Trial · NCT06436742
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
NCT06436742, Phase 1, Active not recruiting, argenx
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS). Trials, record NCT06436742. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT06436742
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
- NCT number
- NCT06436742
- Status
- Active not recruiting
- Phases
- Phase 1
- Enrollment
- 15 participants, actual
- Lead sponsor
- argenx
- Conditions
- Congenital Myasthenic Syndrome, CMS
- Interventions
- ARGX-119, Placebo
- First posted
- 2024-05-31
- Last updated
- 2026-07-24
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Assessment of adverse events (AEs) | Up to week 42 | |
| Primary | Change from active-treatment baseline over time for 6MWT distance | Up to 72 weeks | The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Before and after the 6MWT assessment, the participant's blood pressure, heart rate, and SPO2 will be recorded, and the participant's perception of fatigue and dyspnea will be measured. |
| Secondary | Maximum observed serum concentration (Cmax) of ARGX-119 | Up to 42 weeks + 72 weeks | |
| Secondary | Incidence of ADA against ARGX-119 | Up to 42 weeks + 72 weeks | ADA : anti-drug antibodies |
| Secondary | Change from baseline over time for key components of the QMG scale | Up to 42 weeks + 72 weeks | The Quantitative Myasthenia Gravis (QMG) scale is a standardized quantitative scoring system that was developed to assess disease severity based on impairment of body function and structures in patients with MG. Minimum value: 0 (no disease severity); Maximum value: 39 (highest disease severity). The change from active-treatment baseline will be used for the 72 week timepoint. |
| Secondary | Change from baseline over time for MG-ADL | Up to 42 weeks + 72 weeks | The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item scale that assesses MG symptoms and their effects on daily activities. Minimum value: 0 (normal symptoms); Maximum value: 24 (most severe symptoms). The change from active-treatment baseline will be used for the 72 week timepoint. |
| Secondary | Change from baseline over time for PROMIS-GH scale | Up to 42 weeks | The Patient-Reported Outcomes Measurement Information System Global Health (PROMIS-GH) is a 10-item participant completed quality of life questionnaire that measures global physical health and mental health. The participant records their response to each question on a 5-point Likert scale, with lower scores indicating poorer health (Minimum value: 0, Maximum value: 20) |
| Secondary | Change from active-treatment baseline over time for 6MWT cadence | Up to 72 weeks | The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Before and after the 6MWT assessment, the participant's blood pressure, heart rate, and SPO2 will be recorded, and the participant's perception of fatigue and dyspnea will be measured. |
| Secondary | Change from active-treatment baseline over time for PROMIS PF-WMA-SF | Up to 72 weeks | The PROMIS PF-WMA-SF is an 11-item, participant-completed questionnaire that assesses lower and upper extremity function and associated activities of daily living. The questionnaire asks the participant to rate the items on a 5-point scale of 5 (without any difficulty) to 0 (unable to do). |
| Secondary | Change from active-treatment baseline over time for Neuro-QoL fatigue | Up to 72 weeks |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
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.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT06436742
GET https://conductscience.org/api/trials/v1/NCT06436742/registered