Trial · NCT01255358
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
NCT01255358, Phase 2, Completed, Quince Therapeutics S.p.A.; results first posted 2024-10-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients. Trials, record NCT01255358. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01255358
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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 Teleangiectasia Patients
- NCT number
- NCT01255358
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- Quince Therapeutics S.p.A.
- Conditions
- Nervous System Disorder, Genetic Syndrome
- Interventions
- Dexamethasone
- First posted
- 2010-12-07
- Last updated
- 2024-10-10
- Other registries' IDs
- eudract:2010-022315-19
- Results first posted
- 2024-10-10
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population | At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180) | ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment. |
| Primary | Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population | At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180) | ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment. |
| Secondary | Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population | At visit 7 (final visit, day 180) | An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as: * very much improved (1), * slightly improved (2), * no change (3), * slightly worsened (4), * very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome. |
| Secondary | Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population | At visit 7 (final visit, day 180) | An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as: * very much improved (1), * slightly improved (2), * no change (3), * slightly worsened (4), * very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome. |
| Secondary | Mean Change From V4 to V7 in Ocular Motility - ITT Population | At visit 7 (final visit, day 180) | An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7. At V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5). |
| Secondary | Mean Change From V4 to V7 in Ocular Motility - PP Population | At visit 7 (final visit, day 180) | An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7. At V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5). |
| Secondary | Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population | At visits 4 (90 days) and 7 (180 days, final visit) | VABS aggregate scale is used. VABS is divided into 4 adaptive domains and 11 subdomains: 1. Communication: Receptive, Expressive, Written; 2. Daily Living Skills: Personal, Domestic, Community; 3. Socialisation: Interpersonal Relationships, Play \& Leisure Time, Coping Skills; 4. Motor Skills: Gross, Fine. For each \*question\* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome): * 0 = the subject never performs the behaviour without help or reminders; * 1 = rarely performs (as above); * 2 = sometimes performs (as above); * 3 = often performs (as above); * 4 = almost always performs (as above); The total subdomain score is the sum of each \*question\* scores and the total domain score is the sum of each subdomains' scores. Domains min-max interval scores: 0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome) |
| Secondary | Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population | At visits 4 (90 days) and 7 (180 days, final visit) | VABS aggregate scale is used. VABS is divided into 4 adaptive domains and 11 subdomains: 1. Communication: Receptive, Expressive, Written; 2. Daily Living Skills: Personal, Domestic, Community; 3. Socialisation: Interpersonal Relationships, Play \& Leisure Time, Coping Skills; 4. Motor Skills: Gross, Fine. For each \*question\* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome): * 0 = the subject never performs the behaviour without help or reminders; * 1 = rarely performs (as above); * 2 = sometimes performs (as above); * 3 = often performs (as above); * 4 = almost always performs (as above); The total subdomain score is the sum of each \*question\* scores and the total domain score is the sum of each subdomains' scores. Domains min-max interval scores: 0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome) |
| Secondary | Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs) | Throughout the study, until the end of 6 months of treatment (day 180) | The effect of ERY-DEX on treatment emergent adverse events was expressed as the number of patients showing at least one TEAE or 1 serious AE |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population | At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180) | Intent to treat population (ITT) patients receiving at least one treatment | units on a scale | MEAN |
| PRIMARY | Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population | At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180) | The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:~* compliance with all entry criteria~* absence of major protocol violations~* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation). | units on a scale | MEAN |
| SECONDARY | Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population | At visit 7 (final visit, day 180) | The ITT Population includes all 22 patients enrolled into the study. | units on a scale | MEAN |
| SECONDARY | Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population | At visit 7 (final visit, day 180) | The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:~* compliance with all entry criteria~* absence of major protocol violations~* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation). | units on a scale | MEAN |
| SECONDARY | Mean Change From V4 to V7 in Ocular Motility - ITT Population | At visit 7 (final visit, day 180) | The ITT Population includes all 22 patients enrolled into the study. | units on a scale | MEAN |
| SECONDARY | Mean Change From V4 to V7 in Ocular Motility - PP Population | At visit 7 (final visit, day 180) | The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:~* compliance with all entry criteria~* absence of major protocol violations~* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation). | units on a scale | MEAN |
| SECONDARY | Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population | At visits 4 (90 days) and 7 (180 days, final visit) | The ITT Population includes all 22 patients enrolled into the study. | units on a scale | MEAN |
| SECONDARY | Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population | At visits 4 (90 days) and 7 (180 days, final visit) | The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:~* compliance with all entry criteria~* absence of major protocol violations~* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation). | units on a scale | MEAN |
| SECONDARY | Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs) | Throughout the study, until the end of 6 months of treatment (day 180) | The SP includes the 22 patients who have received at least one dose of planned trial treatment. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population | ERY-DEX | V2 | -2.2 | Standard Deviation 5.6 | units on a scale |
| Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population | ERY-DEX | V4 | -3.4 | Standard Deviation 7.3 | units on a scale |
| Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population | ERY-DEX | V7 | -4.0 | Standard Deviation 7.5 | units on a scale |
| Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population | ERY-DEX | V2 | -2.4 | Standard Deviation 4.9 | units on a scale |
| Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population | ERY-DEX | V4 | -3.9 | Standard Deviation 7.1 | units on a scale |
| Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population | ERY-DEX | V7 | -5.2 | Standard Deviation 7.0 | units on a scale |
| Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population | ERY-DEX | 0.2 | Standard Deviation 0.6 | units on a scale | |
| Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population | ERY-DEX | 0.2 | Standard Deviation 0.6 | units on a scale | |
| Mean Change From V4 to V7 in Ocular Motility - ITT Population | ERY-DEX | -0.3 | Standard Deviation 0.5 | units on a scale | |
| Mean Change From V4 to V7 in Ocular Motility - PP Population | ERY-DEX | -0.3 | Standard Deviation 0.5 | units on a scale |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | EryDex | 22 | |
| Overall Study | COMPLETED | EryDex | 18 | |
| Overall Study | NOT COMPLETED | EryDex | 4 |
Source ClinicalTrials.gov
Adverse events6
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 | General disorders | General disorders | Ery-Dex | 3 | 22 | 3 |
| other | immune disorders | Immune system disorders | Ery-Dex | 2 | 22 | 2 |
| other | infections and infestations | Infections and infestations | Ery-Dex | 15 | 22 | 17 |
| other | respiratory disorders | Respiratory, thoracic and mediastinal disorders | Ery-Dex | 4 | 22 | 4 |
| serious | Bronchopneumonia | Infections and infestations | Ery-Dex | 1 | 22 | 1 |
| serious | Bronchiectasis | Respiratory, thoracic and mediastinal disorders | Ery-Dex | 1 | 22 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ery-Dex | 0 | 22 |
| Total, other adverse events | Ery-Dex | 15 | 22 |
| Total, serious adverse events | Ery-Dex | 2 | 22 |
Source ClinicalTrials.gov
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/NCT01255358
GET https://conductscience.org/api/trials/v1/NCT01255358/registered