Trial Outcomes & Findings for Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI (NCT NCT02437253)
NCT ID: NCT02437253
Last Updated: 2017-09-15
Results Overview
Children's Health Questionnaire - Parent Form 50 bodily pain (BP) standardized score for participants \< 18 years of age. Range is from 0 to 100 (no units) and standardization if determined by comparison to healthy age matched children. Lower values mean increased pain. The difference of change in BP standardized score from day 0 to week 16 during adalimumab treatment minus change in BP standardized score from day 0 to week 16 during placebo treatment is reported.
COMPLETED
PHASE1/PHASE2
2 participants
day 0 to week 16 of treatment with adalimumab versus placebo
2017-09-15
Participant Flow
Participant milestones
| Measure |
Adalimumab First, Then Placebo
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week.
|
Placebo First, Then Adalimumab
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
|
Overall Study
COMPLETED
|
1
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
Baseline characteristics by cohort
| Measure |
Adalimumab First, Then Placebo
n=1 Participants
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
|
Placebo First, Then Adalimumab
n=1 Participants
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
15 years
n=5 Participants
|
6 years
n=7 Participants
|
10.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: day 0 to week 16 of treatment with adalimumab versus placeboPopulation: This was a cross-over study so within-individual changes are reported.
Children's Health Questionnaire - Parent Form 50 bodily pain (BP) standardized score for participants \< 18 years of age. Range is from 0 to 100 (no units) and standardization if determined by comparison to healthy age matched children. Lower values mean increased pain. The difference of change in BP standardized score from day 0 to week 16 during adalimumab treatment minus change in BP standardized score from day 0 to week 16 during placebo treatment is reported.
Outcome measures
| Measure |
Adalimumab First, Then Placebo
n=1 Participants
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
|
Placebo First, Then Adalimumab
n=1 Participants
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
|
|---|---|---|
|
Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score
|
1.4 units on a scale
Interval 1.4 to 1.4
|
1.0 units on a scale
Interval 1.0 to 1.0
|
SECONDARY outcome
Timeframe: Day 0 to week 16 of treatment with adalimumab versus placeboChildren's Health Questionnaire - Parent Form 50 physical function (PF) standardized score for participants \< 18 years of age. Range is from 0 to 100 (no units) and standardization if determined by comparison to healthy age matched children. Lower values mean decreased physical function. The difference of change in PF standardized score from day 0 to week 16 during adalimumab treatment minus change in PF standardized score from day 0 to week 16 during placebo treatment is reported.
Outcome measures
| Measure |
Adalimumab First, Then Placebo
n=1 Participants
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
|
Placebo First, Then Adalimumab
n=1 Participants
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
|
|---|---|---|
|
Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score
|
0 units on a scale
Interval 0.0 to 0.0
|
0.4 units on a scale
Interval 0.4 to 0.4
|
SECONDARY outcome
Timeframe: Day 0 to week 16 of treatment with adalimumab versus placeboPercent of participants with a \>10 mm improvement in either "how you feel now" or "worst pain you had this week" on the VA in the PPQ during adalimumab versus during placebo by either parental or subject report. Range is 0-100 mm; lower number means less pain.
Outcome measures
| Measure |
Adalimumab First, Then Placebo
n=1 Participants
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
|
Placebo First, Then Adalimumab
n=1 Participants
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
|
|---|---|---|
|
Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Day 0 to week 16 of treatment with adalimumab versus placeboNumber of joints with a \>5 degree more positive change during 16 weeks of adalimumab versus 16 weeks of placebo. A total of eight joints were measured.
Outcome measures
| Measure |
Adalimumab First, Then Placebo
n=1 Participants
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
|
Placebo First, Then Adalimumab
n=1 Participants
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
|
|---|---|---|
|
Range of Motion - Bilateral Shoulder, Elbow, Hip, Knee
|
7 joints
|
5 joints
|
SECONDARY outcome
Timeframe: Day 0 to week 16 of treatment with adalimumab versus placeboPopulation: No participants had anti-ERT antibodies
Anti-laronidase antibodies for subjects with MPS I. Anti-idursulfase antibodies for subjects with MPS II.
Outcome measures
| Measure |
Adalimumab First, Then Placebo
n=1 Participants
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
|
Placebo First, Then Adalimumab
n=1 Participants
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.
|
|---|---|---|
|
Anti-ERT Antibodies
|
0 Participants
|
0 Participants
|
Adverse Events
Adalimumab
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Adalimumab
n=2 participants at risk
20 mg subQ every other week (weight 15 to \<30 kg) 40 mg subQ every other week (weight ≥30 kg).
Adalimumab: Subjects will be treated with adalimumab (20 mg \[weight 15-\<30 kg\] or 40 mg \[weight ≥30 kg\] administered subcutaneously \[subQ\] every other week) or placebo for 16 weeks, then cross-over to the other group for 16 weeks.
|
Placebo
n=2 participants at risk
Saline placebo injection
Placebo: Saline placebo
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
1/2 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
50.0%
1/2 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
|
Psychiatric disorders
Mood lability
|
0.00%
0/2 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
|
Infections and infestations
Upper Respiratory Infection
|
0.00%
0/2 • 1 year
|
100.0%
2/2 • Number of events 2 • 1 year
|
|
Nervous system disorders
Headache
|
0.00%
0/2 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
|
Infections and infestations
Sinusitis
|
0.00%
0/2 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Injection site erythema
|
50.0%
1/2 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
Additional Information
Dr. Lynda Polgreen (PI)
Los Angeles Biomedical Research Institute at Harbor-UCLA
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place