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| Name | Class |
|---|---|
| Biotrial | INDUSTRY |
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This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangiectasia (A-T).
The EryDex System (EDS) is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) which is infused into the patient.
In the placebo arm, the subjects will receive autologous erythrocytes prepared with the EDS process using a placebo solution.
Upon completion of all screening assessments for eligibility, subjects meeting all selection criteria at baseline will be randomized in a 1:1 fashion to EryDex or placebo. Approximately 86 subjects 6- to 9-years-old, approximately 43 per group, will be randomized. Approximately 20 subjects 10 years of age and above, 10 per treatment group, may also be enrolled.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dexamethasone sodium phosphate | Experimental | IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) |
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| Placebo | Placebo Comparator | IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dexamethasone sodium phosphate | Drug | Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion |
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| Measure | Description | Time Frame |
|---|---|---|
| Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) | Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old). RmICARS (International Cooperative Ataxia Rating Scale): Score (range 0-29); higher score indicates worse disease. | Baseline to Visit 9 (approximately 6 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Global Impression of Severity (CGI-S) | CGI-S (Clinical Global Impression of Severity): 7-point scale; higher score indicates worse disease. 1 = Normal, not at all ill; 7 = Among the most extremely ill. Percentage of participants improving in CGI-S from baseline to Visit 9. | Baseline to Visit 9 (approximately 6 months) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Dirk Thye, MD | Quince Therapeutics S.p.A. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence | Los Angeles | California | 90095 | United States | ||
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51 were randomized to the eDSP group and 54 were randomized to the placebo group. The primary efficacy analysis was conducted in the ITT (6-9) population, which consisted of 83 6-9-year-old participants, 40 of whom were assigned to the eDSP group and 43 in the placebo group. Per protocol population comprised 39 (98%) of 40 participants in the eDSP group and 40 (93%) participants in the placebo group. All 105 randomized participants were included in the safety population.
Participants were randomized (1:1) to eDSP or placebo group via an independent Interactive Web Response System and were stratified by age (6-9 years or ≥10 years), gender, and region (USA vs. other countries).
105 study participants were randomized and 105 received at least one study treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Dexamethasone Sodium Phosphate | IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using EDS (formerly known as "EryDex System") |
| FG001 | Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 22, 2025 | Feb 25, 2026 |
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| Placebo | Other | Placebo encapsulated in autologous erythrocytes and administered via IV infusion |
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| Clinical Global Impression of Change (CGI-C) |
CGI-C (Clinical Global Impression of Change): 7-point scale; higher score indicates worsening of disease. 1 = Very much improved; 4 = No change; 7 = Very much worse. Percentage of participants improving in CGI-C at Visit 9 |
| Baseline to Visit 9 (approximately 6 months) |
| The Johns Hopkins Hospital, Division of pediatric allergy and immunology |
| Baltimore |
| Maryland |
| 21289 |
| United States |
| Cincinnati Children's Hospital, Division of neurology | Cincinnati | Ohio | 45229 | United States |
| UT Health Houston, Department of pediatrics, division of child & adolescent neurology | Houston | Texas | 77030 | United States |
| Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology | Copenhagen | 2100 | Denmark |
| University Hospital Frankfurt, Pediatric and Adolescent Clinic | Frankfurt | 60590 | Germany |
| IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases | Frankfurt | 60596 | Germany |
| Spedali Civili di Brescia, Pediatric immunology department | Brescia | 25123 | Italy |
| Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health | Roma | 00161 | Italy |
| Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening | Oslo | 0372 | Norway |
| MedPolonia sp zoo | Poznan | 60-693 | Poland |
| Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic | Warsaw | 04-736 | Poland |
| Hospital Universitari Vall d'Hebron, Department of pediatric neurology | Barcelona | 08035 | Spain |
| Hospital Universitario La Paz, Department of pediatric neurology | Madrid | 28046 | Spain |
| University Children's Hospital Zürich - Eleonore Foundation | Zurich | CH-8008 | Switzerland |
| University Hospitals Birmingham NHS Foundation Trust | Birmingham | B152GW | United Kingdom |
| St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection | London | SW17 0RE | United Kingdom |
| Great Ormond Street Hospital for Children, Zayed Centre for Research | London | WC1N 1DZ | United Kingdom |
| Nottingham Children's Hospital, Queen's Medical Center, Children's neurology | Nottingham | NG7 2UH | United Kingdom |
IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS)
| COMPLETED |
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| NOT COMPLETED |
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The ITT population consisted of 105 participants, 51 of whom were assigned to the eDSP group and 54 in the placebo group. Efficacy endpoints were analyzed in the efficacy population, 6-9-year-olds (N=83), while the safety population included all 105 enrolled participants who received at least one dose of treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Dexamethasone Sodium Phosphate | IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using EDS (formerly known as the "EryDex System") |
| BG001 | Placebo | IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly known as "EryDex System") |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | The ITT population consisted of 105 participants, 51 of whom were assigned to the eDSP group and 54 in the placebo group. | Count of Participants | Participants |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | 105 represents safety population analyzed | Number | Participants |
| |||||||||||||||
| Rescored modified version of the International Cooperative Ataxia Rating Scale (RmICARS) | ICARS administered in its entirety but analysis is based on RmICARS in 6-9yrs (83 participants). The Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) is a 9-item, 29-point scale (0-29) derived from the 100-point ICARS by excluding the oculomotor domain and collapsing selected item scores to focus on axial cerebellar features (posture, gait, and speech), where higher scores indicate greater disease severity. | The ITT (6-9) population consisted of 83 6-9-year-old participants, safety population 105, primary efficacy endpoint RmICARS 6-9yrs | Mean | Standard Deviation | rmICARS Score |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) | Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old). RmICARS (International Cooperative Ataxia Rating Scale): Score (range 0-29); higher score indicates worse disease. | Primary analysis population was 6-9 years totaling 83 study participants. | Posted | Jan 2026 | Least Squares Mean | Standard Deviation | RmICARS score (0-29 points) | Baseline to Visit 9 (approximately 6 months) |
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| Secondary | Clinical Global Impression of Severity (CGI-S) | CGI-S (Clinical Global Impression of Severity): 7-point scale; higher score indicates worse disease. 1 = Normal, not at all ill; 7 = Among the most extremely ill. Percentage of participants improving in CGI-S from baseline to Visit 9. | Posted | Count of Participants | Participants | Baseline to Visit 9 (approximately 6 months) |
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| Secondary | Clinical Global Impression of Change (CGI-C) | CGI-C (Clinical Global Impression of Change): 7-point scale; higher score indicates worsening of disease. 1 = Very much improved; 4 = No change; 7 = Very much worse. Percentage of participants improving in CGI-C at Visit 9 | Posted | Count of Participants | Participants | Baseline to Visit 9 (approximately 6 months) |
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Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dexamethasone Sodium Phosphate | IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using EDS (formerly known as the "EryDex System") | 0 | 51 | 2 | 51 | 47 | 51 |
| EG001 | Placebo | IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly known as "EryDex System") | 0 | 54 | 3 | 54 | 50 | 54 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Dystonia | Nervous system disorders | Systematic Assessment |
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| Esophageal varices hemorrhage | Gastrointestinal disorders | Systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | Systematic Assessment |
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| Diplopia | Eye disorders | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
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| Pyrexia | General disorders | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Fatigue | General disorders | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
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| Rhinorrhoea | Infections and infestations | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
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| Rhinitis | Infections and infestations | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | Systematic Assessment |
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| Bronchitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Iron deficiency | Metabolism and nutrition disorders | Systematic Assessment |
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| Blood culture positive | Investigations | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | Quince Therapeutics | +1 415 910 5717 | clinops@quincetx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 14, 2025 | Feb 26, 2026 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D001260 | Ataxia Telangiectasia |
| ID | Term |
|---|---|
| D020754 | Spinocerebellar Ataxias |
| D002524 | Cerebellar Ataxia |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020752 | Neurocutaneous Syndromes |
| D001259 | Ataxia |
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D013684 | Telangiectasis |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000081207 | Primary Immunodeficiency Diseases |
| D049914 | DNA Repair-Deficiency Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C004180 | dexamethasone 21-phosphate |
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| Between 18 and 65 years |
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| >=65 years |
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| Denmark |
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| Germany |
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| Italy |
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| Norway |
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| Poland |
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| Spain |
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| Switzerland |
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| United Kingdom |
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