Trial Outcomes & Findings for Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy (NCT NCT02858908)

NCT ID: NCT02858908

Last Updated: 2025-09-11

Results Overview

Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

12 weeks

Results posted on

2025-09-11

Participant Flow

Subjects were to be male or female aged 12 to 45 years with a diagnosis of genetically confirmed congenital or juvenile-onset type 1 myotonic dystrophy. Subjects were to have a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2) and were to be ambulatory and able to complete the 10-metre walk/run test.

Participant milestones

Participant milestones
Measure	Cohort 1 Subjects were given tideglusib 1000 mg for 12 weeks (weeks 0 to 12)	Cohort 2 Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)
Overall Study STARTED	8	8
Overall Study COMPLETED	8	8
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 n=8 Participants 1000 mg tideglusib	Cohort 2 n=8 Participants 400 mg tideglusib	Total n=16 Participants Total of all reporting groups
Age, Customized Mean	21.8 years STANDARD_DEVIATION 5.5 • n=5 Participants	20.3 years STANDARD_DEVIATION 6.36 • n=7 Participants	21 years STANDARD_DEVIATION 5.8 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	1 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	7 Participants n=7 Participants	10 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or latino	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Race/Ethnicity, Customized White	8 Participants n=5 Participants	7 Participants n=7 Participants	15 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Safety Analysis Set

Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Safety (Adverse Events) Total number of TEAEs	6 Participants	8 Participants
Safety (Adverse Events) Total number of serious TEAEs	0 Participants	0 Participants
Safety (Adverse Events) Total number of TEAEs leading to discontinuation of active treatment	0 Participants	0 Participants
Safety (Adverse Events) Total number of TEAEs leading to withdrawal	0 Participants	0 Participants
Safety (Adverse Events) Total number of TEAEs leading to death	0 Participants	0 Participants
Safety (Adverse Events) Severity: Mild	1 Participants	3 Participants
Safety (Adverse Events) Severity: Moderate	4 Participants	5 Participants
Safety (Adverse Events) Severity: Severe	1 Participants	0 Participants
Safety (Adverse Events) Unrelated to active treatment	6 Participants	6 Participants
Safety (Adverse Events) Related to active treatment	0 Participants	2 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Pharmacokinetic samples were collected to determine Tideglusib plasma concentration

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Plasma Concentration of Tideglusib Maximum plasma concentration (Cmax)	496.94 ng/mL Standard Deviation 104.88	1041.25 ng/mL Standard Deviation 361.44
Plasma Concentration of Tideglusib Minimum plasma concentration (Cmin)	5.14 ng/mL Standard Deviation 4.06	8.63 ng/mL Standard Deviation 5.68
Plasma Concentration of Tideglusib Steady-state concentration (CSS)	54.27 ng/mL Standard Deviation 16.54	133.7 ng/mL Standard Deviation 60.79

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Pharmacokinetic samples were collected to determine time of the maximum plasma concentration and terminal elimination half-life of tideglusib

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Blood Pharmacokinetics of Tideglusib Time of the maximum plasma concentration (Tmax)	0.65 h Standard Deviation 0.29	0.79 h Standard Deviation 0.27
Blood Pharmacokinetics of Tideglusib Terminal elimination half-life (T1/2)	2.68 h Standard Deviation 1.70	1.82 h Standard Deviation 0.64

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Pharmacokinetic samples were collected to determine area under the plasma concentration vs. time curve from 0 to 12 h and area under the plasma concentration vs. time curve from 0 to 24 h of tideglusib

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Area Under the Plasma Concentration vs. Time Curve of Tideglusib Area under the plasma concentration vs. time curve from 0 to 12 h (AUC0-12)	1214.88 ng/mL.h Standard Deviation 390.81	3054.27 ng/mL.h Standard Deviation 1418.30
Area Under the Plasma Concentration vs. Time Curve of Tideglusib Area under the plasma concentration vs. time curve from 0 to 24 h (AUC0-24)	1302.42 ng/mL.h Standard Deviation 396.96	3208.8 ng/mL.h Standard Deviation 1459.03

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The 10-metre walk/run test is a performance measure used to assess walking speed in metres per second over a short distance and was used as an assessment of functional mobility. Time taken to complete the 10m walk/run test at fastest and preferred speed is measured.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
10 Metre Walk/Run Test Preferred speed (observed value at week 12)	9.1475 seconds Standard Deviation 1.72651	9.1041 seconds Standard Deviation 1.34351
10 Metre Walk/Run Test Preferred speed (change from baseline at week 12)	-0.6529 seconds Standard Deviation 0.86109	-0.6616 seconds Standard Deviation 1.96370
10 Metre Walk/Run Test Fastest speed (observed value at week 12)	4.4833 seconds Standard Deviation 2.72809	4.2515 seconds Standard Deviation 2.41439
10 Metre Walk/Run Test Fastest speed (change from baseline at week 12)	-0.0650 seconds Standard Deviation 0.46428	-0.3894 seconds Standard Deviation 0.32806

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Handgrip myometry is used as a measure of myotonia and muscle strength for the dominant hand

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time Observed value (at week 12)	14.1334 kg Standard Deviation 6.86499	14.0899 kg Standard Deviation 7.71583
Computerised Handgrip Myometer Measure of Grip Strength and Muscle Relaxation Time Change from baseline (at week 12)	-1.4893 kg Standard Deviation 2.89328	1.0098 kg Standard Deviation 3.09422

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

FVC is a measure of lung function (the total amount of air exhaled during a Forced Expiratory Volume is measured using a spirometer)

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Respiratory Forced Vital Capacity (FVC) Observed value (at week 12)	2.4220 litres Standard Deviation 1.15969	2.1509 litres Standard Deviation 0.93362
Respiratory Forced Vital Capacity (FVC) Change from baseline (at week 12)	0.0118 litres Standard Deviation 0.36746	-0.0179 litres Standard Deviation 0.32603

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

DXA utilises two low energy X-ray beams, with different energy levels, which are aimed at the subject's bones. The DXA scan is more typically used to measure bone mineral density, however it can also be used to measure total lean muscle mass

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Dual-energy X-ray Absorptiometry (DXA) Arms (observed value at week 12)	4098.5 gram Standard Deviation 1313.53	3102.9 gram Standard Deviation 898.69
Dual-energy X-ray Absorptiometry (DXA) Arms (change from baseline at week 12)	35.9 gram Standard Deviation 357.09	-252.8 gram Standard Deviation 1038.49
Dual-energy X-ray Absorptiometry (DXA) Legs (observed value at week 12)	12262.8 gram Standard Deviation 3225.63	11767.0 gram Standard Deviation 2980.78
Dual-energy X-ray Absorptiometry (DXA) Legs (change from baseline at week 12)	95.0 gram Standard Deviation 299.82	-62.5 gram Standard Deviation 646.22
Dual-energy X-ray Absorptiometry (DXA) Total (observed value at week 12)	37740.4 gram Standard Deviation 8481.27	38267.6 gram Standard Deviation 8274.58
Dual-energy X-ray Absorptiometry (DXA) Total (change from baseline at week 12)	391.9 gram Standard Deviation 763.01	411.6 gram Standard Deviation 2040.84

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on severity of illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. Change in CGI-S was observed in only 1 subject. Consequently statistical analysis was not conducted.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Clinical Global Impressions- Severity (CGI-S) Observed value at week 12	4.9 Values on a scale Standard Deviation 0.83	4.8 Values on a scale Standard Deviation 1.16
Clinical Global Impressions- Severity (CGI-S) Change from baseline at week 12	0.0 Values on a scale Standard Deviation 0.00	-0.1 Values on a scale Standard Deviation 0.35

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The CGI-I requires the clinician to rate how much the subject's illness has improved or worsened relative to a baseline state. A seven point Likert type scale is used from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Clinical Global Impressions- Improvement (CGI-I)	3.0 Scores on a scale Standard Deviation 1.07	3.1 Scores on a scale Standard Deviation 0.83

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Actigraphy is a non-invasive method of monitoring physical activity via an actigraph device. The actigraph device was worn on the waist during waking hours, according to the device's instruction manual.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Actigraphy (3-minute Bouts of Activity) Weekly total number of 3 minute bouts of activity (observed value at week 12)	0.938 3 minute bouts of activity per hour Standard Deviation 0.5268	1.465 3 minute bouts of activity per hour Standard Deviation 0.1780
Actigraphy (3-minute Bouts of Activity) Weekly total number of 3 minute bouts of activity (change from baseline at week 12)	-0.284 3 minute bouts of activity per hour Standard Deviation 0.1805	-0.200 3 minute bouts of activity per hour Standard Deviation 0.3959

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Actigraphy (>10-minute Bouts of Activity) Weekly total number of >10 minute bouts of activity (observed value at week 12)	0.020 10 minute bouts of activity per hour Standard Deviation 0.0245	0.048 10 minute bouts of activity per hour Standard Deviation 0.0621
Actigraphy (>10-minute Bouts of Activity) Weekly total number of >10 minute bouts of activity (change from baseline at week 12)	0.006 10 minute bouts of activity per hour Standard Deviation 0.0313	-0.010 10 minute bouts of activity per hour Standard Deviation 0.0600

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Actigraphy (Steps) Weekly total number of steps (observed value at week 12)	352.518 steps per hour wear time Standard Deviation 263.1992	556.387 steps per hour wear time Standard Deviation 255.6433
Actigraphy (Steps) Weekly total number of steps (change from baseline at week 12)	-53.802 steps per hour wear time Standard Deviation 89.0843	-51.427 steps per hour wear time Standard Deviation 198.9754

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Measure of fine manual dexterity. Time to taken to complete the NHPT (dominant hand) is recorded.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Nine Hole Peg Test (NHPT) Observed value at week 12	24.760 seconds Standard Deviation 3.8275	23.590 seconds Standard Deviation 8.2592
Nine Hole Peg Test (NHPT) Change from baseline at week 12	-0.950 seconds Standard Deviation 1.7080	-2.168 seconds Standard Deviation 3.9007

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study. Subjects, where possible, and caregivers were asked to rate three causes for concern by drawing a vertical mark on a 10 cm long visual analogue scale with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). A score for each concern was to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 3 concerns (minimum = 0 cm, maximum = 30 cm). A higher score represents a worse outcome.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Top 3 Concerns Visual Analogue Scale (VAS) Score Subject Top 3 Concerns VAS total score (observed value at week 12)	15.29 Score on a scale Standard Deviation 5.641	15.66 Score on a scale Standard Deviation 6.331
Top 3 Concerns Visual Analogue Scale (VAS) Score Subject Top 3 Concerns VAS total score (change from baseline at week 12)	-2.00 Score on a scale Standard Deviation 2.598	-1.76 Score on a scale Standard Deviation 3.455
Top 3 Concerns Visual Analogue Scale (VAS) Score Caregiver Top 3 Concerns VAS total score (observed value at week 12)	16.58 Score on a scale Standard Deviation 6.151	18.84 Score on a scale Standard Deviation 3.583
Top 3 Concerns Visual Analogue Scale (VAS) Score Caregiver Top 3 Concerns VAS total score (change from baseline at week 12)	-1.98 Score on a scale Standard Deviation 2.001	-2.39 Score on a scale Standard Deviation 2.669

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The OARS-4 contains the autism signs and symptoms in the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition. These were to be rated with the degree of impairment the subject experiences for the given symptom. The symptoms were to be elicited in a semi-structured interview with the subject's primary caregiver. The assessor was to take both frequency/duration and degree of impairment into account and how much the item interferes with relationships, learning, and/or activities of daily living. The scores for this assessment were from 0 (Never or Rarely; Not a Problem) to 3 (Very Often; A Severe Problem): a score for each symptom of social impairment, communication impairment and restricted patterns were averaged to provide a total impairment score. A higher score represents a worse outcome (minimum = 0, maximum 3).

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Ohio State University (OSU) Autism Rating Scale (OARS) Total impairment mean (change from baseline at week 12)	-0.010 Units on a scale Standard Deviation 0.0283	-0.054 Units on a scale Standard Deviation 0.0782
Ohio State University (OSU) Autism Rating Scale (OARS) Total impairment mean (observed value at week 12)	0.303 Units on a scale Standard Deviation 0.4946	0.239 Units on a scale Standard Deviation 0.3178

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The OSU Autism CGI scale contains separate subscales for symptom severity and for global improvement. These are rated in a similar way to the National Institute of Mental Health (NIMH) CGI Severity scale, but it is focused on autism spectrum symptoms. OSU Autism CGI-severity and OSU Autism CGI-improvement scores use a seven point Likert rating scale from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Ohio State University (OSU) Autism Clinical Global Impression (CGI) OSU autism CGI-Severity (observed value at week 12)	2.3 Units on a scale Standard Deviation 1.83	1.9 Units on a scale Standard Deviation 0.99
Ohio State University (OSU) Autism Clinical Global Impression (CGI) OSU autism CGI-Severity (change from baseline at week 12)	0 Units on a scale Standard Deviation 0	-0.1 Units on a scale Standard Deviation 0.35
Ohio State University (OSU) Autism Clinical Global Impression (CGI) OSU autism CGI-Improvement (observed value at week 12)	4 Units on a scale Standard Deviation 0	3.4 Units on a scale Standard Deviation 0.74

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The Clinician-completed Domain Specific Causes for Concern is a Visual Analogue Scale completed by the clinician that scores the severity of concerns of domains that are clinically relevant in myotonic dystrophy. The severity of the clinician's concern is scored by using a 10 cm visual analogue scale (VAS), with anchors of "not at all severe" at the left end (0 cm) and "very severe" at the right end (10 cm). The clinician is asked to make a vertical line indicating his/her level of concern in each domain, using a time frame of the past week for reference. A score is to be determined by measuring the number of centimeters on the 10 cm VAS line from the anchor point on the left side of the line. A total VAS score for each subject was calculated as the sum of the scores for the 17 domains (minimum = 0, maximum = 170 cm). A higher score represents a worse outcome.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Clinician-completed Domain Specific Cause for Concern Visual Analogue Scale (VAS): Myotonic Dystrophy VAS total score (observed value at week 12)	59.28 Score on a scale Standard Deviation 17.450	51.19 Score on a scale Standard Deviation 8.798
Clinician-completed Domain Specific Cause for Concern Visual Analogue Scale (VAS): Myotonic Dystrophy VAS total score (change from baseline at week 12)	-3.38 Score on a scale Standard Deviation 2.726	-5.80 Score on a scale Standard Deviation 4.855

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

The PPVT-4 scale is a norm-referenced instrument for measuring the receptive (hearing) vocabulary of children and adults. It contains training items and 228 test items, each consisting of four full-colour pictures as response options on a page. For each item, the examiner says a word, and the examinee responds by selecting the picture that best illustrates that word's meaning. Each administration of the test produces a raw score (number of test items answered correctly), which can be converted to a standard score (using age-based norms) with a mean of 100 and a standard deviation of 15. Higher scores mean a better performance/receptive vocabulary.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Peabody Picture Vocabulary Test (PPVT) Age-based standard score (observed value at week 12)	79.8 Units on a scale Standard Deviation 22.23	63.8 Units on a scale Standard Deviation 15.81
Peabody Picture Vocabulary Test (PPVT) Age-based standard score (change from baseline at week 12)	-1.1 Units on a scale Standard Deviation 5.57	-0.1 Units on a scale Standard Deviation 7.97

SECONDARY outcome

Timeframe: 12 weeks

Population: Full Analysis Set

Total levels of GSK3β protein was determined via x-MAP technology in a Luminex 200 platform using the AKT Pathway Total Multispecies 7-Plex Panel from ThermoFisher Scientific.

Outcome measures

Outcome measures
Measure	Cohort 2 n=8 Participants Subjects were given tideglusib 400 mg for 12 weeks (weeks 0 to 12)	Cohort 1 n=8 Participants 1000 mg tideglusib
Biomarker - Lymphocyte GSK3β Levels and Activity	1.95 microgram per microliter Standard Deviation 0.79	1.42 microgram per microliter Standard Deviation 0.81

Adverse Events

Cohort 1

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Cohort 2

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Dr. Mike Snape, Chief Scientific Officer

AMO Pharma Ltd.

Phone: +44 (0) 7775915639

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The investigator has the right to publish study results from his/her specific site. However, any publication that includes AMO Pharma confidential information cannot be submitted for publication without AMO Pharma's prior written approval.
Publication restrictions are in place

Restriction type: OTHER

Other adverse events
Measure	Cohort 1 n=8 participants at risk 1000 mg tideglusib	Cohort 2 n=8 participants at risk 400 mg tideglusib
Injury, poisoning and procedural complications Back injury	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Injury, poisoning and procedural complications Fall	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Injury, poisoning and procedural complications Joint dislocation	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Injury, poisoning and procedural complications Laceration	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Injury, poisoning and procedural complications Limb injury	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 2 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Respiratory, thoracic and mediastinal disorders Cough	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Nervous system disorders Headache	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Nervous system disorders Nervous system cyst	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Nervous system disorders Somnolence	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Gastrointestinal disorders Vomiting	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	25.0% 2/8 • Number of events 2 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Gastrointestinal disorders Diarrhea	25.0% 2/8 • Number of events 2 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Gastrointestinal disorders Abdominal pain	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Gastrointestinal disorders Post-tussive vomiting	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Reproductive system and breast disorders Dysmenorrhea	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Skin and subcutaneous tissue disorders Eczema	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Skin and subcutaneous tissue disorders Erythema	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Skin and subcutaneous tissue disorders Rash papular	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Musculoskeletal and connective tissue disorders Axillary mass	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Metabolism and nutrition disorders Vitamin D deficiency	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Infections and infestations Nasopharyngitis	25.0% 2/8 • Number of events 2 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	37.5% 3/8 • Number of events 4 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Infections and infestations Lower respiratory tract infection	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Infections and infestations Subcutaneous abscess	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.
Infections and infestations Urinary tract infection	0.00% 0/8 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.	12.5% 1/8 • Number of events 1 • Consent to end of study, up to 14 weeks SAEs were collected from the time of informed consent. Non-serious AEs were collected from the time of single-blind dosing. Any non-serious medical occurrences occurring between Screening (visit 1) and Run in (visit 2) were recorded as Medical/Surgical History. Any non-serious signs and symptoms occurring between Screening (visit 1) and Run in (visit 2) were recorded and classified as baseline signs and symptoms.