Trial · NCT05650229
Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
NCT05650229, Phase 2, Recruiting, Pharming Technologies B.V.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease. Trials, record NCT05650229. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05650229
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
- NCT number
- NCT05650229
- Status
- Recruiting
- Phases
- Phase 2
- Enrollment
- 180 participants, estimated
- Lead sponsor
- Pharming Technologies B.V.
- Conditions
- Primary Mitochondrial Disease
- Interventions
- KL1333, Placebo
- First posted
- 2022-12-14
- Last updated
- 2026-06-26
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form | Baseline and 48 Weeks | Change in t-score. Higher scores indicate greater fatigue severity. |
| Primary | Change in 30 Second Site-to-Stand Test. | Baseline and 48 Weeks | Change in number of stands |
| Secondary | Change in Quality of Life in Neurological Disorders (Neuro-QoL) Lower Extremity Function (Mobility) Short Form | Baseline and 48 weeks | Change in t-score. Higher scores indicate greater functional ability. |
| Secondary | Change in Individual Activity Assessments - Interference | Baseline and 48 Weeks | Assessing how much Primary Mitochondrial Disease symptoms get in the way of the ability to perform individually selected activities. A 5 point scale ranging from "No interference" to "Completely interferes" |
| Secondary | Individual Activity Assessment - Change | 48 weeks | Assessing the ability to perform individually selected activities compared to baseline. A 5 point scale ranging from "Much worse" to "Much better" |
| Secondary | Change in Patient Global Impression of Severity | Baseline and 48 weeks | Scale ranging from 1 (none) to 4 (severe). |
| Secondary | Score on Patient Global Impression of Change | 48 weeks | Scale ranging from 2 (much better) to -2 (much worse). |
| Secondary | Change in Clinician Global Impression of Severity | Baseline and 48 weeks | Scale ranging from 1 (none) to 4 (severe). |
| Secondary | Score on Clinician Global Impression of Change | 48 weeks | Scale ranging from 2 (much better) to -2 (much worse). |
| Secondary | Change in Newcastle Mitochondrial Disease Adult Scale (NMDAS) - subscales I-III | Baseline and 48 weeks | Measured on a 6-point rating scale from 0 to 5. Total score across all 3 subscales ranges from 0 to 140. Higher scores indicate more extensive and severe system involvements. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 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/NCT05650229
GET https://conductscience.org/api/trials/v1/NCT05650229/registered