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A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT) (PROSPECT)

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: NCT02477319
Recruitment Status : Completed
First Posted : June 22, 2015
Results First Posted : May 20, 2020
Last Update Posted : May 20, 2020
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Steven M Rowe, University of Alabama at Birmingham

Study Type Observational
Study Design Observational Model: Other;   Time Perspective: Prospective
Condition Cystic Fibrosis
Intervention Other: Observational
Enrollment 452
Recruitment Details Enrollment Period: March 2015 - June 2017 (revised)
Pre-assignment Details  
Arm/Group Title Part A (Cohort 1) Part A (Cohort 2) Part A (Cohort 3) Part B
Hide Arm/Group Description

• Cohort 1: Healthy Controls

Observational

Cohort 2: Partial CFTR function CF (class IV/V)

Observational

Cohort 3: Absent CFTR function CF (Class I/II)

Observational

F508del homozygous CF patients who initiated Lumacaftor/Ivacaftor

Observational (pre/post study)

Period Title: Overall Study
Started 55 40 164 193
Completed 55 37 149 171
Not Completed 0 3 15 22
Arm/Group Title Part A (Cohort 1) Part A (Cohort 2) Part A (Cohort 3) Part B Total
Hide Arm/Group Description

• Cohort 1: Healthy Controls

Observational

• Cohort 2: Partial CFTR function (CF class IV/V)

Observational

• Cohort 3: Absent CFTR function (CF Class I/II)

F508del homozygous CF patients who initiated Lumacaftor/Ivacaftor

Observational (pre/post study)

Total of all reporting groups
Overall Number of Baseline Participants 55 40 164 193 452
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 55 participants 40 participants 164 participants 193 participants 452 participants
25.6  (10.1) 32.3  (18.1) 21.8  (9.6) 19.0  (10.6) 22.0  (11.7)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Age Category Number Analyzed 55 participants 40 participants 164 participants 193 participants 452 participants
>=6 - 11 Y
0
   0.0%
0
   0.0%
0
   0.0%
45
  23.3%
45
  10.0%
>= 12 - 17
13
  23.6%
11
  27.5%
77
  47.0%
72
  37.3%
173
  38.3%
>= 18 - 29
30
  54.5%
13
  32.5%
53
  32.3%
42
  21.8%
138
  30.5%
>= 30
12
  21.8%
16
  40.0%
34
  20.7%
34
  17.6%
96
  21.2%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 55 participants 40 participants 164 participants 193 participants 452 participants
Female
26
  47.3%
24
  60.0%
84
  51.2%
106
  54.9%
240
  53.1%
Male
29
  52.7%
16
  40.0%
80
  48.8%
87
  45.1%
212
  46.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 55 participants 40 participants 164 participants 193 participants 452 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.5%
1
   0.2%
Asian
6
  10.9%
0
   0.0%
0
   0.0%
1
   0.5%
7
   1.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
   3.6%
2
   5.0%
3
   1.8%
0
   0.0%
7
   1.5%
White
42
  76.4%
38
  95.0%
155
  94.5%
189
  97.9%
424
  93.8%
More than one race
3
   5.5%
0
   0.0%
6
   3.7%
2
   1.0%
11
   2.4%
Unknown or Not Reported
2
   3.6%
0
   0.0%
0
   0.0%
0
   0.0%
2
   0.4%
FEV1 % Predicted   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  % Predicted
Number Analyzed 0 participants 39 participants 164 participants 193 participants 396 participants
86.9  (24.6) 81.8  (23.4) 85.0  (22.4) 83.9  (23.1)
[1]
Measure Description: FEV1 % Predicted based on Global Lung Initiative (GLI) equations
[2]
Measure Analysis Population Description: Cohort 1 participants did not perform spirometry procedures (i.e. are missing baseline FEV1 % Predicted). Also, one Cohort 2 participant is missing baseline spirometry.
1.Primary Outcome
Title Sweat Chloride by Cohort (Part A Only)
Hide Description

This is the primary endpoint for Part A per the PROSPECT protocol. Mean sweat chloride was not reported for Part B, as it is not a relevant statistic.

For cohort 1, sweat chloride is from day 0 only. For cohorts 2-3, sweat chloride was averaged from days 0, 14, 90 via a random intercept longitudinal model.

Time Frame For cohort 1, sweat chloride at Day 0 is time frame. For cohorts 2-3, sweat chloride averaged across all 3 visits at days 0, 14 and 90 is time frame.
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with Sweat Chloride measure. For Cohort 1, sweat chloride was collected at Visit 1 only (Day 0). Cohort 2 and 3 were collected at Days 0, 14 and 90.
Arm/Group Title Part A (Cohort 1) Part A (Cohort 2) Part A (Cohort 3)
Hide Arm/Group Description:
Cohort 1: Healthy Controls Observational
Cohort 2: Partial CFTR function (CF class IV/V)
Cohort 3: Absent CFTR function (CF class I/II)
Overall Number of Participants Analyzed 53 40 162
Least Squares Mean (95% Confidence Interval)
Unit of Measure: mmol/L
22.0
(19.2 to 24.7)
81.0
(74.3 to 87.7)
101.4
(100.1 to 102.7)
2.Primary Outcome
Title 6 Month Change in FEV1 Percent Predicted (Part B Only)
Hide Description This is the primary endpoint for Part B per the PROSPECT protocol. Change in FEV1 Percent Predicted is only relevant for Part B as it captures changes in lung function post-initiation of Ivacaftor/Lumacaftor.
Time Frame Baseline and 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Change in FEV1 % predicted was measured in all participants who had baseline visit and 6 month visit.
Arm/Group Title Part B
Hide Arm/Group Description:
Participants who initiated Ivacaftor/Lumacaftor
Overall Number of Participants Analyzed 176
Mean (95% Confidence Interval)
Unit of Measure: Percent Predicted
-0.2
(-1.7 to 1.2)
Time Frame [Not Specified]
Adverse Event Reporting Description This was an observational study. Adverse events were not an endpoint.
 
Arm/Group Title All Arms/Groups
Hide Arm/Group Description Adverse Events were not analyzed by Arm/Group.
All-Cause Mortality
All Arms/Groups
Affected / at Risk (%)
Total   0/0 
Hide Serious Adverse Events
All Arms/Groups
Affected / at Risk (%)
Total   0/0 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
All Arms/Groups
Affected / at Risk (%)
Total   0/0 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Umer Khan (Principal Biostatistician)
Organization: Seattle Children's Hospital
Phone: 206-884-7516
EMail: umer.khan@seattlechildrens.org
Layout table for additonal information
Responsible Party: Steven M Rowe, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT02477319    
Other Study ID Numbers: PROSPECT
First Submitted: May 22, 2015
First Posted: June 22, 2015
Results First Submitted: April 7, 2020
Results First Posted: May 20, 2020
Last Update Posted: May 20, 2020