Phase 2
N=22
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
Nervous System Disorder · Genetic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01255358 ↗Enrolled (actual)
22
Serious AEs
9.1%
Results posted
Oct 2024
Primary outcome: Primary: Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population — -2.2; -3.4; -4.0 units on a scale — p=0.02
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Dexamethasone (Drug)
- Age
- Pediatric, Adult, Older Adult · 3+ yrs
- Sex
- All
- Sponsor
- Quince Therapeutics S.p.A.
- Primary completion
- Sep 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population |
-2.2; -3.4; -4.0 | 0.02 sig |
| PRIMARY Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population |
-2.4; -3.9; -5.2 | 0.0031 sig |
| SECONDARY Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population |
0.2 | — |
| SECONDARY Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population |
0.2 | — |
| SECONDARY Mean Change From V4 to V7 in Ocular Motility - ITT Population |
-0.3 | — |
| SECONDARY Mean Change From V4 to V7 in Ocular Motility - PP Population |
-0.3 | — |
| SECONDARY Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population |
1.0; 1.3 | <0.0001 sig |
| SECONDARY Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population |
1.1; 1.5 | <0.0001 sig |
| SECONDARY Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs) |
15; 2 | — |
Summary
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).
Eligibility Criteria
Inclusion Criteria
- neurological signs of AT
- patients in autonomous gait or helped by a support
- proven molecular diagnosis of AT
- Males and females aged > 3 years
- Body weight >15 kg
- Plasma levels of Lymphocytes CD4+/mm3 > 500 (for patients aged 3-6 years) or > 200 (older than 6 years)
- written IC to participate.
Exclusion Criteria
- Current or previous neoplastic disease
- History of severe impairment of the immunological system
- Chronic conditions representing a contraindication to the use of steroid drugs
- Non compliance with the study request
- Any previous steroid assumption within 30 days before starting ERY-DEX
- Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial
- Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods
Data sourced from ClinicalTrials.gov (NCT01255358). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.