Ataxia Telangiectasia
Conditions
Brief 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).
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.
Interventions
DRUGDexamethasone sodium phosphate
Dexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion
Sponsors
Quince Therapeutics S.p.A.
Biotrial
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* body weight ≥15 kg * participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events | 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 | 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 | through study completion, up to approximately 13 months | Number of participants presenting at least one serious adverse event (SAE) |
Countries
Denmark, Germany, Italy, Norway, Poland, Spain, Switzerland, United Kingdom, United States
Contacts
STUDY_DIRECTORDirk Thye, MD
Quince Therapeutics S.p.A.
Participant flow
Recruitment details
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).
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 101 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 6 Participants |
| Race (NIH/OMB) Black or African American | 5 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race (NIH/OMB) White | 86 Participants |
| Sex: Female, Male Female | 48 Participants |
| Sex: Female, Male Male | 53 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 101 |
| other Total, other adverse events | 71 / 101 |
| serious Total, serious adverse events | 5 / 101 |
Outcome results
None listed