Trial · NCT02770807
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
NCT02770807, Phase 3, Completed, Quince Therapeutics S.p.A.; results first posted 2024-05-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients. Trials, record NCT02770807. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02770807
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
- NCT number
- NCT02770807
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 176 participants, actual
- Lead sponsor
- Quince Therapeutics S.p.A.
- Conditions
- Nervous System Disease, Genetic Syndrome
- Interventions
- EryDex Low dose DSP, EryDex High dose DSP, Pooled Placebo
- First posted
- 2016-05-12
- Last updated
- 2024-05-10
- Other registries' IDs
- eudract:2015-005241-31
- Results first posted
- 2024-05-09
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS) | to Month 6 (Visit 9) | The International Cooperative Ataxia Rating Scale (ICARS) was an assessment of the degree of impairment in patients with cerebellar ataxia and was administered in its entirety; however, the primary efficacy assessment was based on the modified (m)ICARS, which excluded the Oculomotor domain (items 17 to 19) and items 8 to 12 of the Kinetic Functions domain of the ICARS. The mICARS was a 54 points maximum score (min 0) questionnaire divided into 3 sections: * Posture and Gait Disturbance section-7 items (min score 0, max score 34) * Kinetic Function-2 items (min score 0, max score 12) * Speech Disorder- 2 items (min score 0, max score 8). The assessment was designed to be completed within 30 minutes, and higher scores - both for total and subscores - indicate a higher level of disease impairment. The subscores are added to give the total score. |
| Secondary | Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | to Month 6 (Visit 9) | The CGI-C scale assesses the change in the patient's clinical status from baseline using a 7-point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change. Clinicians were required to conduct a full clinical interview and examination of the patient. The interview and examination assessed various aspects of the patient's appearance (grooming, evidence of falls, etc.), ataxia, cognition (orientation, calculation ability, language, ability to follow commands, memory, etc.), apraxia, dysarthria, extrapyramidal motor symptoms, activities of daily living, and mood. The higher the score the worse the outcome. |
| Secondary | Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT | to Visit 9 (Month 6) | The CGI-S scale measures global severity of illness at a given point in time, and is usually rated on a 7-point, Likert-type scale ranging from 1 (normal, not ill at all) to 7 (among the most extremely ill patients). No version of the CGI-S exists which has been specifically adapted for use in patients with A-T; therefore, a 5-point version was developed that considered the severity of the following symptoms of A-T: ataxia (walking), dysarthria, dysmetria, extrapyramidal symptoms (chorea, myoclonus, dystonia, and tremor), and eye movements. Ratings of none (0), mild (1), moderate (2), severe (3), and very severe (4) were selected based on the level of symptomatology. The higher the score the worse the outcome. |
| Secondary | Change From Baseline of Vineland Adaptive Behavior Scale (VABS-II) Scores - Last Observation Carried Forward (LOCF) | to Visit 9 (Month 6) | VABS-II was a questionnaire to assess adaptive behavior. It contained 4 domains each with 2-3 subdomains, every subdomain contained various items (questions): A) communication (receptive, expressive, written) B) daily living skills (personal, domestic, community) C) socialization (interpersonal relationships, play and leisure time, coping skills) D) motor skills (gross motor, fine motor). The expanded version of the VABS consisted of 540 items, 261 of which used in this trial. The possible score for each item was from 0 to 4 based on whether the patient performed the activity "never", "rarely", "sometimes", "often" or "almost always". At the end of each domain section, a total score (the sum of the score for each item) was calculated. Domain A: min score 0, max score 572. Domain B: 0 - 800. Domain C: 0 - 580. Domain D: 0 - 424. A grand total score (A+B+C+D scores) was provided (range:0-2376) The lower the score the higher the disability at each level (domains and subdomains). |
| Secondary | Number of Patients With at Least One Treatment-Emergent Adverse Event (TEAE) at Month 6 | to Visit 9 (Month 6) | TEAE = Treatment Emergent Adverse Events were any AEs started on or after the day of the first infusion through the day just prior to the day of the Visit 9 ("Month 6") infusion, or \<=60 days after last dose if the subject never continued past this period. |
| Secondary | Number of Patients With at Least One Treatment-Emergent Adverse Event (TEAE) at Month 12 | to Visit 15 (Month 12) | TEAE = Treatment Emergent Adverse Events were any AEs started on or after the day of the first infusion through the day just prior to the day of the Visit 15 ("Month 12") infusion. Placebo patients who switched to EryDex treatment at 6 and 9 months were not added to the Extension Treatment Period safety data so that the results reported here under are for those patients who remained on placebo from the start of the study till the end of it. |
Source ClinicalTrials.gov
Outcomes6
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 Modified International Cooperative Ataxia Rating Scale (mICARS) | to Month 6 (Visit 9) | Modified Intention to Treat population - mITT: ll randomized patients who received at least one dose of study medication and had at least one post-baseline efficacy assessment of the primary efficacy variable | score on a scale | MEAN |
| SECONDARY | Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | to Month 6 (Visit 9) | Modified Intention to Treat population - mITT: ll randomized patients who received at least one dose of study medication and had at least one post-baseline efficacy assessment of the primary efficacy variable | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT | to Visit 9 (Month 6) | Modified Intention to Treat population - mITT: ll randomized patients who received at least one dose of study medication and had at least one post-baseline efficacy assessment of the primary efficacy variable | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline of Vineland Adaptive Behavior Scale (VABS-II) Scores - Last Observation Carried Forward (LOCF) | to Visit 9 (Month 6) | Modified Intention to Treat population - mITT: ll randomized patients who received at least one dose of study medication and had at least one post-baseline efficacy assessment of the primary efficacy variable | score on a scale | MEAN |
| SECONDARY | Number of Patients With at Least One Treatment-Emergent Adverse Event (TEAE) at Month 6 | to Visit 9 (Month 6) | Safety Population (SAF): all patients who received any amount of randomized treatment (also referred to as the ITT population). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Patients With at Least One Treatment-Emergent Adverse Event (TEAE) at Month 12 | to Visit 15 (Month 12) | Safety Population: all patients who received any amount of randomized treatment (also referred to as the ITT population). Placebo patients who switched to EryDex treatment at 6 and 9 months were not added to the Extension Treatment Period safety data so that the results reported are for those patients who remained on placebo from the start of the study (N=19) | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group66
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 Modified International Cooperative Ataxia Rating Scale (mICARS) | EryDex Low Dose DSP - mITT | 0.8 | Standard Deviation 3.56 | score on a scale | |
| Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS) | EryDex High Dose DSP - mITT | 1.0 | Standard Deviation 3.32 | score on a scale | |
| Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS) | Placebo - mITT | 2.3 | Standard Deviation 5.03 | score on a scale | |
| Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | EryDex Low Dose DSP - mITT | Improvement (Scores 1-3) | 19 | Participants | |
| Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | EryDex High Dose DSP - mITT | Improvement (Scores 1-3) | 27 | Participants | |
| Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | Placebo - mITT | Improvement (Scores 1-3) | 19 | Participants | |
| Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | EryDex Low Dose DSP - mITT | Stable or Worsening (Scores 4-7) | 37 | Participants | |
| Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | EryDex High Dose DSP - mITT | Stable or Worsening (Scores 4-7) | 27 | Participants | |
| Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) | Placebo - mITT | Stable or Worsening (Scores 4-7) | 35 | Participants | |
| Number of Patients With None to Severe (0 to 4) Scores in Clinical Global Impression of Severity (CGI-S)-Structured of Neurological Symptoms of AT | EryDex Low Dose DSP - mITT | CGI-S Score - 0 | 0 | Participants |
1 to 10 of 66Source ClinicalTrials.gov
See all 66 results by groupParticipant flow33
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | EryDex Low Dose DSP - SAF | 59 | |
| Overall Study | STARTED | EryDex High Dose DSP - SAF | 58 | |
| Overall Study | STARTED | Pooled Placebo - SAF | 59 | |
| Overall Study | Participants Who Took the Study Drug | EryDex Low Dose DSP - SAF | 59 | |
| Overall Study | Participants Who Took the Study Drug | EryDex High Dose DSP - SAF | 57 | |
| Overall Study | Participants Who Took the Study Drug | Pooled Placebo - SAF | 59 | |
| Overall Study | Modified Intention to Treat Population - mITT | EryDex Low Dose DSP - SAF | 56 | |
| Overall Study | Modified Intention to Treat Population - mITT | EryDex High Dose DSP - SAF | 54 | |
| Overall Study | Modified Intention to Treat Population - mITT | Pooled Placebo - SAF | 54 | |
| Overall Study | Safety Population - SAF | EryDex Low Dose DSP - SAF | 59 |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 participant flowAdverse events1,120
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 | Anemia | Blood and lymphatic system disorders | EryDex Low Dose DSP - SAF | 0 | 59 | |
| other | Anemia | Blood and lymphatic system disorders | EryDex High Dose DSP - SAF | 3 | 57 | |
| other | Anemia | Blood and lymphatic system disorders | Non-switch Placebo - SAF | 0 | 19 | |
| other | Anemia | Blood and lymphatic system disorders | Placebo Patients Switch to Low Dose - Month 6 | 0 | 9 | |
| other | Anemia | Blood and lymphatic system disorders | Placebo Patients Switch to High Dose - Month 6 | 0 | 9 | |
| other | Anemia | Blood and lymphatic system disorders | Placebo Patients Switch to Low Dose - Month 9 | 0 | 11 | |
| other | Anemia | Blood and lymphatic system disorders | Placebo Patients Switch to High Dose - Month 9 | 1 | 11 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | EryDex Low Dose DSP - SAF | 0 | 59 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | EryDex High Dose DSP - SAF | 0 | 57 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | Non-switch Placebo - SAF | 0 | 19 |
1 to 10 of 1,120Source ClinicalTrials.gov
See all 1,120 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | EryDex Low Dose DSP - SAF | 0 | 59 |
| Total, all-cause mortality | EryDex High Dose DSP - SAF | 0 | 57 |
| Total, all-cause mortality | Non-switch Placebo - SAF | 1 | 19 |
| Total, all-cause mortality | Placebo Patients Switch to Low Dose - Month 6 | 0 | 9 |
| Total, all-cause mortality | Placebo Patients Switch to High Dose - Month 6 | 0 | 9 |
| Total, all-cause mortality | Placebo Patients Switch to Low Dose - Month 9 | 0 | 11 |
| Total, all-cause mortality | Placebo Patients Switch to High Dose - Month 9 | 0 | 11 |
| Total, other adverse events | EryDex Low Dose DSP - SAF | 45 | 59 |
| Total, other adverse events | EryDex High Dose DSP - SAF | 50 | 57 |
| Total, other adverse events | Non-switch Placebo - SAF | 15 | 19 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers2
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/NCT02770807
GET https://conductscience.org/api/trials/v1/NCT02770807/results?page=2