Ataxia TelangiectasiaPhase 3NEATNCT06193200
Evaluate the Neurological Effects of EryDex on Subjects With A-T
A Phase 3 study testing Dexamethasone sodium phosphate in people with Ataxia Telangiectasia, run by Quince Therapeutics S.p.A. at 19 sites in 9 countries.
Trial summary
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… 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?
- Yes, some participants may receive a placebo
- How many people
- 105 participants
- Where
- 19 sitesin 9 countries
- Study dates
- 24 Jun 2024to 17 Dec 2025
- 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
- Clinical diagnosis of A-T
- In autonomous gait or is helped by periodic use of a support
- Genetic confirmation of A-T
- Body weight ≥15 kg
Who cannot join
- Participation in another clinical study
- Immune impairment
- History of severe impairment of the immunological system
- Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years
- Severe or unstable pulmonary disease
- Uncontrolled diabetes
Show all 9Show fewer
- Current chronic or acute significant renal and/or hepatic impairment
- Any previous oral or parenteral steroid use within 6 weeks before Baseline. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted
- A disability that may prevent the subject from completing all study requirements
Trial locations and contacts
19 sites in 9 countries.
19 sites in 9 countries
United States4 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
United Kingdom4 sites
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B152GW
St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection
London, SW17 0RE
Great Ormond Street Hospital for Children, Zayed Centre for Research
London, WC1N 1DZ
Nottingham Children's Hospital, Queen's Medical Center, Children's neurology
Nottingham, NG7 2UH
Germany2 sites
University Hospital Frankfurt, Pediatric and Adolescent Clinic
Frankfurt, 60590
IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
Frankfurt, 60596
Italy2 sites
Spedali Civili di Brescia, Pediatric immunology department
Brescia, 25123
Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
Roma, 00161
Poland2 sites
MedPolonia sp zoo
Poznan, 60-693
Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic
Warsaw, 04-736
Spain2 sites
Hospital Universitari Vall d'Hebron, Department of pediatric neurology
Barcelona, 08035
Hospital Universitario La Paz, Department of pediatric neurology
Madrid, 28046
Denmark1 site
Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
Copenhagen, 2100
Norway1 site
Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening
Oslo, 0372
Switzerland1 site
University Children's Hospital Zürich - Eleonore Foundation
Zurich, CH-8008
About this study
Official title: A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT)
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).
Read the detailed description
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.
- Conditions
- Ataxia Telangiectasia
- Being tested
- Dexamethasone sodium phosphate, Placebo
- Keywords
- Ataxia Telangiectasia, A-T, Louis-Bar syndrome, Cerebello-oculocutaneous telangiectasia
- 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
Also called DSP
Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion
PlaceboOther
Placebo encapsulated in autologous erythrocytes and administered via IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Dexamethasone sodium phosphate | Experimental | IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) Receives: Drug: Dexamethasone sodium phosphate |
| Placebo | Placebo comparator | IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS) Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to Visit 9 (approximately 6 months)
Secondary outcomes (2)
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.
Measured: Baseline to Visit 9 (approximately 6 months)
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
Measured: Baseline to Visit 9 (approximately 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2024
- Study start24 Jun 2024
- Primary completion17 Dec 2025
- Study completion17 Dec 2025
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 105 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
- NCT06193200
- Sponsor's protocol ID
- IEDAT-04-2022
- Acronym
- NEAT
- Status last verified
- Mar 2026
- First submitted
- 11 Dec 2023
- Last updated
- 30 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Russo S, Zielen S, Pietrucha B, Trischler J, Perlman S, Zielonka D, Del Mar Garcia Romero M, Ip W, Stray-Pedersen A, Borresen ML, Gomez-Andres D, Lissauer S, Pachlopnik-Schmid J, Karampatsas K, Wu S, Pisani F, Lederman H, Badolato R, Kern J, Kornmann O, Donath H, Whitehouse WP, Koenig MK, Abolhassani H, Schaefer C, Roden M, Thye D, Smith A, Horn B, Prayle A. Neurological effects of encapsulated dexamethasone sodium phosphate in children aged 6-9 years with ataxia telangiectasia (NEAT): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Child Adolesc Health. 2026 Aug 14:S2352-4642(26)00125-2. doi: 10.1016/S2352-4642(26)00125-2. Online ahead of print.
PubMed 42600624
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT)
- ClinicalTrials.gov ID
- NCT06193200
- Acronym / sponsor ID
- NEAT / IEDAT-04-2022
- Conditions
- Ataxia Telangiectasia
- Keywords
- Ataxia Telangiectasia, A-T, Louis-Bar syndrome, Cerebello-oculocutaneous telangiectasia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 105 participants (actual)
- Lead sponsor
- Quince Therapeutics S.p.A.Industry (pharma, biotech, devices)
- Collaborators
- Biotrial
- Study start
- 24 Jun 2024 (actual)
- Primary completion
- 17 Dec 2025 (actual)
- Study completion
- 17 Dec 2025 (actual)
- First posted
- 5 Jan 2024 (actual)
- Results
- Posted 30 Apr 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, 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).
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dexamethasone sodium phosphate | Experimental | Drug: Dexamethasone sodium phosphateIV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) |
| Placebo | Placebo comparator | Other: PlaceboIV infusion of placebo 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 (4 listed)
- Clinical diagnosis of A-T
- In autonomous gait or is helped by periodic use of a support
- Genetic confirmation of A-T
- Body weight ≥15 kg
Key exclusion criteria (9 listed)
- Participation in another clinical study
- Immune impairment
- History of severe impairment of the immunological system
- Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years
- Severe or unstable pulmonary disease
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 phosphateAlso: DSPDexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion | Drug | Dexamethasone sodium phosphate |
| PlaceboPlacebo encapsulated in autologous erythrocytes and administered via IV infusion | Other | Placebo |
Primary outcome
Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS)
Time frame: Baseline to Visit 9 (approximately 6 months)
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.
2 secondary outcomes
Clinical Global Impression of Severity (CGI-S)
Time frame: Baseline to Visit 9 (approximately 6 months)
Clinical Global Impression of Change (CGI-C)
Time frame: Baseline to Visit 9 (approximately 6 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42600624Russo S, Zielen S, Pietrucha B, Trischler J, Perlman S, Zielonka D, Del Mar Garcia Romero M, Ip W, Stray-Pedersen A, Borresen ML, Gomez-Andres D, Lissauer S, Pachlopnik-Schmid J, Karampatsas K, Wu S, Pisani F, Lederman H, Badolato R, Kern J, Kornmann O, Donath H, Whitehouse WP, Koenig MK, Abolhassani H, Schaefer C, Roden M, Thye D, Smith A, Horn B, Prayle A. Neurological effects of encapsulated dexamethasone sodium phosphate in children aged 6-9 years with ataxia telangiectasia (NEAT): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Child Adolesc Health. 2026 Aug 14:S2352-4642(26)00125-2. doi: 10.1016/S2352-4642(26)00125-2. Online ahead of print.
- 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.
19 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 19 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 4 inclusion and 9 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 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, United Kingdom, Germany, Italy, Poland 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. Some participants in this study may receive a placebo. 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 30 Apr 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?
Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) (time frame: Baseline to Visit 9 (approximately 6 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06193200, which also lists 1 linked publication.
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 30 Apr 2026.