Nervous System DisorderPhase 2IEDAT-01NCT01255358
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
A Phase 2 study testing Dexamethasone in people with Nervous System Disorder and Genetic Syndrome, run by Quince Therapeutics S.p.A. at 2 sites in Italy.
Trial summary
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes). Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Nervous System Disorder, Genetic Syndrome
- What is being tested?
- Dexamethasone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 2 sitesItaly
- Study dates
- Feb 2011to Dec 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- neurological signs of AT
- patients in autonomous gait or helped by a support
- proven molecular diagnosis of AT
- Males and females aged > 3 years
- Body weight >15 kg
- Plasma levels of Lymphocytes CD4+/mm3 > 500 (for patients aged 3-6 years) or > 200 (older than 6 years)
- written IC to participate.
Who cannot join
- Current or previous neoplastic disease
- History of severe impairment of the immunological system
- Chronic conditions representing a contraindication to the use of steroid drugs
- Non compliance with the study request
- Any previous steroid assumption within 30 days before starting ERY-DEX
- Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial
- Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods
Trial locations and contacts
2 sites in 1 country.
About this study
Official title: Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).
Read the detailed description
This is a multi-centre, single arm, open label, 6 months, phase II study to evaluate the effect of ERY-DEX in improving Central Nervous System (CNS) symptoms in patients with Ataxia Teleangectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).
- Conditions
- Nervous System Disorder, Genetic Syndrome
- Being tested
- Dexamethasone
- Keywords
- Ataxia Teleangiectasia, AT, ERY-DEX, Dexamethasone, Dexamethasone sodium phosphate
- Medical subject headings
- Nervous System Diseases, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexamethasoneDrug
Also called Dexamethasone sodium phosphate, Dex 21P
Dexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient).
The treatment has to be repeated at intervals of 30 days (±10 days)
| Study group | Role | What they receive |
|---|---|---|
| ERY-DEX | Experimental | Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes) Receives: Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population
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.
Measured: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population
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.
Measured: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
Secondary outcomes (7)
Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population
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.
Measured: At visit 7 (final visit, day 180)
Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population
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.
Measured: At visit 7 (final visit, day 180)
Mean Change From V4 to V7 in Ocular Motility - ITT Population
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).
Measured: At visit 7 (final visit, day 180)
Mean Change From V4 to V7 in Ocular Motility - PP Population
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).
Measured: At visit 7 (final visit, day 180)
Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population
VABS aggregate scale is used.
VABS is divided into 4 adaptive domains and 11 subdomains:
- Communication: Receptive, Expressive, Written;
- Daily Living Skills: Personal, Domestic, Community;
- Socialisation: Interpersonal Relationships, Play \& Leisure Time, Coping Skills;
- 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)
Measured: At visits 4 (90 days) and 7 (180 days, final visit)
Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population
VABS aggregate scale is used.
VABS is divided into 4 adaptive domains and 11 subdomains:
- Communication: Receptive, Expressive, Written;
- Daily Living Skills: Personal, Domestic, Community;
- Socialisation: Interpersonal Relationships, Play \& Leisure Time, Coping Skills;
- 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)
Measured: At visits 4 (90 days) and 7 (180 days, final visit)
Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)
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
Measured: Throughout the study, until the end of 6 months of treatment (day 180)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2010 (estimated)
- Study startFeb 2011
- Primary completionSep 2011
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Nervous System DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Quince Therapeutics S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Luciana Chessa, MD, Principal investigator, A.O. Sant'Andrea Rome Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01255358
- Sponsor's protocol ID
- IEDAT-01
- Other IDs
- 2010-022315-19
- Acronym
- IEDAT-01
- Status last verified
- Jul 2024
- First submitted
- 3 Dec 2010
- Last updated
- 10 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients
- ClinicalTrials.gov ID
- NCT01255358
- Acronym / sponsor ID
- IEDAT-01 / IEDAT-01
- Conditions
- Nervous System Disorder, Genetic Syndrome
- Keywords
- Ataxia Teleangiectasia, AT, ERY-DEX, Dexamethasone, Dexamethasone sodium phosphate
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Quince Therapeutics S.p.A.Industry (pharma, biotech, devices)
- Study start
- Feb 2011 (actual)
- Primary completion
- Sep 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 7 Dec 2010 (estimated)
- Results
- Posted 10 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).
Detailed description
This is a multi-centre, single arm, open label, 6 months, phase II study to evaluate the effect of ERY-DEX in improving Central Nervous System (CNS) symptoms in patients with Ataxia Teleangectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ERY-DEX | Experimental | Drug: DexamethasonePatients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes) |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- neurological signs of AT
- patients in autonomous gait or helped by a support
- proven molecular diagnosis of AT
- Males and females aged > 3 years
- Body weight >15 kg
Key exclusion criteria (7 listed)
- Current or previous neoplastic disease
- History of severe impairment of the immunological system
- Chronic conditions representing a contraindication to the use of steroid drugs
- Non compliance with the study request
- Any previous steroid assumption within 30 days before starting ERY-DEX
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DexamethasoneAlso: Dexamethasone sodium phosphate, Dex 21PDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). The treatment has to be repeated at intervals of 30 days (±10 days) | Drug | ERY-DEX |
Primary outcome
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population
Time frame: 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.
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population
Time frame: 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.
7 secondary outcomes
Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population
Time frame: At visit 7 (final visit, day 180)
Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population
Time frame: At visit 7 (final visit, day 180)
Mean Change From V4 to V7 in Ocular Motility - ITT Population
Time frame: At visit 7 (final visit, day 180)
Mean Change From V4 to V7 in Ocular Motility - PP Population
Time frame: At visit 7 (final visit, day 180)
Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population
Time frame: At visits 4 (90 days) and 7 (180 days, final visit)
Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population
Time frame: At visits 4 (90 days) and 7 (180 days, final visit)
Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)
Time frame: Throughout the study, until the end of 6 months of treatment (day 180)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Luciana Chessa, MD principal investigator
A.O. Sant'Andrea Rome Italy
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dexamethasone in people with Nervous System Disorder and Genetic Syndrome, run by Quince Therapeutics S.p.A. at 2 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population (time frame: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Oct 2024.