Trial Outcomes & Findings for S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM) (NCT NCT04141670)
NCT ID: NCT04141670
Last Updated: 2024-08-22
Results Overview
Composite safety and tolerability profile of S48168 (ARM210) based on adverse event reporting
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
7 participants
Primary outcome timeframe
42 days
Results posted on
2024-08-22
Participant Flow
Participant milestones
| Measure |
Low Dose Group
Experimental:120 mg S48168 (ARM210; 6 x 20 mg tablets) daily for 29 days
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
High Dose Group
Experimental: 200 mg S48168 (ARM210; 10 x 20 mg tablets) daily for 28 days (1 participant) or 29 days (3 participants)
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
4
|
|
Overall Study
COMPLETED
|
3
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
Baseline characteristics by cohort
| Measure |
Low Dose Group
n=3 Participants
Experimental: 120 mg S48168 (ARM210; 6 x 20 mg tablets) daily for 29 days
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
High Dose Group
n=4 Participants
Experimental: 200 mg S48168 (ARM210; 10 x 20 mg tablets) daily for 28 days (1 participant) or 29 days (3 participants)
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
Total
n=7 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Body mass index
|
24 kg/m^2
STANDARD_DEVIATION 5 • n=5 Participants
|
31 kg/m^2
STANDARD_DEVIATION 5 • n=7 Participants
|
28 kg/m^2
STANDARD_DEVIATION 6 • n=5 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 42 daysComposite safety and tolerability profile of S48168 (ARM210) based on adverse event reporting
Outcome measures
| Measure |
Low Dose Group
n=3 Participants
Experimental: 120 mg S48168 (ARM210; 6 x 20 mg tablets) daily for 29 days
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
High Dose Group
n=4 Participants
Experimental: 200 mg S48168 (ARM210; 10 x 20 mg tablets) daily for 28 days (1 participant) or 29 days (3 participants)
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
|---|---|---|
|
Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210)
Participants with fatal adverse events (all cause mortality)
|
0 Participants
|
0 Participants
|
|
Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210)
Participants with serious adverse events
|
0 Participants
|
0 Participants
|
|
Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210)
Participants with non-serious adverse events
|
3 Participants
|
3 Participants
|
Adverse Events
Low Dose Group
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
High Dose Group
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Low Dose Group
n=3 participants at risk
Experimental: 120 mg S48168 (ARM210; 6 x 20 mg tablets) daily for 29 days
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
High Dose Group
n=4 participants at risk
Experimental: 200 mg S48168 (ARM210; 10 x 20 mg tablets) daily for 28 days (1 participant) or 29 days (3 participants)
S48168: A novel oral small molecule which is designed to repair leaky RYR1 channels
|
|---|---|---|
|
Blood and lymphatic system disorders
Eosinophilia
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
1/3 • Number of events 2 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Gastrointestinal disorders
Dyspepsia
|
33.3%
1/3 • Number of events 2 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/3 • Approximately two months
|
25.0%
1/4 • Number of events 1 • Approximately two months
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/3 • Approximately two months
|
25.0%
1/4 • Number of events 1 • Approximately two months
|
|
Injury, poisoning and procedural complications
Skin laceration
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Investigations
Blood creatine phosphokinase increased
|
33.3%
1/3 • Number of events 2 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
25.0%
1/4 • Number of events 1 • Approximately two months
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
25.0%
1/4 • Number of events 1 • Approximately two months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
25.0%
1/4 • Number of events 2 • Approximately two months
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • Approximately two months
|
50.0%
2/4 • Number of events 2 • Approximately two months
|
|
Nervous system disorders
Sinus headache
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Renal and urinary disorders
Haematuria
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Skin and subcutaneous tissue disorders
Mechanical utricaria
|
0.00%
0/3 • Approximately two months
|
25.0%
1/4 • Number of events 1 • Approximately two months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
|
Vascular disorders
Hypertension
|
33.3%
1/3 • Number of events 1 • Approximately two months
|
0.00%
0/4 • Approximately two months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place