Trial Outcomes & Findings for Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I (NCT NCT02232477)
NCT ID: NCT02232477
Last Updated: 2023-10-31
Results Overview
Mean intra-subject change in total recall score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit. The Hopkins Verbal Learning Test measures verbal memory. The subject is read a list of words and asked to memorize as many words as possible. The scale ranges from 0 to 36, with higher scores indicating a better outcome. Two relevant time points: Baseline and the subject's final visit. Because this study ended prematurely for due to the pandemic, the subject's final visit was at the 12-month time point for one subject, 24-months for one subject, 36-months for two subjects, and 48-months for two subjects. Calculation: The total recall score at Baseline was subtracted from the total recall score at the subject's final visit and mean change and standard deviation were reported.
TERMINATED
NA
6 participants
zero to 48 months
2023-10-31
Participant Flow
Participant milestones
| Measure |
Open-label Treatment
laronidase 1.74 mg IT q 3 months for five years
Intrathecal recombinant human alpha iduronidase: Intrathecal recombinant human alpha iduronidase every 3 months
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Baseline characteristics by cohort
| Measure |
Open-label Treatment
n=6 Participants
laronidase 1.74 mg IT q 3 months for five years
Intrathecal recombinant human alpha iduronidase: Intrathecal recombinant human alpha iduronidase every 3 months
|
|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
19.33 years
STANDARD_DEVIATION 3.25 • n=99 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
5 Participants
n=99 Participants
|
|
Region of Enrollment
Canada
|
1 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: zero to 48 monthsMean intra-subject change in total recall score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit. The Hopkins Verbal Learning Test measures verbal memory. The subject is read a list of words and asked to memorize as many words as possible. The scale ranges from 0 to 36, with higher scores indicating a better outcome. Two relevant time points: Baseline and the subject's final visit. Because this study ended prematurely for due to the pandemic, the subject's final visit was at the 12-month time point for one subject, 24-months for one subject, 36-months for two subjects, and 48-months for two subjects. Calculation: The total recall score at Baseline was subtracted from the total recall score at the subject's final visit and mean change and standard deviation were reported.
Outcome measures
| Measure |
Open-label Treatment
n=6 Participants
laronidase 1.74 mg IT q 3 months for five years
Intrathecal recombinant human alpha iduronidase: Intrathecal recombinant human alpha iduronidase every 3 months
|
|---|---|
|
Hopkins Verbal Learning Test
|
-1.67 score on a scale
Standard Deviation 2.58
|
Adverse Events
Open-label Treatment
Serious adverse events
| Measure |
Open-label Treatment
n=6 participants at risk
laronidase 1.74 mg IT q 3 months for five years
Intrathecal recombinant human alpha iduronidase: Intrathecal recombinant human alpha iduronidase every 3 months
|
|---|---|
|
Nervous system disorders
headache
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
pneumonia
|
33.3%
2/6 • Number of events 4 • 5 years, 6 months
|
|
Eye disorders
corneal transplant
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Nervous system disorders
spinal cord injury
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Psychiatric disorders
psychiatric symptoms
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Nervous system disorders
hydrocephalus
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Renal and urinary disorders
urinary tract infection
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Nervous system disorders
VP shunt infection
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Gastrointestinal disorders
small bowel obstruction
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
Other adverse events
| Measure |
Open-label Treatment
n=6 participants at risk
laronidase 1.74 mg IT q 3 months for five years
Intrathecal recombinant human alpha iduronidase: Intrathecal recombinant human alpha iduronidase every 3 months
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
General disorders
aphthous ulcer
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Renal and urinary disorders
urinary incontinence
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
sleep apnea
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory infection
|
16.7%
1/6 • Number of events 2 • 5 years, 6 months
|
|
Nervous system disorders
Headache
|
33.3%
2/6 • Number of events 3 • 5 years, 6 months
|
|
Cardiac disorders
mitral valve disease
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Psychiatric disorders
anxiety
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Musculoskeletal and connective tissue disorders
back pain
|
66.7%
4/6 • Number of events 18 • 5 years, 6 months
|
|
Ear and labyrinth disorders
otitis media
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Musculoskeletal and connective tissue disorders
stress fracture
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Eye disorders
glaucoma
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Eye disorders
corneal transplant
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
|
Eye disorders
blurred vision
|
16.7%
1/6 • Number of events 1 • 5 years, 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place