Trial · NCT04556656
PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
NCT04556656, Phase 3, Completed, Prilenia; results first posted 2025-03-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD. Trials, record NCT04556656. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04556656
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
- NCT number
- NCT04556656
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 499 participants, actual
- Lead sponsor
- Prilenia
- Conditions
- Huntington Disease
- Interventions
- Pridopidine, Placebo
- First posted
- 2020-09-21
- Last updated
- 2025-03-12
- Other registries' IDs
- eudract:2020-002822-10
- Results first posted
- 2025-03-12
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT) | From baseline to Week 65 | The primary efficacy endpoint for this study was the change from baseline to Week 65 in the TFC (defined as the sum of all TFC 5-items ratings \[domestic chores, activities of daily living, finances, care level, and occupation\]). The TFC is the standard and well-accepted clinical scale for staging and tracking the progression of HD using functional capacity. Scores range from 0 to 13, with 13 as the least affected and 0 as complete incapacity. |
| Primary | Change From Baseline to Week 65 in the UHDRS TFC Score (ITT) | From baseline to Week 65. | The primary efficacy endpoint for this study was the change from baseline to Week 65 in the TFC (defined as the sum of all TFC 5-items ratings \[domestic chores, activities of daily living, finances, care level, and occupation\]). The TFC is the standard and well-accepted clinical scale for staging and tracking the progression of HD using functional capacity. Scores range from 0 to 13, with 13 as the least affected and 0 as complete incapacity. |
| Secondary | Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT) | From baseline to Week 65 | The composite Unified Huntington Disease Rating Scale (cUHDRS) uses 4 components: Total Motor Score (TMS) assesses motor features (oculomotor, dysarthria, chorea, dystonia, gait, postural stability). Higher score = worse outcome. Best score=0. Worst score= 124. Stroop Word Reading (SWR) measures attention and mental flexibility. Pat. reads names of colors printed in black ink. Scores reflect correct responses in 45 sec. Higher score = better outcome. Best score=100. Worst score=0. Symbol Digit Modalities Test (SDMT) tests psychomotor speed and working memory. Participant has 90 sec to match numbers with symbols. Scores = correct answers in 90 sec. Higher score = better outcome. Best score=120. Worst score=0. Total Functional Capacity (TFC) tests the capacity to maintain daily living, finances, care level, occupation. Higher score = better outcome. Best score=13. Worst score=0. Total integrated cUHDRS scale range: -7.6 to 24.8. The higher, the better. |
| Other | Change From Baseline in cUHDRS Total Score - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78 | The composite Unified Huntington Disease Rating Scale (cUHDRS) assesses 4 components (see secondary outcome for details): Total Motor Score (TMS) for motor features. Higher score = worse outcome. Worst = 124. Stroop Word Reading (SWR) measures attention and mental flexibility. Higher score = better outcome. Symbol Digit Modalities Test (SDMT) tests psychomotor speed and working memory. Higher score = better outcome. Total Functional Capacity (TFC) tests the capacity to maintain daily living, finances, care level, occupation. The higher, the better. Total cUHDRS scale range: -7.6 to 24.8 (assuming 150 as the max score of SWR). The higher, the better. This sensitivity analysis was performed in a sub-group of patients who were off neuroleptics AND off vesicular monoamine transporter-2 (VMAT2) inhibitors (together called antidopaminergics, or ADMs) at any time during the study. |
| Other | Change From Baseline in Q-Motor Finger Tapping Inter-Onset Interval (IOI) Mean - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 52, Week 65, and Week 78. | Quantitative (Q)-motor is a clinical assessment of fine motor skills. It is based on the application of pre-calibrated and temperature-controlled force transducers and 3-dimensional position sensors. The index finger is positioned above a force transducer and is to tap as fast as possible. The start was defined as a rise of the force by 0.05 Newton above maximal baseline level. The tap ended when it dropped to 0.05 N before the maximal baseline level was reached again. The IOI refers to the time between the onset of consecutive taps (the faster, the better). In addition to the main efficacy analyses, sensitivity analyses were performed in a sub-group of patients who were off neuroleptics AND off vesicular monoamine transporter-2 (VMAT2) inhibitors (together called antidopaminergics, or ADMs) at any time during the study. |
| Other | Change From Baseline in Q-Motor Pronation/Supination Inter-Tap-Interval (ITI) Mean - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 52, Week 65, and Week 78. | Quantitative (Q)-motor is a clinical assessment of fine motor skills that are crucial for daily activities. It is based on the application of pre-calibrated and temperature-controlled force transducers and 3-dimensional position sensors. Pronation/Supination assesses the regularity of hand taps. The force and duration of the hand taps were recorded similarly to the speeded tapping task. One pronation/supination hand tapping measure is ITI (the faster, the better). In addition to the main efficacy analyses, sensitivity analyses were performed in a sub-group of patients who were off neuroleptics AND off vesicular monoamine transporter-2 (VMAT2) inhibitors (together called antidopaminergics, or ADMs) at any time during the study. |
| Other | Change in Q-Motor Pronation/Supination Inter-Onset-Interval (IOI) Mean - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 52, Week 65, and Week 78. | Quantitative (Q)-motor is a clinical assessment of fine motor skills that are crucial for daily activities. It is based on the application of pre-calibrated and temperature-controlled force transducers and 3-dimensional position sensors. Pronation/Supination assess the regularity of hand taps. The force and duration of the hand taps were recorded similarly to the speeded tapping task. One pronation/supination hand tapping measure is IOI (the faster, the better). In addition to the main efficacy analyses, sensitivity analyses were performed in a sub-group of patients who were off neuroleptics AND off vesicular monoamine transporter-2 (VMAT2) inhibitors (together called antidopaminergics, or ADMs) at any time during the study. |
| Other | Change From Baseline in the UHDRS TFC Score - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78 | Total Functional Capacity (TFC) tests the capacity to maintain domestic chores, activities of daily living, finances, care level, and occupation. Scores from 0 - 13. Higher score = better outcome. In addition to the main efficacy analyses, sensitivity analyses were performed in a sub-group of patients who were off neuroleptics AND off vesicular monoamine transporter-2 (VMAT2) inhibitors (together called antidopaminergics, or ADMs) at any time during the study. |
| Other | Change From Baseline in Stroop Word Reading (SWR) - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78 | Stroop Word Reading (SWR) measures attention and mental flexibility. Pat. reads names of colors printed in black ink. Scores reflect correct responses in 45 sec. Higher score = better outcome. In addition to the main efficacy analyses, sensitivity analyses were performed in a sub-group of patients who were off neuroleptics AND off vesicular monoamine transporter-2 (VMAT2) inhibitors (together called antidopaminergics, or ADMs) at any time during the study. |
| Other | Change From Baseline in Symbol Digit Modalities Test (SDMT) - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78. | Symbol Digit Modalities Test (SDMT) tests psychomotor speed and working memory. Participant has 90 sec to write match numbers with symbols. Scoring sums correct substitutions in 90 second interval (max = 110). Higher score = better outcome. In addition to the main efficacy analyses, sensitivity analyses were performed in a sub-group of patients who were off neuroleptics AND off vesicular monoamine transporter-2 (VMAT2) inhibitors (together called antidopaminergics, or ADMs) at any time during the study. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT) | From baseline to Week 65 | The modified intent to treat (mITT) population included all participants in the ITT population who received at least one dose of study drug and had valid in clinic TFC scores both at baseline and at least one post-baseline timepoint. The mITT population was analyzed according to the treatment to which the participant was randomized.~The mITT population was the main analysis population for the primary endpoint in non-EMA regions. Note that the analysis was based on observed data. | score on a scale | LEAST_SQUARES_MEAN |
| PRIMARY | Change From Baseline to Week 65 in the UHDRS TFC Score (ITT) | From baseline to Week 65. | The intent to treat (ITT) population included all randomized participants. The ITT population was analyzed according to the treatment to which the participant was randomized.~The ITT population was the main analysis population for the primary endpoint in the EMA region. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT) | From baseline to Week 65 | The modified intent to treat (mITT) population included all participants in the ITT population who received at least one dose of study drug and had valid in-clinic TFC scores both at baseline and at least one post-baseline timepoint. The mITT population was analyzed according to the treatment to which the participant was randomized. Note that the analysis was based on as observed data only, patient numbers are therefore lower. | score on a scale | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in cUHDRS Total Score - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78 | The mITT population was used for subgroup analyses. Note: Analyses/p-values were generated on observed data. Patient numbers at individual weeks varied. | score on a scale | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Q-Motor Finger Tapping Inter-Onset Interval (IOI) Mean - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 52, Week 65, and Week 78. | The mITT population was used for subgroup analyses. Note: Analyses/p-values were generated on observed data. Patient numbers at individual weeks varied. | milliseconds (decrease = improvement) | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Q-Motor Pronation/Supination Inter-Tap-Interval (ITI) Mean - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 52, Week 65, and Week 78. | The mITT population was used for subgroup analyses. Note: Analyses/p-values were generated on observed data. Patient numbers at individual weeks varied. | milliseconds | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change in Q-Motor Pronation/Supination Inter-Onset-Interval (IOI) Mean - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 52, Week 65, and Week 78. | The mITT population was used for subgroup analyses. Note: Analyses/p-values were generated on observed data. Patient numbers at individual weeks varied. | milliseconds | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in the UHDRS TFC Score - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78 | The mITT population was used for subgroup analyses. Note: Analyses/p-values were generated on observed data. Patient numbers at individual weeks varied. | score on a scale | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Stroop Word Reading (SWR) - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78 | The mITT population was used for subgroup analyses. Note: Analyses/p-values were generated on observed data. Patient numbers at individual weeks varied. | score on a scale | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Symbol Digit Modalities Test (SDMT) - Patients Off ADMs (mITT) | Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78. | Note: Analyses/p-values were generated on observed data. Patient numbers at individual weeks varied. | score on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group80
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT) | Pridopidine | -1.18 | Standard Error 0.119 | score on a scale | |
| Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT) | Placebo | -0.95 | Standard Error 0.119 | score on a scale | |
| Change From Baseline to Week 65 in the UHDRS TFC Score (ITT) | Pridopidine | -1.17 | Standard Error 0.120 | score on a scale | |
| Change From Baseline to Week 65 in the UHDRS TFC Score (ITT) | Placebo | -0.94 | Standard Error 0.120 | score on a scale | |
| Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT) | Pridopidine | -0.99 | Standard Error 0.109 | score on a scale | |
| Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT) | Placebo | -0.88 | Standard Error 0.108 | score on a scale | |
| Change From Baseline in cUHDRS Total Score - Patients Off ADMs (mITT) | Pridopidine | Week 26 | 0.15 | Standard Error 0.114 | score on a scale |
| Change From Baseline in cUHDRS Total Score - Patients Off ADMs (mITT) | Placebo | Week 26 | -0.31 | Standard Error 0.108 | score on a scale |
| Change From Baseline in cUHDRS Total Score - Patients Off ADMs (mITT) | Pridopidine | Week 39 | 0.10 | Standard Error 0.130 | score on a scale |
| Change From Baseline in cUHDRS Total Score - Patients Off ADMs (mITT) | Placebo | Week 39 | -0.34 | Standard Error 0.125 | score on a scale |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pridopidine | 250 | |
| Overall Study | STARTED | Placebo | 249 | |
| Overall Study | COMPLETED | Pridopidine | 222 | |
| Overall Study | COMPLETED | Placebo | 227 | |
| Overall Study | NOT COMPLETED | Pridopidine | 28 | |
| Overall Study | NOT COMPLETED | Placebo | 22 |
Source ClinicalTrials.gov
Adverse events130
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 | Weight decreased | Investigations | Pridopidine | 13 | 250 | 13 |
| other | Weight decreased | Investigations | Placebo | 7 | 249 | 7 |
| other | Fall | Injury, poisoning and procedural complications | Pridopidine | 55 | 250 | 114 |
| other | Fall | Injury, poisoning and procedural complications | Placebo | 57 | 249 | 111 |
| other | Contusion | Injury, poisoning and procedural complications | Pridopidine | 12 | 250 | 20 |
| other | Contusion | Injury, poisoning and procedural complications | Placebo | 14 | 249 | 15 |
| other | Headache | Nervous system disorders | Pridopidine | 16 | 250 | 18 |
| other | Headache | Nervous system disorders | Placebo | 25 | 249 | 34 |
| other | Diarrhea | Gastrointestinal disorders | Pridopidine | 21 | 250 | 27 |
| other | Diarrhea | Gastrointestinal disorders | Placebo | 22 | 249 | 32 |
1 to 10 of 130Source ClinicalTrials.gov
See all 130 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pridopidine | 4 | 250 |
| Total, all-cause mortality | Placebo | 1 | 249 |
| Total, other adverse events | Pridopidine | 204 | 250 |
| Total, other adverse events | Placebo | 212 | 249 |
| Total, serious adverse events | Pridopidine | 34 | 250 |
| Total, serious adverse events | Placebo | 21 | 249 |
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Pridopidine vs Placebo: deaths from any cause | Trial comparisons |
| Pridopidine vs Placebo: serious adverse events | Trial comparisons |
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Symbol Coding | Secondary outcome |
Source ConductCognition
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.
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