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TerminatedNCT03563053OLE-IEDATUpdated Oct 8, 2024Results posted

Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study

A Phase 3 interventional study of EryDex System in Ataxia Telangiectasia and Genetic Syndrome, sponsored by Quince Therapeutics S.p.A.. Terminated at 17 sites in 11 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2024-10-08.

Sponsored by Quince Therapeutics S.p.A. · Phase 3, Interventional, and Treatment

Why this study was terminated
terminated by sponsor
Phase
Phase 3
Study type
Interventional
Enrollment
104
Allocation
Not applicable
Ages
6 Years and older
Sex
All
01

Study summary

Primary Objective

To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.

Secondary Objective

To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).

Exploratory Objective:

To evaluate the long-term effect of EDS-EP in treating central nervous system (CNS) symptoms, as measured by the "Modified" International Cooperative Ataxia Rating Scale (mICARS), and Clinical Global Impression of severity and change (CGI-S/C).

Read the detailed description

This was an international (North America, Europe, Africa, Asia and Australia), multi-center, prospective, open-label treatment study, designed to continue to provide the study medication to all patients who completed 12 months of treatment (including those treated with placebo) in the ATTeST-IEDAT-02-2015 trial, completed the study assessments, do not present safety contraindication to continuation of treatment, and provided informed consent.

The study aimed to collect information on the long-term safety and efficacy of the trial treatment.

Patients meeting all selection criteria received monthly infusions of EDS-EP (dose range of \~14-22 mg DSP/infusion). If this dose of EDS-EP was not tolerated, the patient was discontinued from the study.

During the study, long-term efficacy assessments were performed every 6 months, while safety parameters were assessed at each monthly visit. The Schedule of Visits and assessments for the first 12 months were replicated for the second year onwards for patients who continued EryDex treatment beyond 12 months.

The analysis of the EryDex long-term safety and tolerability was based on the occurrence of Treatment-Emergent Adverse Events (TEAEs), including Serious AEs and discontinuations due to AEs. The long-term effect was measured by the "Modified" International Cooperative Ataxia Rating Scale, (mICARS), Rescored mICARS, Clinical Global Impression of severity and change (CGI-S/C) and health-related Quality of Life (QoL; EQ-5D-5L scale).

The ICARS, EQ-5D-5L and the CGI-C / S were administered by a properly qualified rater identified at each site who performed the ratings on the efficacy measures. The ICARS rater remained blinded to other assessments and did not have access to the safety data. The CGI rater did not have access to the ICARS ratings or safety data but was able to refer to other scales in scoring the CGI.

All patients enrolled in this study have participated in Study ATTeST-IEDAT-02-2015, and there was no de novo enrollment of new patients.

EryDel S.p.A (now Quince Therapeutics) decided to discontinue the IEDAT-03-2018 study in India due to the COVID-19 pandemic. On 13 Apr 2022, after the last patient completed one year of EryDex treatment, EryDel S.p.A. communicated the end of the open label extension study IEDAT-03-2018 and the stop of the EryDex treatment for patients in the context of this study.

02

Conditions studied

  • Ataxia Telangiectasia
  • Genetic Syndrome

Keywords

  • Ataxia Telangiectasia
  • AT
  • EryDex system
  • Dexamethasone
  • Dexamethasone sodium phosphate
03

In context

Ataxia Telangiectasia

41 studies on the registry are indexed under Ataxia Telangiectasia; 6 are open to participants now.

This study's enrollment of 104 is above the median of 30 across 27 interventional studies indexed under Ataxia Telangiectasia.

Browse Ataxia Telangiectasia studies →

Lead sponsor

Quince Therapeutics S.p.A. is the lead sponsor of 10 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Patient completed the double-blind period in the ATTeST study and completed the final (Visit 15 / Month 12) Efficacy Assessments of ATTeST or discontinued the study during the COVID-19 pandemic.
  2. Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe events / serious adverse events.
  3. Body weight > 15 kg.
  4. The patient and his / her parent / caregiver (if below the age of consent), or a legal representative, provided written informed consent to participate. If consent was provided solely by the caregiver in accordance with local regulations, the patient also was asked to provide their assent to participate in the study.
  5. Patient did not present safety contraindication for continuation of treatment, as determined by the Principal Investigator (PI) according to the procedures described below.

Moreover, patients who were discontinued from the ATTeST study during the COVID-19 pandemic were eligible to receive the EryDex treatment in the IEDAT-03-2018 study, in the absence of safety contraindications to continuation of the treatment, and after signing the informed consent.

There were no de novo enrolled patients.

Exclusion criteria

Exclusion Criteria:

Patients who met one or more of the following criteria were not considered to be eligible to participate in the study:

General

  1. Females that were:

    1. Pregnant, or were breast-feeding (for EU countries only)
    2. Of childbearing potential, pregnant, or were breast-feeding (for US and Rest of World countries).

    Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, were eligible.

  2. A disability that may prevent the patient from completing all study requirements.

  3. Current participation in another clinical study with another investigational drug.

    Medical History and Current Status

  4. Cluster differential 4 positive (CD4+) lymphocytes count \< 400 / mm3 (for patients 6 years of age) or \< 150 / mm3 (for patients > 6 years). In presence of oral infections, like oral candidiasis, documented at the screening or recurrent as per medical history documentation, the limit increases to \< 200 / mm3 (for patients > 6 years).
  5. Current neoplastic disease.
  6. Severe impairment of the immunological system.
  7. Severe or unstable pulmonary disease.
  8. Uncontrolled diabetes. Patients with diabetes that had been stabilized (i.e., no hypoglycemic or hyperglycemic episodes in the past 3 months) were eligible.
  9. Any other severe, unstable, or serious disease or condition that in the Investigator's opinion would put the patient at risk for imminent life-threatening morbidity, need for hospitalization, or mortality.
  10. Eligibility of patients with abnormal laboratory test values were determined by the Investigator.
  11. Confirmed haemoglobinopathies, e.g., haemoglobin C disease, sickle cell anaemia, or thalassemia.
  12. Moderate or severe renal and / or hepatic impairment.

  13. Patients who experienced moderate / severe steroid side effects, or moderate / severe adverse events associated with the EryDex treatment administered in the ATTeST study.

    Prior / Concomitant Medication

  14. Requires treatment with an oral or parenteral steroid. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids were permitted.
  15. Requires any other concomitant medication prohibited by the protocol.
  16. Use of any drug that is a strong inducer / inhibitor of Cytochrome P450 3A4 (CYP3A4).
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
104 participants (actual)

Study arms

  • Experimental
    active drug

    \~14-22 mg dexamethasone sodium phosphate (DSP) for 12 months. The duration of EryDex treatment could be extended further and continue until patients eventually withdrew consent, or the Investigator decided to discontinue treatment based on a risk / benefit assessment.

    Combination Product: EryDex System

Interventions

  • Combination productEryDex System

    EryDex System was a combination product that was used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EDS) creating the EDS-end product which was infused into the patients. EryDex treatment consisted of a dose range correspondent to the ATTeST High Dose (obtained by loading 125 mg of DSP to the EryDex process and that, in the ATTeST, resulted in a mean of 17.4 ± 5.4 mg (mean ± standard deviation) of DSP infused to patients) administered via ex vivo encapsulation into EDS that were infused into the patient with A-T. The dose of EryDex treatment was selected to allow collection of long-term safety data on the dose of erythrocyte encapsulated DSP that was considered more effective among the two different doses that were employed in the ATTeST study, based on the analysis of the ATTeST blinded, Phase 3 study data.

06

What researchers measure

Primary outcomes

  1. Number of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study

    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.

    Time frame: From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months)

Secondary outcomes

  1. Change From Baseline in Quality of Life Using EQ-5D-5L Scale 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.

    Time frame: From Baseline (Visit 1- Day 0) to Month 36

  2. 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

    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.

    Time frame: From Baseline (Visit 1- Day 0) to Month 36

  3. 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

    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.

    Time frame: From Baseline (Visit 1- Day 0) to Month 36

  4. Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until 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.

    Time frame: From Baseline (Visit 1- Day 0) to Month 36

07

Results

Posted Oct 8, 2024
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.

Participant flow

Out of a total of 108 patients who completed the full treatment period (including placebo treatment) in the ATTeST - IEDAT-02 study, 104 patients were enrolled in the IEDAT-03-2018 study (OLE-IEDAT) and comprised the Total Set. These patients signed the ICF, completed the ATTeST study assessments, and did not present safety contraindication prior continuation with the EryDex treatment.

Participant flow — Overall Study
MilestoneActive Drug
Started104
Total set104
Safety set104
Full analysis set80
Month 1264
Month 2433
Month 3621
Month 487
Month 50.51
Completed0
Not completed104
Withdrew: Withdrawal by subject17
Withdrew: Due to the covid-19 pandemic12
Withdrew: Adverse event3
Withdrew: Physician decision1
Withdrew: Study terminated by sponsor71

Outcome measures

PrimaryNumber of Treatment-Emergent Adverse Event (TEAE), Treatment-emergent Serious Adverse Events (TESAE), and Adverse Events of Special Interest (AESI) Throughout the Study

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.

Time frame:
From Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months)
Reported as:
Number · 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
Number of eventsOverall Population (SAF)
Number of TEAEs1232
Number of Serious TEAEs19
Number of TEAE leading to Death0
Number of TEAE leading to Permanent Withdrawal of Treatment5
Adverse Events During COVID-19 Interruption - On Dose5
Adverse Events During COVID-19 Interruption - Off Dose3
Adverse Events During COVID-19 Interruption - Restart55
TEAE by Worst Intensity - Mild1028
TEAE by Worst Intensity - Moderate187
TEAE by Worst Intensity - Severe17
TEAE by Closest Relationship to Treatment - Probable283
TEAE by Closest Relationship to Treatment - Possible167
TEAE by Closest Relationship to Treatment - Unlikely178
TEAE by Closest Relationship to Treatment - Not related604
Number of AESI83
SecondaryChange From Baseline in Quality of Life Using EQ-5D-5L Scale 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.

Time frame:
From Baseline (Visit 1- Day 0) to Month 36
Reported as:
Mean · score on a scale
Change From Baseline in Quality of Life Using EQ-5D-5L Scale to Month 36
score on a scaleOverall Population (Total Set)
to Month 60.015 ± 0.1131
to Month 12-0.022 ± 0.1145
to Month 180.007 ± 0.1233
to Month 240.011 ± 0.1746
to Month 30-0.021 ± 0.1117
to Month 360.011 ± 0.1242
SecondaryNumber 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

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.

Time frame:
From Baseline (Visit 1- Day 0) to Month 36
Reported as:
Count of participants · Participants
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
ParticipantsOverall Population (Total Set)
Month 6 - Improved (Score 1-3)9
Month 6 - No change or Worsened (Score 4-7)68
Month 12 - Improved (Score 1-3)14
Month 12 - No change or Worsened (Score 4-7)45
Month 18 - Improved (Score 1-3)6
Month 18 - No change or Worsened (Score 4-7)41
Month 24 - Improved (Score 1-3)5
Month 24 - No change or Worsened (Score 4-7)26
Month 30 - Improved (Score 1-3)2
Month 30 - No change or Worsened (Score 4-7)17
Month 36 - Improved (Score 1-3)3
Month 36 - No change or Worsened (Score 4-7)16
SecondaryNumber 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

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.

Time frame:
From Baseline (Visit 1- Day 0) to Month 36
Reported as:
Count of participants · 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 From Baseline (Visit 1 - Day 0) to Month 36
ParticipantsOverall Population (Total Set)
Baseline - CGI-S Score - 02
Baseline - CGI-S Score - 118
Baseline - CGI-S Score - 231
Baseline - CGI-S Score - 323
Baseline - CGI-S Score - 40
Month 6 - CGI-S Score - 01
Month 6 - CGI-S Score - 120
Month 6 - CGI-S Score - 230
Month 6 - CGI-S Score - 322
Month 6 - CGI-S Score - 43
Month 12 - CGI-S Score - 00
Month 12 - CGI-S Score - 112
Month 12 - CGI-S Score - 228
Month 12 - CGI-S Score - 317
Month 12 - CGI-S Score - 42
Month 18 - CGI-S Score - 00
Month 18 - CGI-S Score - 113
Month 18 - CGI-S Score - 221
Month 18 - CGI-S Score - 312
Month 18 - CGI-S Score - 41
Month 24 - CGI-S Score - 00
Month 24 - CGI-S Score - 110
Month 24 - CGI-S Score - 210
Month 24 - CGI-S Score - 39
Month 24 - CGI-S Score - 42
Month 30 - CGI-S Score - 00
Month 30 - CGI-S Score - 15
Month 30 - CGI-S Score - 210
Month 30 - CGI-S Score - 34
Month 30 - CGI-S Score - 40
Month 36 - CGI-S Score - 00
Month 36 - CGI-S Score - 13
Month 36 - CGI-S Score - 210
Month 36 - CGI-S Score - 36
Month 36 - CGI-S Score - 40
SecondaryChange From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until 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.

Time frame:
From Baseline (Visit 1- Day 0) to Month 36
Reported as:
Mean · score on a scale
Change From Baseline of the Modified International Cooperative Ataxia Rating Scale (mICARS) Until Month 36
score on a scaleOverall Population (Total Set)
to Month 60.7 ± 4.09
to Month 121.5 ± 3.36
to Month 182.7 ± 4.28
to Month 243.9 ± 4.96
to Month 304.5 ± 5.41
to Month 3634.0 ± 5.17

Adverse events

Collected over TEAEs were all the AEs reported from Baseline (Visit 1 - Day 0) to Follow-up (~60 days after last infusion, i.e. up to 50.5 months).. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Overall Population (SAF)0/104 (0%)12/104 (11.5%)98/104 (94.2%)
Most frequent serious events
Showing 10 of 13
Most frequent serious events
EventOverall Population (SAF)
Forearm fractureInjury, poisoning and procedural complications2/104
Lower respiratory tract infectionInfections and infestations1/104
PneumoniaInfections and infestations1/104
Viral infectionInfections and infestations1/104
B-Cell limphomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)1/104
Hodgkin's DiseaseNeoplasms benign, malignant and unspecified (incl cysts and polyps)1/104
Odontogenic cystNeoplasms benign, malignant and unspecified (incl cysts and polyps)1/104
Trombocytopenic PurpuraBlood and lymphatic system disorders1/104
DysphagiaGastrointestinal disorders1/104
PyrexiaGeneral disorders1/104
Most frequent other events
Showing 10 of 26
Most frequent other events
EventOverall Population (SAF)
PyrexiaGeneral disorders33/104
Infusion Related ReactionInjury, poisoning and procedural complications28/104
Upper respiratory tract infectionInfections and infestations23/104
DiarrhoeaGastrointestinal disorders23/104
NasopharyngitisInfections and infestations21/104
VomitingGastrointestinal disorders20/104
CoughRespiratory, thoracic and mediastinal disorders19/104
Corona Virus InfectionInfections and infestations17/104
FatigueGeneral disorders16/104
Iron DeficiencyMetabolism and nutrition disorders16/104

Baseline characteristics

In this trial, the Total or Overall Set (N=104) numerically corresponds to the Safety set, which included all patients who provided informed consent or assent and who received any dose of study medication during ATTeST-IEDAT-03-2018 study (i.e., "Date performed" given on the "EDS-EP Infusion" eCRF page at any Visit).

Age, Categorical
Age, Categorical(Participants)Overall Population (Total Set)
<=18 years104
Between 18 and 65 years0
>=65 years0
Age, Continuous
Age, Continuous(years)Overall Population (Total Set)
Mean11.4 ± 4.57
Sex: Female, Male
Sex: Female, Male(Participants)Overall Population (Total Set)
Female49
Male55
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Overall Population (Total Set)
Hispanic or Latino5
Not Hispanic or Latino99
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Overall Population (Total Set)
American Indian or Alaska Native0
Asian21
Native Hawaiian or Other Pacific Islander0
Black or African American3
White80
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(participants)Overall Population (Total Set)
Belgium3
United States25
Poland10
Italy2
Australia2
Tunisia7
Germany13
India15
Spain11
Norway5
United Kingdom11
08

Study locations

17 sites
  • Ataxia Center and HD Center of Excellence - UCLA
    Los Angeles, California 90095, United States
  • Division of Pediatric Allergy and Immunology - Johns Hopkins Hospital
    Baltimore, Maryland 21287-3923, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Department of Pediatrics Division of Child and Adolescent Neurology Mitochondrial Clinic - University of Texas Medical School
    Houston, Texas 77030, United States
  • Department of Neurology Royal Children's Hospital
    Parkville, Victoria 3052, Australia
  • Laboratory of Pediatric Immunology UZ Leuven
    Leuven, 3000, Belgium
  • Klinik für Kinder- und Jugendmedizin, Allergologie, pneumologie und Mukoviszidose, Universitätsklinikum Frankfurt
    Frankfurt, 60590, Germany
  • Vijaya Health Centre, Department of Neurology
    Chennai, Tamil Nadu, India
  • National Institute of Mental Health and Neurosciences (NIMHANS) Department of Neurology
    Bangalore, 560 029, India
  • Nizam's Institute of Medical Sciences Department of Neurology
    Hyderabad, 500 082, India
  • Jaslok Hospital and Research Center Department of Pediatric Neurology
    Mumbai, 400 026, India
  • Dipartimento Neuroscienze umane e salute mentale, Policlinico Umberto I Università La Sapienza
    Roma, Italy
  • Norwegian National Unit for Newborn Screening, Division of Pediatric and Adolescent Medicine, Oslo University Hospital
    Oslo, Norway
  • Department of Clinical Immunology - The Children's Memorial Health Institute
    Warsaw, 04-730, Poland
  • Servicio de Neurolgia Pediatrica, Hospital Materno-Infantil La Paz
    Madrid, 28046, Spain
  • Razi Hospital, Clinical Investigation Center-Neuroscience
    Tunis, Tunisia
  • Nottingham University Hospitals NHS Trust - Queen's Medical Centre
    Nottingham, Nottinghamshire, United Kingdom
09

References and documents

Publications

  • Koenig MK, Leuzzi V, Gouider R, Yiu EM, Pietrucha B, Stray-Pedersen A, Perlman SL, Wu S, Burgers T, Borgohain R, Kandadai RM, Meyts I, Bucciol G, Udwadia-Hegde A, Yadav R, Roberts D, Dane A, Roden M, Thye D, Horn B, Lederman HM, Whitehouse WP. Long-term safety of dexamethasone sodium phosphate encapsulated in autologous erythrocytes in pediatric patients with ataxia telangiectasia. Front Neurol. 2025 Jan 23;15:1526914. doi: 10.3389/fneur.2024.1526914. eCollection 2024. PubMed 39917433 ↗

Study documents

  • Study protocol · Dec 1, 2020
  • Statistical analysis plan · Jan 25, 2023

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 8, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03563053
Lead sponsor
Quince Therapeutics S.p.A.
Responsible party
Sponsor
First posted
Jun 20, 2018
Start date
Jun 12, 2018
Primary completion
Sep 2, 2022
Completion
Sep 2, 2022
Results posted
Oct 8, 2024
Last update
Oct 8, 2024

Study contacts

Guenter R Janhofer, MD, PhD
study director · EryDel S.p.A.

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Sep 2024. You cannot join it, but the record below documents what was studied.

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Discussion

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