 Participants
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Number of Participants With Serious Adverse Events (SAEs)
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0 Participants
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PRIMARY outcome
Timeframe: Up to Week 12Population: Safety population included all the participants who received any amount of Alpha-1 MP.
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether considered related to the medicinal product. TEAEs were defined as any AE occurring after or on the first Alpha-1 MP infusion until the final visit of study. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
Outcome measures
| Measure |
Alpha-1 MP
n=4 Participants
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Number of Discontinuations Due to Adverse Events (AEs) or Serious Adverse Events (SAEs)
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0 Participants
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PRIMARY outcome
Timeframe: Up to Week 12Population: Safety population included all the participants who received any amount of Alpha-1 MP.
COPD exacerbation was defined as an increase in respiratory symptoms (dyspnea, increased cough, and/or production of sputum) over baseline that usually requires medical intervention.
Outcome measures
| Measure |
Alpha-1 MP
n=4 Participants
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations
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0 Participants
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SECONDARY outcome
Timeframe: Baseline (Week 1), Weeks 7, 8 (prior to the start of infusions of Alpha-1 MP) and Week 9 (168 hours post infusion)Population: Pharmacokinetic (PK) population included all the participants who received Alpha-1 MP dose and had sufficient samples to calculate the PK parameters. Number analyzed is the number of participants with data available for analyses at the given timepoint.
Outcome measures
| Measure |
Alpha-1 MP
n=4 Participants
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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Trough Level of Total alpha1-PI for Weekly IV Infusions of 60 mg/kg Alpha-1 MP
Week 1
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11.450 milligram/decilitre
Standard Deviation 3.5162
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Trough Level of Total alpha1-PI for Weekly IV Infusions of 60 mg/kg Alpha-1 MP
Week 7
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55.567 milligram/decilitre
Standard Deviation 7.2231
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Trough Level of Total alpha1-PI for Weekly IV Infusions of 60 mg/kg Alpha-1 MP
Week 8
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55.425 milligram/decilitre
Standard Deviation 7.2325
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Trough Level of Total alpha1-PI for Weekly IV Infusions of 60 mg/kg Alpha-1 MP
Week 9
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56.900 milligram/decilitre
Standard Deviation 12.9378
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Adverse Events
Alpha-1 MP
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Alpha-1 MP
n=4 participants at risk
Participants received 8 IV infusions of 60 mg/kg Alpha-1 MP administered weekly at an infusion rate not exceeding 0.08 mL/kg/min over approximately 15 minutes, up to Week 8.
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|---|---|
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General disorders
Fatigue
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25.0%
1/4 • From the time of informed consent through the end of study (Up to Week 12).
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Hepatobiliary disorders
Hepatic function abnormal
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25.0%
1/4 • From the time of informed consent through the end of study (Up to Week 12).
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Infections and infestations
Viral upper respiratory tract infection
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25.0%
1/4 • From the time of informed consent through the end of study (Up to Week 12).
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Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
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25.0%
1/4 • From the time of informed consent through the end of study (Up to Week 12).
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Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
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50.0%
2/4 • From the time of informed consent through the end of study (Up to Week 12).
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Site may publish results from the Study, after providing Sponsor thirty days' notice prior to submitting a manuscript or other materials related to the Study to any outside party. At Sponsors' request, Site will remove any Confidential Information (other than Study results), and Site will upon Sponsors' request, delay publication or presentation for a period of up to one hundred twenty days to allow Sponsor to protect its interests in any Sponsor Inventions.
- Publication restrictions are in place
Restriction type: OTHER