Trial Outcomes & Findings for A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT) (NCT NCT02477319)

Last Updated: 2020-05-20

Results Overview

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.

Recruitment status

COMPLETED

Target enrollment

452 participants

Primary outcome timeframe

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.

Results posted on

2020-05-20

Participant Flow

Enrollment Period: March 2015 - June 2017 (revised)

Participant milestones

Participant milestones
Measure	Part A (Cohort 1) • Cohort 1: Healthy Controls Observational	Part A (Cohort 2) Cohort 2: Partial CFTR function CF (class IV/V) Observational	Part A (Cohort 3) Cohort 3: Absent CFTR function CF (Class I/II) Observational	Part B F508del homozygous CF patients who initiated Lumacaftor/Ivacaftor Observational (pre/post study)
Overall Study STARTED	55	40	164	193
Overall Study COMPLETED	55	37	149	171
Overall Study NOT COMPLETED	0	3	15	22

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cohort 1 participants did not perform spirometry procedures (i.e. are missing baseline FEV1 % Predicted). Also, one Cohort 2 participant is missing baseline spirometry.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part A (Cohort 1) n=55 Participants • Cohort 1: Healthy Controls Observational	Part A (Cohort 2) n=40 Participants • Cohort 2: Partial CFTR function (CF class IV/V) Observational	Part A (Cohort 3) n=164 Participants • Cohort 3: Absent CFTR function (CF Class I/II)	Part B n=193 Participants F508del homozygous CF patients who initiated Lumacaftor/Ivacaftor Observational (pre/post study)	Total n=452 Participants Total of all reporting groups
Age, Continuous	25.6 years STANDARD_DEVIATION 10.1 • n=55 Participants	32.3 years STANDARD_DEVIATION 18.1 • n=40 Participants	21.8 years STANDARD_DEVIATION 9.6 • n=164 Participants	19.0 years STANDARD_DEVIATION 10.6 • n=193 Participants	22.0 years STANDARD_DEVIATION 11.7 • n=452 Participants
Age, Customized Age Category · >=6 - 11 Y	0 Participants n=55 Participants	0 Participants n=40 Participants	0 Participants n=164 Participants	45 Participants n=193 Participants	45 Participants n=452 Participants
Age, Customized Age Category · >= 12 - 17	13 Participants n=55 Participants	11 Participants n=40 Participants	77 Participants n=164 Participants	72 Participants n=193 Participants	173 Participants n=452 Participants
Age, Customized Age Category · >= 18 - 29	30 Participants n=55 Participants	13 Participants n=40 Participants	53 Participants n=164 Participants	42 Participants n=193 Participants	138 Participants n=452 Participants
Age, Customized Age Category · >= 30	12 Participants n=55 Participants	16 Participants n=40 Participants	34 Participants n=164 Participants	34 Participants n=193 Participants	96 Participants n=452 Participants
Sex: Female, Male Female	26 Participants n=55 Participants	24 Participants n=40 Participants	84 Participants n=164 Participants	106 Participants n=193 Participants	240 Participants n=452 Participants
Sex: Female, Male Male	29 Participants n=55 Participants	16 Participants n=40 Participants	80 Participants n=164 Participants	87 Participants n=193 Participants	212 Participants n=452 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=55 Participants	0 Participants n=40 Participants	0 Participants n=164 Participants	1 Participants n=193 Participants	1 Participants n=452 Participants
Race (NIH/OMB) Asian	6 Participants n=55 Participants	0 Participants n=40 Participants	0 Participants n=164 Participants	1 Participants n=193 Participants	7 Participants n=452 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=55 Participants	0 Participants n=40 Participants	0 Participants n=164 Participants	0 Participants n=193 Participants	0 Participants n=452 Participants
Race (NIH/OMB) Black or African American	2 Participants n=55 Participants	2 Participants n=40 Participants	3 Participants n=164 Participants	0 Participants n=193 Participants	7 Participants n=452 Participants
Race (NIH/OMB) White	42 Participants n=55 Participants	38 Participants n=40 Participants	155 Participants n=164 Participants	189 Participants n=193 Participants	424 Participants n=452 Participants
Race (NIH/OMB) More than one race	3 Participants n=55 Participants	0 Participants n=40 Participants	6 Participants n=164 Participants	2 Participants n=193 Participants	11 Participants n=452 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=55 Participants	0 Participants n=40 Participants	0 Participants n=164 Participants	0 Participants n=193 Participants	2 Participants n=452 Participants
FEV1 % Predicted	—	86.9 % Predicted STANDARD_DEVIATION 24.6 • n=39 Participants • Cohort 1 participants did not perform spirometry procedures (i.e. are missing baseline FEV1 % Predicted). Also, one Cohort 2 participant is missing baseline spirometry.	81.8 % Predicted STANDARD_DEVIATION 23.4 • n=164 Participants • Cohort 1 participants did not perform spirometry procedures (i.e. are missing baseline FEV1 % Predicted). Also, one Cohort 2 participant is missing baseline spirometry.	85.0 % Predicted STANDARD_DEVIATION 22.4 • n=193 Participants • Cohort 1 participants did not perform spirometry procedures (i.e. are missing baseline FEV1 % Predicted). Also, one Cohort 2 participant is missing baseline spirometry.	83.9 % Predicted STANDARD_DEVIATION 23.1 • n=396 Participants • Cohort 1 participants did not perform spirometry procedures (i.e. are missing baseline FEV1 % Predicted). Also, one Cohort 2 participant is missing baseline spirometry.

PRIMARY outcome

Timeframe: 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.

Population: 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.

Outcome measures

Outcome measures
Measure	Part A (Cohort 1) n=53 Participants Cohort 1: Healthy Controls Observational	Part A (Cohort 2) n=40 Participants Cohort 2: Partial CFTR function (CF class IV/V)	Part A (Cohort 3) n=162 Participants Cohort 3: Absent CFTR function (CF class I/II)
Sweat Chloride by Cohort (Part A Only)	22.0 mmol/L Interval 19.2 to 24.7	81.0 mmol/L Interval 74.3 to 87.7	101.4 mmol/L Interval 100.1 to 102.7

PRIMARY outcome

Timeframe: Baseline and 6 months

Population: Change in FEV1 % predicted was measured in all participants who had baseline visit and 6 month visit.

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.

Outcome measures

Outcome measures
Measure	Part A (Cohort 1) n=176 Participants Cohort 1: Healthy Controls Observational	Part A (Cohort 2) Cohort 2: Partial CFTR function (CF class IV/V)	Part A (Cohort 3) Cohort 3: Absent CFTR function (CF class I/II)
6 Month Change in FEV1 Percent Predicted (Part B Only)	-0.2 Percent Predicted Interval -1.7 to 1.2	—	—

Adverse Events

All Arms/Groups

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

Umer Khan (Principal Biostatistician)

Seattle Children's Hospital

Phone: 206-884-7516

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place