Trial Outcomes & Findings for Alpha-1 Carrier Genomics Study (NCT NCT02810327)

NCT ID: NCT02810327

Last Updated: 2019-07-22

Results Overview

Additional SERPINA1 variant of known or possible significance detected by next generation sequencing.

Recruitment status

COMPLETED

Target enrollment

117 participants

Primary outcome timeframe

End of study NGS Result

Results posted on

2019-07-22

Participant Flow

Participant milestones

Participant milestones
Measure
MZ Heterozygote With Symptoms of COPD
Individuals who have previously had testing for Alpha-1 antitrypsin deficiency with genotype MZ (PiMZ) results. Individuals in this cohort have symptoms or clinical diagnosis of COPD. Genetic Sequencing: Sequencing of the SERPINA1 gene
MZ Heterozygote Without Symptoms of COPD
Individuals who have previously had testing for Alpha-1 antitrypsin deficiency with genotype MZ (PiMZ) results. Individuals in this cohort do not have symptoms or clinical diagnosis of COPD. Genetic Sequencing: Sequencing of the SERPINA1 gene
Overall Study
STARTED
76
41
Overall Study
COMPLETED
69
34
Overall Study
NOT COMPLETED
7
7

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

14 consented participants did not return their test kits and/or questionnaires.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
MZ Heterozygote With Symptoms of COPD
n=76 Participants
Individuals who have previously had testing for Alpha-1 antitrypsin deficiency with genotype MZ (PiMZ) results. Individuals in this cohort have symptoms or clinical diagnosis of COPD. Genetic Sequencing: Sequencing of the SERPINA1 gene
MZ Heterozygote Without Symptoms of COPD
n=41 Participants
Individuals who have previously had testing for Alpha-1 antitrypsin deficiency with genotype MZ (PiMZ) results. Individuals in this cohort do not have symptoms or clinical diagnosis of COPD. Genetic Sequencing: Sequencing of the SERPINA1 gene
Total
n=117 Participants
Total of all reporting groups
Age, Continuous
Age
51.7 years
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
52.8 years
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
52 years
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Sex: Female, Male
Female
50 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
28 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
78 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Sex: Female, Male
Male
19 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
6 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
25 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Race (NIH/OMB)
Asian
0 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Race (NIH/OMB)
Black or African American
1 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
1 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Race (NIH/OMB)
White
68 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
34 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
102 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Race (NIH/OMB)
More than one race
0 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=69 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=34 Participants • 14 consented participants did not return their test kits and/or questionnaires.
0 Participants
n=103 Participants • 14 consented participants did not return their test kits and/or questionnaires.

PRIMARY outcome

Timeframe: End of study NGS Result

Additional SERPINA1 variant of known or possible significance detected by next generation sequencing.

Outcome measures

Outcome measures
Measure
MZ Heterozygote With Symptoms of COPD
n=69 samples
Individuals who have previously had testing for Alpha-1 antitrypsin deficiency with genotype MZ (PiMZ) results. Individuals in this cohort have symptoms or clinical diagnosis of COPD. Genetic Sequencing: Sequencing of the SERPINA1 gene
MZ Heterozygote Without Symptoms of COPD
n=34 samples
Individuals who have previously had testing for Alpha-1 antitrypsin deficiency with genotype MZ (PiMZ) results. Individuals in this cohort do not have symptoms or clinical diagnosis of COPD. Genetic Sequencing: Sequencing of the SERPINA1 gene
Number of Participants With Abnormal Sequences in SERPINA1 Genes
6 participants
0 participants

Adverse Events

MZ Heterozygote With Symptoms of COPD

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

MZ Heterozygote Without Symptoms of COPD

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Kimberly Brown Foil

Medical University of South Carolina

Phone: 843-792-6474

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place