Ataxia TelangiectasiaPhase 3OLE_NEATNCT06664853
Open-Label Extension of EryDex Study IEDAT-04-2022
A Phase 3 study testing Dexamethasone sodium phosphate in people with Ataxia Telangiectasia, run by Quince Therapeutics S.p.A. at 21 sites in 9 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Ataxia Telangiectasia
- What is being tested?
- Dexamethasone sodium phosphate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 101 participants
- Where
- 21 sitesin 9 countries
- Study dates
- 11 Dec 2024to 30 Jan 2026
- Phase
- Phase 3
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- body weight ≥15 kg
- participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments
Who cannot join
- safety contraindications for continuation of treatment, as determined by the investigator
- clinically significant immune impairment that, in the opinion of the Investigator, precludes further treatment with corticosteroids
- Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.
- requiring treatment with a systemic corticosteroid
Trial locations and contacts
21 sites in 9 countries.
21 sites in 9 countries
United States5 sites
The Johns Hopkins Hospital, Division of pediatric allergy and immunology
Baltimore, Maryland, 21289
Cincinnati Children's Hospital, Division of neurology
Cincinnati, Ohio, 45229
UT Health Houston, Department of pediatrics, division of child & adolescent neurology
Houston, Texas, 77030
University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
Los Angeles, California, 90095
Saint Peter's University Hospital, Inc.
New Brunswick, New Jersey, 08901
United Kingdom4 sites
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B152GW
Great Ormond Street Hospital for Children, Zayed Centre for Research
London, United Kingdom, WC1N 1DZ
St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection
London, SE1 7EH
Nottingham Children's Hospital, Queen's Medical Center, Children's neurology
Nottingham, United Kingdom, NG7 2UH
Spain3 sites
Hospital Universitari Vall d'Hebron, Department of pediatric neurology
Barcelona, Spain, 08035
Hospital Universitario La Paz, Department of pediatric neurology
Madrid, Spain, 28046
Universitary Hospital Virgen del Rocío
Seville, 41013
Germany2 sites
University Hospital Frankfurt, Pediatric and Adolescent Clinic
Frankfurt, Germany, 60590
IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
Frankfurt am Main, Hesse, 60596
Italy2 sites
Spedali Civili di Brescia, Pediatric immunology department
Brescia, Italy, 25213
Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
Roma, Italy, 00161
Poland2 sites
MedPolonia sp zoo
Poznan, 60-693
Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic
Warsaw, 04-736
Denmark1 site
Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
Copenhagen, Denmark, 2100
Norway1 site
Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening
Oslo, Norway, 0372
Switzerland1 site
University Children's Hospital Zürich - Eleonore Foundation
Zurich, CH 8008
About this study
Official title: An Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)
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).
Read the detailed description
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.
- Conditions
- Ataxia Telangiectasia
- Being tested
- Dexamethasone sodium phosphate
- Medical subject headings
- Ataxia Telangiectasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dexamethasone sodium phosphateDrug
Dexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Dexamethasone sodium phosphate | Experimental | intravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) Receives: Drug: Dexamethasone sodium phosphate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events
Number of participants presenting at least one treatment emergent adverse event (TEAE)
Measured: 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
Measured: 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)
Measured: through study completion, up to approximately 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2024
- Study start11 Dec 2024
- Primary completion30 Jan 2026
- Study completion30 Jan 2026
Why it stopped: eDSP development discontinued
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 Ataxia TelangiectasiaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 101 participants (actual)
Sponsor and investigators
- Lead sponsor
- Quince Therapeutics S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Biotrial
- Responsible party
- Sponsor
- Investigators
- Dirk Thye, MD, Study director, Quince Therapeutics S.p.A.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06664853
- Sponsor's protocol ID
- IEDAT-05-2024
- Acronym
- OLE_NEAT
- Status last verified
- Apr 2026
- First submitted
- 25 Oct 2024
- Last updated
- 5 May 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)
- ClinicalTrials.gov ID
- NCT06664853
- Acronym / sponsor ID
- OLE_NEAT / IEDAT-05-2024
- Conditions
- Ataxia Telangiectasia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: eDSP development discontinued
- Enrolment
- 101 participants (actual)
- Lead sponsor
- Quince Therapeutics S.p.A.Industry (pharma, biotech, devices)
- Collaborators
- Biotrial
- Study start
- 11 Dec 2024 (actual)
- Primary completion
- 30 Jan 2026 (actual)
- Study completion
- 30 Jan 2026 (actual)
- First posted
- 30 Oct 2024 (actual)
- Results
- Posted 5 May 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dexamethasone sodium phosphate | Experimental | Drug: Dexamethasone sodium phosphateintravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- body weight ≥15 kg
- participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments
Key exclusion criteria (4 listed)
- safety contraindications for continuation of treatment, as determined by the investigator
- clinically significant immune impairment that, in the opinion of the Investigator, precludes further treatment with corticosteroids
- Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.
- requiring treatment with a systemic corticosteroid
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 |
|---|---|---|
| Dexamethasone sodium phosphateDexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion | Drug | Dexamethasone sodium phosphate |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events
Time frame: through study completion, up to approximately 13 months
Number of participants presenting at least one treatment emergent adverse event (TEAE)
Number of Participants With Treatment Emergent Adverse Events Leading to Intervention Discontinuation
Time frame: through study completion, up to approximately 13 months
Number of participants presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation
Number of Participants With Serious Adverse Events
Time frame: through study completion, up to approximately 13 months
Number of participants presenting at least one serious adverse event (SAE)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
- 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.
Dirk Thye, MD study director
Quince Therapeutics S.p.A.
21 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dexamethasone sodium phosphate in people with Ataxia Telangiectasia, run by Quince Therapeutics S.p.A. at 21 sites in 9 countries.
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 2 inclusion and 4 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: stopped early, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, United Kingdom, Spain, Germany, Italy and 4 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 5 May 2026). 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?
Number of Participants With Treatment Emergent Adverse Events (time frame: through study completion, up to approximately 13 months). The study lists 3 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 5 May 2026.