Trial · NCT03563053
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
NCT03563053, Phase 3, Terminated, Quince Therapeutics S.p.A.; results first posted 2024-10-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study. Trials, record NCT03563053. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03563053
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
- NCT number
- NCT03563053
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 104 participants, actual
- Lead sponsor
- Quince Therapeutics S.p.A.
- Conditions
- Ataxia Telangiectasia, Genetic Syndrome
- Interventions
- EryDex System
- First posted
- 2018-06-20
- Last updated
- 2024-10-08
- Other registries' IDs
- eudract:2018-000338-36
- Results first posted
- 2024-10-08
- Limitations and caveats
India was particularly affected by COVID-19-dependent treatment and visit interruptions.This long interruption would have resulted in insufficient long-term safety data. For this reason, EryDel (now Quince Therapeutics) decided to discontinue the IEDAT-03-2018 study in India.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months) | Assessment of TEAEs, treatment-emergent serious adverse events (TESAE), and adverse events of special interest (AESI) were performed throughout the study, from the time of signing of the ICF at Baseline Visit through to the Final Study Visit (Month 12 or early discontinuation). All patients were to be followed up through 30 days after the Final Visit (Month 12 or early discontinuation) or at least 60 days after the final infusion, whichever was longer. |
| Secondary | Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | The EQ-5D included single item measures of 5 health dimensions: * mobility, * self-care, * usual activities, * pain / discomfort, and * anxiety / depression. The EQ-5D-5L included five levels of severity (i.e., no problems, slight problems, moderate problems, severe problems, and extreme problems) for each of the five EQ-5D dimensions. These levels were scored from 1 = no problems to 5 = extreme problems: from 5, min/worst, to 25, best/max).The EQ-5D results were converted into a continuous index score: as for this index score, the closer the value is to 1, the better is the health status. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations. |
| Secondary | Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | 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. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations. |
| 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 From Baseline (Visit 1 - Day 0) to Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | 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. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations. |
| Secondary | Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | 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 0, max 12) * Speech Disorder- 2 items (min 0, max 8). An higher scores - both for total and subscores - indicate a higher level of disease impairment. The subscores are added to give the total score. The efficacy data focus mainly up to Month 36 as afterwards the number of patients / assessments dropped below 20% of the initial sample size, which was required for the efficacy evaluations. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months) | Safety Population (SAF): the Safety Analysis Set consisted of all patients who provided informed consent or assent and who received any dose of study medication during IEDAT-03-2018 study. The Safety Analysis Set was used for all safety analyses. | Number of events | NUMBER |
| SECONDARY | Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | Full Analysis Set Population (FAS): the FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study. | score on a scale | MEAN |
| SECONDARY | Number of Patients With Improving, Stable or Worsening Score Using a Clinical Global Impression of Change (CGI-C) From Baseline (Visit 1- Day 0) to Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | The FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study. | 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 From Baseline (Visit 1 - Day 0) to Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | The FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36 | From Baseline (Visit 1- Day 0) to Month 36 | The FAS comprised all patients who entered IEDAT-03-2018 study, had a Baseline Efficacy Assessment in IEDAT-03-2018 and who received at least one dose of study medication and had at least one post-Baseline Efficacy Assessment of the ICARS in this extension study. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group74
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | Number of TEAEs | 1232 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | Number of Serious TEAEs | 19 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | Number of TEAE leading to Death | 0 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | Number of TEAE leading to Permanent Withdrawal of Treatment | 5 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | Adverse Events During COVID-19 Interruption - On Dose | 5 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | Adverse Events During COVID-19 Interruption - Off Dose | 3 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | Adverse Events During COVID-19 Interruption - Restart | 55 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | TEAE by Worst Intensity - Mild | 1028 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | TEAE by Worst Intensity - Moderate | 187 | Number of events | |
| Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study | Overall Population (SAF) | TEAE by Worst Intensity - Severe | 17 | Number of events |
1 to 10 of 74Source ClinicalTrials.gov
See all 74 results by groupParticipant flow11
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Active Drug | 104 | |
| Overall Study | Total Set | Active Drug | 104 | |
| Overall Study | Safety Set | Active Drug | 104 | |
| Overall Study | Full Analysis Set | Active Drug | 80 | |
| Overall Study | Month 12 | Active Drug | 64 | |
| Overall Study | Month 24 | Active Drug | 33 | |
| Overall Study | Month 36 | Active Drug | 21 | |
| Overall Study | Month 48 | Active Drug | 7 | |
| Overall Study | Month 50.5 | Active Drug | 1 | |
| Overall Study | COMPLETED | Active Drug | 0 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 participant flowAdverse events39
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 | Upper respiratory tract infection | Infections and infestations | Overall Population (SAF) | 23 | 104 | 36 |
| other | Nasopharyngitis | Infections and infestations | Overall Population (SAF) | 21 | 104 | 30 |
| other | Corona Virus Infection | Infections and infestations | Overall Population (SAF) | 17 | 104 | 19 |
| other | Bronchitis | Infections and infestations | Overall Population (SAF) | 10 | 104 | 15 |
| other | Urinary Tract Infection | Infections and infestations | Overall Population (SAF) | 6 | 104 | 7 |
| other | Pyrexia | General disorders | Overall Population (SAF) | 33 | 104 | 54 |
| other | Fatigue | General disorders | Overall Population (SAF) | 16 | 104 | 20 |
| other | Infusion Related Reaction | Injury, poisoning and procedural complications | Overall Population (SAF) | 28 | 104 | 199 |
| other | Fall | Injury, poisoning and procedural complications | Overall Population (SAF) | 7 | 104 | 10 |
| other | Diarrhoea | Gastrointestinal disorders | Overall Population (SAF) | 23 | 104 | 32 |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Overall Population (SAF) | 0 | 104 |
| Total, other adverse events | Overall Population (SAF) | 98 | 104 |
| Total, serious adverse events | Overall Population (SAF) | 12 | 104 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39917433 | DERIVED |
Source PubMed
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/NCT03563053
GET https://conductscience.org/api/trials/v1/NCT03563053/results?page=2