Trial Outcomes & Findings for Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy (NCT NCT01855360)

NCT ID: NCT01855360

Last Updated: 2020-12-04

Results Overview

Outcome measure, namely changes in longitudinal echocardiographic strain, will be compared to previously determined changes derived from a cohort of patients with TTR cardiac amyloidosis who were not receiving specific therapy for amyloid deposition.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 40 participants
• Primary outcome timeframe: Time Frame: * (FDAAA) outcome measure is assessed every 6 months by serial echocardiography, with final measurement 18 months after enrollment, change at 12 months reported as pre-defined primary endpoint.
• Results posted on: 2020-12-04

Participant Flow

Participant milestones

Measure	TUDCA and Doxycycline INTERVENTION: Patients meeting study criteria were prescribed TUDCA taken orally, 250 mg three times daily. and doxycycline taken orally, 100 mg twice daily. Tauroursodeoxycholic Acid and Doxycycline
Overall Study STARTED	40
Overall Study COMPLETED	30
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Number analyzed is number of patients completing 12 month visit and who had a technically analyzable echocardiogram.

Baseline characteristics by cohort

Measure	TUDCA and Doxycycline n=40 Participants INTERVENTION: Patients meeting study criteria were prescribed TUDCA taken orally, 250 mg three times daily. and doxycycline taken orally, 100 mg twice daily. Tauroursodeoxycholic Acid and Doxycycline
Age, Categorical <=18 years	0 Participants n=40 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=40 Participants
Age, Categorical >=65 years	40 Participants n=40 Participants
Age, Continuous	71.3 years STANDARD_DEVIATION 3.5 • n=30 Participants • Number analyzed is number of patients completing 12 month visit and who had a technically analyzable echocardiogram.
Sex: Female, Male Female	1 Participants n=30 Participants • Number analyzed is number of patients completing 12 month visit and who had a technically analyzable echocardiogram (this was the primary endpoint).
Sex: Female, Male Male	29 Participants n=30 Participants • Number analyzed is number of patients completing 12 month visit and who had a technically analyzable echocardiogram (this was the primary endpoint).
Region of Enrollment United States	30 Participants n=30 Participants • Number analyzed is number of patients completing 12 month visit and who had a technically analyzable echocardiogram.

PRIMARY outcome

Timeframe: Time Frame: * (FDAAA) outcome measure is assessed every 6 months by serial echocardiography, with final measurement 18 months after enrollment, change at 12 months reported as pre-defined primary endpoint.

Population: Patients who completed 18 month visit (primary outcome) and had a technically adequate echocardiogram to analyze strain imaging

Outcome measure, namely changes in longitudinal echocardiographic strain, will be compared to previously determined changes derived from a cohort of patients with TTR cardiac amyloidosis who were not receiving specific therapy for amyloid deposition.

Outcome measures

Measure	TUDCA and Doxycycline n=30 Participants INTERVENTION: Patients meeting study criteria were prescribed TUDCA taken orally, 250 mg three times daily. and doxycycline taken orally, 100 mg twice daily. Tauroursodeoxycholic Acid and Doxycycline
Changes in Strain Echocardiography	9.1 percent LV shortening Standard Deviation .9

SECONDARY outcome

Timeframe: 18 months

Outcome measures

Measure	TUDCA and Doxycycline n=40 Participants INTERVENTION: Patients meeting study criteria were prescribed TUDCA taken orally, 250 mg three times daily. and doxycycline taken orally, 100 mg twice daily. Tauroursodeoxycholic Acid and Doxycycline
Number of Patients With Adverse Events	7 Participants

Adverse Events

TUDCA and Doxycycline

Serious events: 1 serious events

Other events: 6 other events

Deaths: 6 deaths

Serious adverse events

Measure	TUDCA and Doxycycline n=40 participants at risk INTERVENTION: Patients meeting study criteria were prescribed TUDCA taken orally, 250 mg three times daily. and doxycycline taken orally, 100 mg twice daily. Tauroursodeoxycholic Acid and Doxycycline
Renal and urinary disorders acute kidney injury	2.5% 1/40 • Number of events 1 • Adverse events collected over the course of 18 months.

Other adverse events

Measure	TUDCA and Doxycycline n=40 participants at risk INTERVENTION: Patients meeting study criteria were prescribed TUDCA taken orally, 250 mg three times daily. and doxycycline taken orally, 100 mg twice daily. Tauroursodeoxycholic Acid and Doxycycline
Gastrointestinal disorders gastrointestinal upset	15.0% 6/40 • Number of events 6 • Adverse events collected over the course of 18 months.

Additional Information

Rodney H Falk

Brigham and WQomen's Hospital

Phone: 6175257053

Email: rfalk@bwh.harvard.edu

Results disclosure agreements

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