Trial · NCT04141670
S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
NCT04141670, Phase 1, Completed, RyCarma Therapeutics, Inc.; results first posted 2024-04-19
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM). Trials, record NCT04141670. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04141670
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
- NCT number
- NCT04141670
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 7 participants, actual
- Lead sponsor
- RyCarma Therapeutics, Inc.
- Conditions
- RYR-1 Myopathy
- Interventions
- S48168
- First posted
- 2019-10-28
- Last updated
- 2024-08-22
- Results first posted
- 2024-04-19
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | 42 days | Composite safety and tolerability profile of S48168 (ARM210) based on adverse event reporting |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | 42 days | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | Low Dose Group | Participants with fatal adverse events (all cause mortality) | 0 | Participants | |
| Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | High Dose Group | Participants with fatal adverse events (all cause mortality) | 0 | Participants | |
| Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | Low Dose Group | Participants with serious adverse events | 0 | Participants | |
| Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | High Dose Group | Participants with serious adverse events | 0 | Participants | |
| Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | Low Dose Group | Participants with non-serious adverse events | 3 | Participants | |
| Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) | High Dose Group | Participants with non-serious adverse events | 3 | Participants |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Low Dose Group | 3 | |
| Overall Study | STARTED | High Dose Group | 4 | |
| Overall Study | COMPLETED | Low Dose Group | 3 | |
| Overall Study | COMPLETED | High Dose Group | 4 | |
| Overall Study | NOT COMPLETED | Low Dose Group | 0 | |
| Overall Study | NOT COMPLETED | High Dose Group | 0 |
Source ClinicalTrials.gov
Adverse events36
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Eosinophilia | Blood and lymphatic system disorders | Low Dose Group | 1 | 3 | 1 |
| other | Eosinophilia | Blood and lymphatic system disorders | High Dose Group | 0 | 4 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Low Dose Group | 1 | 3 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | High Dose Group | 0 | 4 | 0 |
| other | Dyspepsia | Gastrointestinal disorders | Low Dose Group | 1 | 3 | 2 |
| other | Dyspepsia | Gastrointestinal disorders | High Dose Group | 0 | 4 | 0 |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Low Dose Group | 1 | 3 | 1 |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | High Dose Group | 0 | 4 | 0 |
| other | Vomiting | Gastrointestinal disorders | Low Dose Group | 0 | 3 | 0 |
| other | Vomiting | Gastrointestinal disorders | High Dose Group | 1 | 4 | 1 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Low Dose Group | 0 | 3 |
| Total, all-cause mortality | High Dose Group | 0 | 4 |
| Total, other adverse events | Low Dose Group | 3 | 3 |
| Total, other adverse events | High Dose Group | 3 | 4 |
| Total, serious adverse events | Low Dose Group | 0 | 3 |
| Total, serious adverse events | High Dose Group | 0 | 4 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38318125 | DERIVED |
Source PubMed
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| ZIANS003129 | Molecular and Clinical Manifestations of Matrix and Aggregate Myopathies | BÖNNEMANN, CARSTEN | NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE | NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE | 2011 to 2025 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Low Dose Group vs High Dose Group: deaths from any cause | Trial comparisons |
| Low Dose Group vs High Dose Group: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04141670
GET https://conductscience.org/api/trials/v1/NCT04141670/events?page=2