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eDSP development discontinued
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| Name | Class |
|---|---|
| Biotrial | INDUSTRY |
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This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied the neurological effects of EryDex on subjects with ataxia telangiectasia (NEAT trial).
The IEDAT-05-2024 study aims to provide EryDex (dexamethasone sodium phosphate encapsulated into autologous erythrocytes) treatment to all participants in the IEDAT-04-2022 (NEAT) study, who complete the study assessments, do not -have safety contraindications to continuation of treatment, and who provide informed consent. The open-label extension (OLE) treatment period will be 12 months. Participants will be considered to have completed the study when Visit 14 (Safety Follow-up) has been performed.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dexamethasone sodium phosphate | Experimental | intravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dexamethasone sodium phosphate | Drug | Dexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events | Number of participants presenting at least one treatment emergent adverse event (TEAE) | through study completion, up to approximately 13 months |
| Number of Participants With Treatment Emergent Adverse Events Leading to Intervention Discontinuation | Number of participants presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation | through study completion, up to approximately 13 months |
| Number of Participants With Serious Adverse Events | Number of participants presenting at least one serious adverse event (SAE) | through study completion, up to approximately 13 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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102 participants completed the full treatment period in the IEDAT-04-2022 study (NEAT), 101 NEAT study participants subsequently consented and enrolled in the open-label extension study IEDAT-05-2024 study (NEAT-OLE).
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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") |
| 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_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 10, 2025 |
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| The Johns Hopkins Hospital, Division of pediatric allergy and immunology |
| Baltimore |
| Maryland |
| 21289 |
| United States |
| Saint Peter's University Hospital, Inc. | New Brunswick | New Jersey | 08901 | 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 | Denmark | 2100 | Denmark |
| University Hospital Frankfurt, Pediatric and Adolescent Clinic | Frankfurt | Germany | 60590 | Germany |
| IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases | Frankfurt am Main | Hesse | 60596 | Germany |
| Spedali Civili di Brescia, Pediatric immunology department | Brescia | Italy | 25213 | Italy |
| Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health | Roma | Italy | 00161 | Italy |
| Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening | Oslo | Norway | 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 | Spain | 08035 | Spain |
| Hospital Universitario La Paz, Department of pediatric neurology | Madrid | Spain | 28046 | Spain |
| Universitary Hospital Virgen del RocÃo | Seville | 41013 | Spain |
| University Children's Hospital Zürich - Eleonore Foundation | Zurich | CH 8008 | Switzerland |
| Great Ormond Street Hospital for Children, Zayed Centre for Research | London | United Kingdom | WC1N 1DZ | United Kingdom |
| Nottingham Children's Hospital, Queen's Medical Center, Children's neurology | Nottingham | United Kingdom | NG7 2UH | United Kingdom |
| 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 | SE1 7EH | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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The total set (N=101) included all patients who provided informed consent or assent as documented by a date of informed consent or date of assent.
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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 "EryDex System") |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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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 | Number of Participants With Treatment Emergent Adverse Events | Number of participants presenting at least one treatment emergent adverse event (TEAE) | The analysis population contains all participants who received at least one study drug infusion in this open label extension study | Posted | Count of Participants | Participants | through study completion, up to approximately 13 months |
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| ||||||||||||||||||||||||||
| Primary | Number of Participants With Treatment Emergent Adverse Events Leading to Intervention Discontinuation | Number of participants presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation | Posted | Count of Participants | Participants | through study completion, up to approximately 13 months |
|
| ||||||||||||||||||||||||||||
| Primary | Number of Participants With Serious Adverse Events | Number of participants presenting at least one serious adverse event (SAE) | Posted | Count of Participants | Participants | through study completion, up to approximately 13 months |
|
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From first dose through study completion approximately 13 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 "EryDex System") | 0 | 101 | 5 | 101 | 71 | 101 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Gastrostomy tube site complication | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Influenza A | Infections and infestations | Systematic Assessment |
| ||
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Lymphadenopathy in nasopharynx | General disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pruritus | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Erythema | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastroenteritis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Viral infection | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Rhinitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | Quince Therapeutics | +1 415 910 5717 | clinops@quincetx.com |
| Apr 15, 2026 |
| Prot_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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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
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| Title | Denominators | Categories |
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