Trial · NCT06843811
Sirolimus for Leigh Syndrome
NCT06843811, Phase 2, Enrolling by invitation, Matthew Demczko
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Sirolimus for Leigh Syndrome. Trials, record NCT06843811. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT06843811
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Sirolimus for Leigh Syndrome
- NCT number
- NCT06843811
- Status
- Enrolling by invitation
- Phases
- Phase 2
- Enrollment
- 15 participants, estimated
- Lead sponsor
- Matthew Demczko
- Conditions
- Leigh Syndrome
- Interventions
- Sirolimus
- First posted
- 2025-02-25
- Last updated
- 2026-03-31
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate of Adverse Events | Up to 2.5 years | Adverse events will be measured according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE). Incidence of AEs will be reported. |
| Primary | Rate of Intercurrent Infection and Hospitalization | Up to 2.5 years | Each infection and hospitalization will be reported. |
| Primary | Incidence of abnormal safety lab values | Up to 2.5 years | Count of clinically significant changes from baseline in safety labs |
| Primary | Sirolimus Trough Level | Up to 2.5 years | Measure of Sirolimus level in the blood. Sirolimus dosage will be adjusted as needed in order to maintain a sirolimus trough level within a range of 5 to 10 ng/mL. The number of out-of-range results will be reported. |
| Secondary | Change in MM-COAST Score | Baseline up to end of study (up to 2.5 years) | Mitochondrial Myopathy-Composite Assessment Tool (MM-COAST) is a composite assessment tool used to measure the domains of muscle strength, muscle fatigue, balance, dexterity, and exercise intolerance. Scores are assigned for each domain assessment, based on z-score or % decrement for muscle fatigue, summed and averaged to achieve a domain score. The mean domain score is reported as the MM-COAST Composite Score. |
| Secondary | Change in GMFM (Gross Motor Function Measure) Score | Baseline up to end of study (up to 2.5 years) | The GMFM is a clinical tool used assess changes in gross motor functions in 5 dimensions (1. lying and rolling, 2. sitting, 3. crawling and kneeling, 4. standing, and 5. walking, running, and jumping). Each task is scored as 0-does not initiate, 1-initiates, 2-partially completes, and 3-completed. Item scores are summed to calculate raw and percent scores for each of the five dimensions. Dimension percent scores are averaged to obtain an overall total score. |
| Secondary | Change in Movement Disorder-Childhood Rating Score (MDCRS) | Baseline up to end of study (up to 2.5 years) | The MDCRS is a scale used to evaluate the impact of movement disorder on patient motor function and daily living activities. Severity of movement disorder in different regions of the body at rest, and during specific tasks, is ranked from 0 (no movement disorder) to 4 (severe movement disorder). Item scores are summed to calculate total score, with a higher total score indicating greater overall impact of movement disorder on daily functioning. |
| Secondary | CGI Scale | Baseline up to end of study (up to 2.5 years) | The CGI (Clinical Global Impression Scale) is a 3-item clinician-rated scale used to assess severity of illness, global improvement, and therapeutic response. Degree of global improvement for participants receiving sirolimus will be assessed by the investigator and rated on a scale 7-point scale based on improvement from baseline from 1 (very much improved) to 7 (very much worse). A lower score indicates greater response to treatment. |
| Secondary | Change in Barry-Albright Dystonia Scale (BADS) | Baseline up to end of study (up to 2.5 years) | The Barry-Albright Dystonia Scale is used to assess dystonia in 8 bodily regions: eyes, mouth, neck, trunk, and each upper and lower extremity. Each bodily region is scored based on a scale from 0 (absent) to 4 (severe symptoms). Total BADS score ranges from 0 (dystonia absent) to 32 (severe dystonia). |
| Secondary | Change in Scale for the Assessment and Rating of Ataxia (SARA) | Baseline up to end of study (up to 2.5 years) | The SARA is an 8-item performance-based scale that assesses ataxia. Patients are evaluated based on their ability to perform a series of tasks with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia). |
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.
Papers4
The papers the trial lists on ClinicalTrials.gov.
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/NCT06843811
GET https://conductscience.org/api/trials/v1/NCT06843811/registered