Trial Outcomes & Findings for Fibroblast Growth Factor Regeneration of Tympanic Membrane Perforations (NCT NCT02307916)

NCT ID: NCT02307916

Last Updated: 2020-12-31

Results Overview

The efficacy of the treatment is determined by the number of patients whose tympanic membrane perforations closed after study treatments. Subjects from both treatment arms received up to three treatments before determining non-closure of the tympanic membrane.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 57 participants
• Primary outcome timeframe: ~60 days
• Results posted on: 2020-12-31

Participant Flow

Patients who met exclusion criteria were considered "screen-fail" and were not assigned to a randomization group.

Participant milestones

Measure	FGF-2 FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.
Overall Study STARTED	42	15
Overall Study COMPLETED	40	14
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	FGF-2 n=42 Participants FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo n=15 Participants Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.	Total n=57 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=42 Participants	0 Participants n=15 Participants	0 Participants n=57 Participants
Age, Categorical Between 18 and 65 years	32 Participants n=42 Participants	10 Participants n=15 Participants	42 Participants n=57 Participants
Age, Categorical >=65 years	10 Participants n=42 Participants	5 Participants n=15 Participants	15 Participants n=57 Participants
Age, Continuous	51.7 years STANDARD_DEVIATION 13.9 • n=42 Participants	55.8 years STANDARD_DEVIATION 15 • n=15 Participants	52.7 years STANDARD_DEVIATION 14.2 • n=57 Participants
Sex: Female, Male Female	23 Participants n=42 Participants	8 Participants n=15 Participants	31 Participants n=57 Participants
Sex: Female, Male Male	19 Participants n=42 Participants	7 Participants n=15 Participants	26 Participants n=57 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	42 participants n=42 Participants	15 participants n=15 Participants	57 participants n=57 Participants

PRIMARY outcome

Timeframe: ~60 days

The efficacy of the treatment is determined by the number of patients whose tympanic membrane perforations closed after study treatments. Subjects from both treatment arms received up to three treatments before determining non-closure of the tympanic membrane.

Outcome measures

Measure	FGF-2 n=40 Participants FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo n=14 Participants Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.
Number of Patients With Tympanic Membrane Closure Following Treatment	23 Participants	10 Participants

SECONDARY outcome

Timeframe: Baseline, 60 days

measured by pre- and post-treatment audiograms.

Outcome measures

Measure	FGF-2 n=40 Participants FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo n=14 Participants Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.
Measurement of Changes in Pure-tone Averages	7.9 dB Standard Deviation 8.8	5.9 dB Standard Deviation 9.1

SECONDARY outcome

Timeframe: Baseline, 60 days

Speech discrimination score is determined by having the patient listen to words through headphones and having the patient repeat the words back to the audiologist. A score of 100 percent (100%) is the best outcome, and 0 percent (0%) is the worst. Pre- and post- treatment tests are compared and analyzed.

Outcome measures

Measure	FGF-2 n=40 Participants FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo n=14 Participants Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.
Measurement of Changes in Speech Discrimination Scores	3.2 percent Standard Deviation 8.1	1.7 percent Standard Deviation 4.7

SECONDARY outcome

Timeframe: 60 days

Out of the patients who healed, some healed with 1 treatment, some with 2 treatments, and some with 3 treatments.

Outcome measures

Measure	FGF-2 n=40 Participants FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo n=14 Participants Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.
Number of Treatments Required for Closure of the Tympanic Membrane Perforation	1.4 treatments Standard Deviation 0.6	1.7 treatments Standard Deviation 0.6

SECONDARY outcome

Timeframe: 60 days

Tympanometry is an examination used to test the condition of the middle ear and the mobility of the eardrum. Any type of perforation, regardless of size, would cause the result of the tympanometry to be abnormal. We use this test to determine if the eardrum closed completely.

Outcome measures

Measure	FGF-2 n=40 Participants FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo n=14 Participants Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.
Number of Patients With Normal Mobility of the Eardrum as Assessed by Tympanometry	23 Participants	10 Participants

Adverse Events

FGF-2

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	FGF-2 n=42 participants at risk FGF-2 given a minimum of 1 and maximum of 3 times within, approximately, 60 days. FGF-2: The FGF-2 method of use comprises a pledget of gelatin foam with human fibroblast growth factor-2 (FGF-2) added to the pledget just before topical application to the tympanic membrane, and the use of Tisseel, a fibrin glue used for applications in ear surgery.	Placebo n=15 participants at risk Equal concentration of saline is given a minimum of 1 and maximum of 3 times within, approximately, 60 days. Placebo: Placebo comparator using a sterile saline solution.
Ear and labyrinth disorders Ear drainage post-treatment	11.9% 5/42 • Number of events 9 • Adverse event data were collected from the time of enrollment to the final follow-up visit, usually within 6 months.	13.3% 2/15 • Number of events 2 • Adverse event data were collected from the time of enrollment to the final follow-up visit, usually within 6 months.
Ear and labyrinth disorders Ear pain during procedure	7.1% 3/42 • Number of events 3 • Adverse event data were collected from the time of enrollment to the final follow-up visit, usually within 6 months.	13.3% 2/15 • Number of events 4 • Adverse event data were collected from the time of enrollment to the final follow-up visit, usually within 6 months.
Ear and labyrinth disorders Ear infection	11.9% 5/42 • Number of events 7 • Adverse event data were collected from the time of enrollment to the final follow-up visit, usually within 6 months.	6.7% 1/15 • Number of events 2 • Adverse event data were collected from the time of enrollment to the final follow-up visit, usually within 6 months.

Additional Information

Dr. Brad Welling

Massachusetts Eye and Ear

Phone: 617-573-4075

Email: brad_welling@meei.harvard.edu

Results disclosure agreements

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