Trial Outcomes & Findings for COLA: A Pilot Clinical Trial of COX-2 Inhibition in LAM and TSC (NCT NCT02484664)

NCT ID: NCT02484664

Last Updated: 2022-01-20

Results Overview

Number of Participants with Adverse Events as a Measure of Safety and Tolerability in LAM patients

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 12 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2022-01-20

Participant Flow

Participant milestones

Measure	Celecoxib Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.
Overall Study STARTED	12
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

COLA: A Pilot Clinical Trial of COX-2 Inhibition in LAM and TSC

Baseline characteristics by cohort

Measure	Celecoxib n=12 Participants Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	48 years n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	12 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	12 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Number of Participants with Adverse Events as a Measure of Safety and Tolerability in LAM patients

Outcome measures

Measure

Celecoxib n=12 Participants Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.

Number of Participants With Adverse Events as a Measure of Safety and Tolerability

11 Participants

SECONDARY outcome

Timeframe: 1 year

Forced expiratory volume in 1 second

Outcome measures

Measure

Celecoxib n=12 Participants Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.

FEV1

2583 ml Standard Error 166

SECONDARY outcome

Timeframe: 1 year

We are reporting the number of participants in this trial who had angiomyolipoma either at the beginning or end of the study.

Outcome measures

Measure

Celecoxib n=12 Participants Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.

Angiomyolipoma Size Measured Volumetrically on MRI

3 Participants

SECONDARY outcome

Timeframe: 1 year

Population: Nine of 12 patients who enrolled on the study stayed on study until the end

St. George's Respiratory Questionnaire is a commonly used questionnaire to assess the respiratory function of an individual.

The minimum and maximyum socres on this Questionnaire are: 0 and 100. A higher score shows more limitations, so a lower score is better in terms of respiratory function.

There are no subscales. Below we are providing mean scores for all 9 participants in this trial.

Outcome measures

Measure

Celecoxib n=9 Participants Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.

St. George's Respiratory Questionnaire

20.2 score on a scale Standard Deviation 4.9

SECONDARY outcome

Timeframe: 6 months

Population: those who stayed on study until the end

VEGF-D serum levels

Outcome measures

Measure

Celecoxib n=9 Participants Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.

VEGF-D Serum Levels

656 pg/mL Interval 279.0 to 1865.0

SECONDARY outcome

Timeframe: 1 year

We had intended to perform Exhaled breath condensate prostaglandin metabolites. However, this proved to be impossible, and no data was obtained.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: all participants

We had planned to determine a circulating LAM cell count. However, this proved to be impossible. Therefore, no data was collected.

Outcome measures

Outcome data not reported

Adverse Events

Celecoxib

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Celecoxib n=12 participants at risk Celecoxib 200mg PO QD for 6 months Celecoxib: We will perform a pilot clinical trial to investigate the safety and tolerability of celecoxib therapy as a single agent for patients with LAM. LAM subjects who are not taking everolimus or rapamycin will be treated with celecoxib at 200mg PO QD for 6 months. They will be monitored for respiratory function and angiomyolipoma size. At the end of the 6 month period, celecoxib will be discontinued, and subjects will be monitored for another 6 months.
Gastrointestinal disorders abdominal pain	25.0% 3/12 • Number of events 6 • 1 year same as that used by clinicaltrials.gov
Nervous system disorders headache	25.0% 3/12 • Number of events 6 • 1 year same as that used by clinicaltrials.gov
General disorders fatigue	25.0% 3/12 • Number of events 3 • 1 year same as that used by clinicaltrials.gov
General disorders malaise	16.7% 2/12 • Number of events 2 • 1 year same as that used by clinicaltrials.gov

Additional Information

Dr. David Kwiatkowski

Brigham and Women's Hospital

Phone: 8573070781

Email: dk@rics.bwh.harvard.edu

Results disclosure agreements

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