Trial Outcomes & Findings for Evaluate the Neurological Effects of EryDex on Subjects With A-T (NCT NCT06193200)
NCT ID: NCT06193200
Last Updated: 2026-04-30
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
105 participants
Primary outcome timeframe
Baseline to Visit 9 (approximately 6 months)
Results posted on
2026-04-30
Participant Flow
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.
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.
Participant milestones
| Measure |
Dexamethasone Sodium Phosphate
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using EDS (formerly known as "EryDex System")
|
Placebo
IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS)
|
|---|---|---|
|
Overall Study
STARTED
|
51
|
54
|
|
Overall Study
COMPLETED
|
49
|
52
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
105 represents safety population analyzed
Baseline characteristics by cohort
| Measure |
Dexamethasone Sodium Phosphate
n=51 Participants
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using EDS (formerly known as the "EryDex System")
|
Placebo
n=54 Participants
IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly known as "EryDex System")
|
Total
n=105 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
51 Participants
n=51 Participants
|
54 Participants
n=54 Participants
|
105 Participants
n=105 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=51 Participants
|
0 Participants
n=54 Participants
|
0 Participants
n=105 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=51 Participants
|
0 Participants
n=54 Participants
|
0 Participants
n=105 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=51 Participants
|
26 Participants
n=54 Participants
|
50 Participants
n=105 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=51 Participants
|
28 Participants
n=54 Participants
|
55 Participants
n=105 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=51 Participants
|
0 Participants
n=54 Participants
|
0 Participants
n=105 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=51 Participants
|
2 Participants
n=54 Participants
|
6 Participants
n=105 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=51 Participants
|
0 Participants
n=54 Participants
|
0 Participants
n=105 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=51 Participants
|
2 Participants
n=54 Participants
|
6 Participants
n=105 Participants
|
|
Race (NIH/OMB)
White
|
41 Participants
n=51 Participants
|
47 Participants
n=54 Participants
|
88 Participants
n=105 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=51 Participants
|
0 Participants
n=54 Participants
|
0 Participants
n=105 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=51 Participants
|
3 Participants
n=54 Participants
|
5 Participants
n=105 Participants
|
|
Region of Enrollment
United States
|
13 Participants
n=51 Participants • 105 represents safety population analyzed
|
15 Participants
n=54 Participants • 105 represents safety population analyzed
|
28 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
Denmark
|
1 Participants
n=51 Participants • 105 represents safety population analyzed
|
1 Participants
n=54 Participants • 105 represents safety population analyzed
|
2 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
Germany
|
10 Participants
n=51 Participants • 105 represents safety population analyzed
|
12 Participants
n=54 Participants • 105 represents safety population analyzed
|
22 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
Italy
|
2 Participants
n=51 Participants • 105 represents safety population analyzed
|
1 Participants
n=54 Participants • 105 represents safety population analyzed
|
3 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
Norway
|
3 Participants
n=51 Participants • 105 represents safety population analyzed
|
2 Participants
n=54 Participants • 105 represents safety population analyzed
|
5 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
Poland
|
5 Participants
n=51 Participants • 105 represents safety population analyzed
|
9 Participants
n=54 Participants • 105 represents safety population analyzed
|
14 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
Spain
|
6 Participants
n=51 Participants • 105 represents safety population analyzed
|
6 Participants
n=54 Participants • 105 represents safety population analyzed
|
12 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
Switzerland
|
1 Participants
n=51 Participants • 105 represents safety population analyzed
|
2 Participants
n=54 Participants • 105 represents safety population analyzed
|
3 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Region of Enrollment
United Kingdom
|
10 Participants
n=51 Participants • 105 represents safety population analyzed
|
6 Participants
n=54 Participants • 105 represents safety population analyzed
|
16 Participants
n=105 Participants • 105 represents safety population analyzed
|
|
Rescored modified version of the International Cooperative Ataxia Rating Scale (RmICARS)
|
9.8 rmICARS Score
STANDARD_DEVIATION 5.25 • n=40 Participants • The ITT (6-9) population consisted of 83 6-9-year-old participants, safety population 105, primary efficacy endpoint RmICARS 6-9yrs
|
8.6 rmICARS Score
STANDARD_DEVIATION 5.66 • n=43 Participants • The ITT (6-9) population consisted of 83 6-9-year-old participants, safety population 105, primary efficacy endpoint RmICARS 6-9yrs
|
9.2 rmICARS Score
STANDARD_DEVIATION 5.46 • n=83 Participants • The ITT (6-9) population consisted of 83 6-9-year-old participants, safety population 105, primary efficacy endpoint RmICARS 6-9yrs
|
PRIMARY outcome
Timeframe: Baseline to Visit 9 (approximately 6 months)Population: Primary analysis population was 6-9 years totaling 83 study participants.
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.
Outcome measures
| Measure |
Placebo
n=43 Participants
IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly referred to as the "EryDex System")
|
Dexamethasone Sodium Phosphate
n=40 Participants
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EDS (formerly referred to "EryDex System")
|
|---|---|---|
|
Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS)
|
2.24 RmICARS score (0-29 points)
Standard Deviation 3.42
|
0.94 RmICARS score (0-29 points)
Standard Deviation 4.13
|
SECONDARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Placebo
n=43 Participants
IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly referred to as the "EryDex System")
|
Dexamethasone Sodium Phosphate
n=40 Participants
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EDS (formerly referred to "EryDex System")
|
|---|---|---|
|
Clinical Global Impression of Severity (CGI-S)
|
7 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: 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
Outcome measures
| Measure |
Placebo
n=43 Participants
IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly referred to as the "EryDex System")
|
Dexamethasone Sodium Phosphate
n=40 Participants
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EDS (formerly referred to "EryDex System")
|
|---|---|---|
|
Clinical Global Impression of Change (CGI-C)
|
6 Participants
|
13 Participants
|
Adverse Events
Dexamethasone Sodium Phosphate
Serious events: 2 serious events
Other events: 47 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 50 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Dexamethasone Sodium Phosphate
n=51 participants at risk
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using EDS (formerly known as the "EryDex System")
|
Placebo
n=54 participants at risk
IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly known as "EryDex System")
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Bronchiectasis
|
0.00%
0/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
1.9%
1/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Nervous system disorders
Dystonia
|
0.00%
0/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
1.9%
1/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Gastrointestinal disorders
Esophageal varices hemorrhage
|
0.00%
0/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
1.9%
1/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Infections and infestations
Lower respiratory tract infection
|
2.0%
1/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
0.00%
0/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Eye disorders
Diplopia
|
2.0%
1/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
0.00%
0/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
Other adverse events
| Measure |
Dexamethasone Sodium Phosphate
n=51 participants at risk
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using EDS (formerly known as the "EryDex System")
|
Placebo
n=54 participants at risk
IV infusion of placebo encapsulated in autologous erythrocytes using EDS (formerly known as "EryDex System")
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
11.1%
6/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
2.0%
1/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
9.3%
5/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Metabolism and nutrition disorders
Iron deficiency
|
7.8%
4/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
3.7%
2/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Nervous system disorders
Headache
|
15.7%
8/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
9.3%
5/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
General disorders
Fatigue
|
13.7%
7/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
9.3%
5/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Investigations
Blood culture positive
|
7.8%
4/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
11.1%
6/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Infections and infestations
Upper respiratory tract infection
|
11.8%
6/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
7.4%
4/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Infections and infestations
Rhinorrhoea
|
9.8%
5/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
7.4%
4/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Gastrointestinal disorders
Abdominal pain
|
7.8%
4/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
7.4%
4/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Infections and infestations
Rhinitis
|
3.9%
2/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
9.3%
5/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Gastrointestinal disorders
Vomiting
|
21.6%
11/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
24.1%
13/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
General disorders
Pyrexia
|
23.5%
12/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
13.0%
7/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
27.5%
14/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
3.7%
2/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Infections and infestations
Nasopharyngitis
|
9.8%
5/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
20.4%
11/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.8%
5/51 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
16.7%
9/54 • Timeframe is from baseline (or first dose, as defined) to Visit 10 (approximately 6 months).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60