Trial Outcomes & Findings for Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy (NCT NCT00159250)
NCT ID: NCT00159250
Last Updated: 2019-12-05
Results Overview
Number of Subjects with Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
7 participants
Primary outcome timeframe
Baseline up to Day 120
Results posted on
2019-12-05
Participant Flow
Participant milestones
| Measure |
Low Dose AVI-4658
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
5
|
|
Overall Study
COMPLETED
|
2
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Low Dose AVI-4658
n=2 Participants
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 Participants
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
Total
n=7 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=2 Participants
|
5 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=2 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=2 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Age, Continuous
|
14.5 years
n=2 Participants
|
11.8 years
n=5 Participants
|
13.1 years
n=7 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=2 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=2 Participants
|
5 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
United Kingdom
|
2 participants
n=2 Participants
|
5 participants
n=5 Participants
|
7 participants
n=7 Participants
|
|
Mobility
Wheelchair for 11 years
|
1 Participants
n=2 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
|
Mobility
Wheelchair for 10 years; rides static bike for 10
|
1 Participants
n=2 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
|
Mobility
Wheelchair for 10 years
|
0 Participants
n=2 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
|
Mobility
Walks indoors
|
0 Participants
n=2 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
|
Mobility
Walks unaided
|
0 Participants
n=2 Participants
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Day 120Number of Subjects with Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Outcome measures
| Measure |
Low Dose AVI-4658
n=2 Participants
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 Participants
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Number of Participants With Adverse Events Related to AVI-4568
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: From the Day of Screening to Day 3Outcome measures
| Measure |
Low Dose AVI-4658
n=2 Participants
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 Participants
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Number of Participants With Injection Site Reactions
|
2 Participants
|
4 Participants
|
PRIMARY outcome
Timeframe: From the Day of Screening up to Day 28Assessed by light microscopy and immunocytochemistry to detect the differences in inflammatory infiltrates between the AVI-4568 and placebo-treated EDB muscles
Outcome measures
| Measure |
Low Dose AVI-4658
n=2 Participants
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 Participants
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Number of Subjects With Clinically Significant Change From Baseline in Laboratory Values
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 14 to Day 28Induced Skipping of Exon 51 in the Treated Extensor Digitorum Brevis (EDB) Muscle Determined by Reverse Transcription Polymerase Chain Reaction was assessed by Sequencing of the RT-PCR products
Outcome measures
| Measure |
Low Dose AVI-4658
n=2 Participants
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 Participants
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Number of Participants With Induced Skipping of Exon 51 in the Treated Extensor Digitorum Brevis (EDB) Muscle Determined by Reverse Transcription Polymerase Chain Reaction
|
2 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Day 14 to Day 28Outcome measures
| Measure |
Low Dose AVI-4658
n=2 Participants
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 Participants
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Number of Participants With Restoration of Dystrophin Protein Expression Measured by Immunocytochemistry
|
0 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Day 14 to Day 28Outcome measures
| Measure |
Low Dose AVI-4658
n=2 Participants
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 Participants
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Number of Participants With Restoration of Dystrophin Protein Expression Measured by Western Blot Analysis
|
0 Participants
|
5 Participants
|
Adverse Events
Low Dose AVI-4658
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
High Dose AVI-4658
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Low Dose AVI-4658
n=2 participants at risk
Low dose of AVI-4658
0.09 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
High Dose AVI-4658
n=5 participants at risk
High dose of AVI-4658
0.9 mg doses were diluted in 900 μL normal saline (0·9%) and injected to the extensor digitorum brevis (EDB) muscle
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Bilateral erythema
|
100.0%
2/2 • Number of events 2 • 120 days
|
20.0%
1/5 • Number of events 1 • 120 days
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
50.0%
1/2 • Number of events 1 • 120 days
|
60.0%
3/5 • Number of events 4 • 120 days
|
|
Immune system disorders
Myoglobinuria
|
0.00%
0/2 • 120 days
|
60.0%
3/5 • Number of events 3 • 120 days
|
|
Skin and subcutaneous tissue disorders
Bilateral oedema
|
50.0%
1/2 • Number of events 1 • 120 days
|
0.00%
0/5 • 120 days
|
|
Cardiac disorders
Decline in cardiac function
|
50.0%
1/2 • Number of events 1 • 120 days
|
0.00%
0/5 • 120 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place