Ataxia Telangiectasia
Conditions
Keywords
Ataxia Telangiectasia, A-T, Louis-Bar syndrome, Cerebello-oculocutaneous telangiectasia
Brief 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 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.
Interventions
DRUGDexamethasone sodium phosphate
Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion
OTHERPlacebo
Placebo encapsulated in autologous erythrocytes and administered via IV infusion
Sponsors
Quince Therapeutics S.p.A.
Biotrial
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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 * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Global Impression of Severity (CGI-S) | Baseline to Visit 9 (approximately 6 months) | 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. |
| Clinical Global Impression of Change (CGI-C) | Baseline to Visit 9 (approximately 6 months) | 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 |
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
Participants were randomized (1:1) to eDSP or placebo group via an independent Interactive Web Response System and were stratified by age (6-9 years or ≥10 years), gender, and region (USA vs. other countries). 105 study participants were randomized and 105 received at least one study treatment.
Pre-assignment details
51 were randomized to the eDSP group and 54 were randomized to the placebo group. The primary efficacy analysis was conducted in the ITT (6-9) population, which consisted of 83 6-9-year-old participants, 40 of whom were assigned to the eDSP group and 43 in the placebo group. Per protocol population comprised 39 (98%) of 40 participants in the eDSP group and 40 (93%) participants in the placebo group. All 105 randomized participants were included in the safety population.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 54 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 | 2 Participants |
| Race (NIH/OMB) Black or African American | 2 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 | 5 Participants |
| Race (NIH/OMB) White | 88 Participants |
| Region of Enrollment Denmark | 1 Participants |
| Region of Enrollment Germany | 12 Participants |
| Region of Enrollment Italy | 3 Participants |
| Region of Enrollment Norway | 2 Participants |
| Region of Enrollment Poland | 5 Participants |
| Region of Enrollment Spain | 6 Participants |
| Region of Enrollment Switzerland | 3 Participants |
| Region of Enrollment United Kingdom | 16 Participants |
| Region of Enrollment United States | 13 Participants |
| Rescored modified version of the International Cooperative Ataxia Rating Scale (RmICARS) | 9.2 rmICARS Score STANDARD_DEVIATION 5.46 |
| Sex: Female, Male Female | 24 Participants |
| Sex: Female, Male Male | 55 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 51 | 0 / 54 |
| other Total, other adverse events | 47 / 51 | 50 / 54 |
| serious Total, serious adverse events | 2 / 51 | 3 / 54 |
Outcome results
None listed