Trial · NCT05358717
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT05358717, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2026-01-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD). Trials, record NCT05358717. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05358717
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
- NCT number
- NCT05358717
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 159 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Huntington Disease
- Interventions
- PTC518, Placebo
- First posted
- 2022-05-03
- Last updated
- 2026-06-16
- Other registries' IDs
- eudract:2021-003852-18
- Results first posted
- 2026-01-13
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline up to Month 18 | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug. A summary of other non-serious AEs and all serious adverse events (SAEs), regardless of causality is located in the 'Reported AE section'. |
| Primary | Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3 | Baseline, Month 3 | Least square (LS) mean and 95% confidence interval (CI) were calculated using mixed-model repeated measures (MMRM). The blood tHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes. |
| Secondary | Percent Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12 | Baseline, Month 12 | LS mean and standard error (SE) were calculated using MMRM. |
| Secondary | Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 12 | Baseline, Month 12 | cUHDRS includes the Total Functional Capacity (range, 0-13; higher score means better functioning), Total Motor Score (range, 0-124; higher score means worse motor severity), Symbol Digit Modality Test (range, 0-110, correctly paired numbers-symbols in 90 seconds; higher score means better cognitive performance), and Stroop Word Reading (range, 0-no max value, correctly read colour words in 45 seconds; higher score means better cognitive performance) scores. The total score for the cUHDRS ranges from approximately 3 to 18, where higher scores indicated better functioning. LS mean and SE were calculated using analysis of covariance (ANCOVA) model. |
| Secondary | Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 12 | Baseline, Month 12 | LS mean and 95% CI were calculated using MMRM. The blood tHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes. |
| Secondary | Percent Change From Baseline in Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) at Month 12 | Baseline, Month 12 | LS mean and 95% CI were calculated using MMRM. The CSF mHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes. |
| Secondary | Percent Change From Baseline in Blood mHTT Protein at Month 12 | Baseline, Month 12 | LS mean and 95% CI were calculated using MMRM. The blood mHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| SECONDARY | Percent Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12 | Baseline, Month 12 | ITT Population included all randomized participants who took at least 1 dose of the study drug. | percent change | LEAST_SQUARES_MEAN |
| PRIMARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline up to Month 18 | Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3 | Baseline, Month 3 | ITT Population included all randomized participants who took at least 1 dose of the study drug. | percent change | GEOMETRIC_MEAN |
| SECONDARY | Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 12 | Baseline, Month 12 | ITT Population included all randomized participants who took at least 1 dose of the study drug. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 12 | Baseline, Month 12 | ITT Population included all randomized participants who took at least 1 dose of the study drug. | percent change | GEOMETRIC_MEAN |
| SECONDARY | Percent Change From Baseline in Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) at Month 12 | Baseline, Month 12 | ITT Population included all randomized participants who took at least 1 dose of the study drug. | percent change | GEOMETRIC_MEAN |
| SECONDARY | Percent Change From Baseline in Blood mHTT Protein at Month 12 | Baseline, Month 12 | ITT Population included all randomized participants who took at least 1 dose of the study drug. | percent change | GEOMETRIC_MEAN |
Source ClinicalTrials.gov
Results by group21
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12 | Placebo | 4.42 | Standard Error 0.413 | percent change | |
| Percent Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12 | PTC518 5 mg | 4.99 | Standard Error 0.413 | percent change | |
| Percent Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12 | PTC518 10 mg | 5.74 | Standard Error 0.391 | percent change | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Placebo | 46 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | PTC518 5 mg | 43 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | PTC518 10 mg | 49 | Participants | ||
| Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3 | Placebo | -1.40 | 95% Confidence Interval -7.25 | percent change | |
| Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3 | PTC518 5 mg | -17.36 | 95% Confidence Interval -22.36 | percent change | |
| Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3 | PTC518 10 mg | -28.67 | 95% Confidence Interval -32.74 | percent change | |
| Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 12 | Placebo | -0.64 | Standard Error 0.168 | units on a scale |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 53 | |
| Overall Study | STARTED | PTC518 5 mg | 52 | |
| Overall Study | STARTED | PTC518 10 mg | 54 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Placebo | 53 | |
| Overall Study | Received at Least 1 Dose of Study Drug | PTC518 5 mg | 52 | |
| Overall Study | Received at Least 1 Dose of Study Drug | PTC518 10 mg | 54 | |
| Overall Study | COMPLETED | Placebo | 51 | |
| Overall Study | COMPLETED | PTC518 5 mg | 47 | |
| Overall Study | COMPLETED | PTC518 10 mg | 51 | |
| Overall Study | NOT COMPLETED | Placebo | 2 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events87
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 | Lymphopenia | Blood and lymphatic system disorders | Placebo | 0 | 53 | 0 |
| other | Lymphopenia | Blood and lymphatic system disorders | PTC518 5 mg | 1 | 52 | 1 |
| other | Lymphopenia | Blood and lymphatic system disorders | PTC518 10 mg | 3 | 54 | 3 |
| other | Constipation | Gastrointestinal disorders | Placebo | 0 | 53 | 0 |
| other | Constipation | Gastrointestinal disorders | PTC518 5 mg | 0 | 52 | 0 |
| other | Constipation | Gastrointestinal disorders | PTC518 10 mg | 3 | 54 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 3 | 53 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | PTC518 5 mg | 4 | 52 | 5 |
| other | Diarrhoea | Gastrointestinal disorders | PTC518 10 mg | 5 | 54 | 6 |
| other | Nausea | Gastrointestinal disorders | Placebo | 1 | 53 | 1 |
1 to 10 of 87Source ClinicalTrials.gov
See all 87 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 1 | 53 |
| Total, all-cause mortality | PTC518 5 mg | 1 | 52 |
| Total, all-cause mortality | PTC518 10 mg | 0 | 54 |
| Total, other adverse events | Placebo | 32 | 53 |
| Total, other adverse events | PTC518 5 mg | 34 | 52 |
| Total, other adverse events | PTC518 10 mg | 41 | 54 |
| Total, serious adverse events | Placebo | 4 | 53 |
| Total, serious adverse events | PTC518 5 mg | 1 | 52 |
| Total, serious adverse events | PTC518 10 mg | 2 | 54 |
Source ClinicalTrials.gov
Papers2
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/NCT05358717
GET https://conductscience.org/api/trials/v1/NCT05358717/results?page=2