Trial Outcomes & Findings for Use of ACTIMMUNE in Patients With ADO2 (NCT NCT02584608)
NCT ID: NCT02584608
Last Updated: 2021-01-08
Results Overview
Evaluate for changes in bone resorption markers including CTX, NTX/creatinine ratio between baseline and 14 weeks
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
baseline, 14 weeks
Results posted on
2021-01-08
Participant Flow
Participant milestones
| Measure |
Treatment
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
ACTIMMUNE
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Total matches.
Baseline characteristics by cohort
| Measure |
Treatment
n=12 Participants
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
ACTIMMUNE
|
|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
Total
|
30.48 years
n=5 Participants • Total matches.
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: baseline, 14 weeksPopulation: All subjects
Evaluate for changes in bone resorption markers including CTX, NTX/creatinine ratio between baseline and 14 weeks
Outcome measures
| Measure |
Treatment
n=12 Participants
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
ACTIMMUNE
|
|---|---|
|
Changes in Bone Resorption Markers From Baseline to 14 Weeks.
Percent change of CTX
|
2.2 percentage of change
Interval -35.7 to 90.5
|
|
Changes in Bone Resorption Markers From Baseline to 14 Weeks.
Percent change of NTX/creatinine ratio
|
-2.1 percentage of change
Interval -50.5 to 55.5
|
SECONDARY outcome
Timeframe: 6-12 weeksPopulation: all subjects
Evaluate for changes in bone turnover markers including TRAP5b, NTX, CTX/TRAP5b ratio after 6-12 weeks of treatment.
Outcome measures
| Measure |
Treatment
n=12 Participants
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
ACTIMMUNE
|
|---|---|
|
Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment
TRAP5B
|
-15.34 percentage of change
Interval -57.07 to 13.1
|
|
Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment
NTX
|
-2.09 percentage of change
Interval -50.51 to 55.54
|
|
Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment
CTX/TRAP5b ratio
|
26.31 percentage of change
Interval -35.91 to 144.57
|
Adverse Events
Treatment
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment
n=12 participants at risk
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
ACTIMMUNE
|
|---|---|
|
Nervous system disorders
Headache
|
83.3%
10/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Nervous system disorders
Fatigue
|
75.0%
9/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Gastrointestinal disorders
nausea
|
75.0%
9/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Product Issues
flu like symptoms
|
66.7%
8/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Musculoskeletal and connective tissue disorders
myalgia
|
58.3%
7/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Blood and lymphatic system disorders
white blood cell decreased
|
50.0%
6/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
41.7%
5/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Gastrointestinal disorders
diarrhea
|
41.7%
5/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Blood and lymphatic system disorders
neutrophil count decreased
|
41.7%
5/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Gastrointestinal disorders
vomiting
|
41.7%
5/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Musculoskeletal and connective tissue disorders
back pain
|
33.3%
4/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Skin and subcutaneous tissue disorders
injection site reaction
|
33.3%
4/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Musculoskeletal and connective tissue disorders
pain in extremity
|
33.3%
4/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Musculoskeletal and connective tissue disorders
bone pain
|
33.3%
4/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Immune system disorders
chills
|
25.0%
3/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Investigations
CPK increased
|
25.0%
3/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Endocrine disorders
hypertriglycerides
|
25.0%
3/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Blood and lymphatic system disorders
platelet count decrease
|
25.0%
3/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Blood and lymphatic system disorders
anemia
|
16.7%
2/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Gastrointestinal disorders
constipation
|
16.7%
2/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Nervous system disorders
dizziness
|
16.7%
2/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Psychiatric disorders
agitation
|
8.3%
1/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Blood and lymphatic system disorders
aspartate Aminotransferase increased
|
8.3%
1/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Musculoskeletal and connective tissue disorders
chest wall pain
|
8.3%
1/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
8.3%
1/12 • From signing consent till final followup at 18 weeks (4 weeks after stopping study drug)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place