ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 10 of 76 for:    ALPHA-1-ANTITRYPSIN DEFICIENCY

A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02282527
Recruitment Status : Completed
First Posted : November 4, 2014
Results First Posted : March 13, 2017
Last Update Posted : March 13, 2017
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Alpha₁-Antitrypsin Deficiency
Interventions: Biological: Liquid Alpha₁-PI
Biological: Prolastin-C

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
This study was performed at 6 investigative centers in the US.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Liquid Alpha₁-PI/Prolastin-C

Subjects were treated first with Liquid Alpha₁-PI and then treated with Prolastin-C

Liquid Alpha₁-PI: Liquid Alpha₁-PI, 60 mg/kg, 8 weekly intravenous infusions

Prolastin-C: Prolastin-C, 60 mg/kg, 8 weekly intravenous infusions

Prolastin-C/Liquid Alpha₁-PI

Subjects were treated first with Prolastin-C and then treated with Liquid Alpha₁-PI

Prolastin-C: Prolastin-C, 60 mg/kg, 8 weekly intravenous infusions

Liquid Alpha₁-PI: Liquid Alpha₁-PI, 60 mg/kg, 8 weekly intravenous infusions


Participant Flow:   Overall Study
    Liquid Alpha₁-PI/Prolastin-C   Prolastin-C/Liquid Alpha₁-PI
STARTED   16   16 
COMPLETED   15   15 
NOT COMPLETED   1   1 
Lost to Follow-up                0                1 
Lack of home health aid                1                0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Liquid Alpha₁-PI/Prolastin-C

Subjects were treated first with Liquid Alpha₁-PI and then treated with Prolastin-C

Liquid Alpha₁-PI: Liquid Alpha₁-PI, 60 mg/kg, 8 weekly intravenous infusions

Prolastin-C: Prolastin-C, 60 mg/kg, 8 weekly intravenous infusions

Prolastin-C/Liquid Alpha₁-PI

Subjects were treated first with Prolastin-C and then treated with Liquid Alpha₁-PI

Prolastin-C: Prolastin-C, 60 mg/kg, 8 weekly intravenous infusions

Liquid Alpha₁-PI: Liquid Alpha₁-PI, 60 mg/kg, 8 weekly intravenous infusions

Total Total of all reporting groups

Baseline Measures
   Liquid Alpha₁-PI/Prolastin-C   Prolastin-C/Liquid Alpha₁-PI   Total 
Overall Participants Analyzed 
[Units: Participants]
 16   16   32 
Age 
[Units: Years]
Mean (Standard Deviation)
     
Participants Analyzed   16   16   32 
   60.7  (7.94)   63.1  (5.93)   61.9  (7.00) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Participants Analyzed   16   16   32 
Female      8  50.0%      6  37.5%      14  43.8% 
Male      8  50.0%      10  62.5%      18  56.3% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Participants Analyzed   16   16   32 
Hispanic or Latino      0   0.0%      1   6.3%      1   3.1% 
Not Hispanic or Latino      16 100.0%      15  93.8%      31  96.9% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
     
United States       
Participants Analyzed   16   16   32 
United States   16   16   32 
Alpha1-PI concentration [1] [2] 
[Units: mg/mL]
Mean (Standard Deviation)
     
Naive       
Participants Analyzed   1   3   4 
Naive   0.31 [2]   0.21  (0.040)   0.24  (0.059) 
Non-naive       
Participants Analyzed   14   13   27 
Non-naive   0.72  (0.283)   0.69  (0.215)   0.70  (0.248) 
[1] One subject in the Liquid Alpha₁-PI/Prolastin -C non-naïve population was missing baseline alpha₁-PI concentration due to sample instability.
[2] The standard deviation was not calculated as only 1 subject was assessed.


  Outcome Measures

1.  Primary:   AUC(0-7 Days) Based on Antigenic Content   [ Time Frame: pre-dose, 0, 15 min, 30 min, 1 hour, 2 hours, 4 hours, 8 hours, 1 day, 2 days, 5 days, 7 days post dose ]

2.  Secondary:   AUC(0-7 Days) Based on Functional Activity   [ Time Frame: pre-dose, 0, 15 min, 30 min, 1 hour, 2 hours, 4 hours, 8 hours, 1 day, 2 days, 5 days, 7 days post dose ]

3.  Secondary:   Number of Subjects With Immunogenicity Response   [ Time Frame: Weeks 1, 9, 17, and 20 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Henry Li
Organization: Grifols Therapeutics Inc.
phone: 1-919-316-6042
e-mail: henry.li@grifols.com



Responsible Party: Grifols Therapeutics LLC
ClinicalTrials.gov Identifier: NCT02282527     History of Changes
Other Study ID Numbers: GTI1402
First Submitted: October 31, 2014
First Posted: November 4, 2014
Results First Submitted: November 11, 2016
Results First Posted: March 13, 2017
Last Update Posted: March 13, 2017