Trial Outcomes & Findings for Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis (NCT NCT00877604)

Results Overview

Responder patients were defined as those subjects showing an improvement of at least 15% in the ALSFRS-R slope during the treatment period as compared to the lead-in period.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

34 participants

Primary outcome timeframe

1 year

Results posted on

2014-11-26

Participant Flow

Recruitment is started on eightth of october 2009, in medical clinics (Besta Institute in Milan, University II department of Neurology of Naples, University of Palermo department of Neurology).

34 patients enrolled, 5 of whom dropped out during the lead in phase.

Participant milestones

Participant milestones
Measure	TUDCA tauroursodeoxycholic acid di-hydrate tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year	Placebo excipient lactose Placebo: identical placebo by oral route at the same dosing schedule composed of excipient lactose
Overall Study STARTED	17	17
Overall Study COMPLETED	15	14
Overall Study NOT COMPLETED	2	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	TUDCA n=17 Participants tauroursodeoxycholic acid di-hydrate tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year	Placebo n=17 Participants excipient lactose Placebo: identical placebo by oral route at the same dosing schedule	Total n=34 Participants Total of all reporting groups
Age, Continuous	54.0 years STANDARD_DEVIATION 12.2 • n=5 Participants	58.2 years STANDARD_DEVIATION 12.9 • n=7 Participants	56.1 years STANDARD_DEVIATION 12.3 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	10 Participants n=7 Participants	21 Participants n=5 Participants
Region of Enrollment Italy	17 participants n=5 Participants	17 participants n=7 Participants	34 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Responder patients were defined as those subjects showing an improvement of at least 15% in the ALSFRS-R slope during the treatment period as compared to the lead-in period.

Outcome measures

Outcome measures
Measure	TUDCA n=15 Participants tauroursodeoxycholic acid di-hydrate tauroursodeoxycholic acid (TUDCA): Oral route at the dose of 1 g b.i.d. (2 g daily) for 1 year	Placebo n=14 Participants excipient lactose Placebo: identical placebo by oral route at the same dosing schedule composed of excipient lactose
The Proportion of Responder Patients in the Two Treatment Groups According the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS)-R Slope.	13 participants	6 participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

laboratory tests, patients' reports and the investigator's judgments

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

Adverse Events

TUDCA

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

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

Alberto Albanese

Fondazione Istituto Neurologico Carlo Besta

Phone: +39 02 2392 4552

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place