Trial Outcomes & Findings for An Open-Label Safety Study of Memantine in Pediatric Patients With Autism, Asperger's Disorder, or Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS) (NCT NCT01592786)
NCT ID: NCT01592786
Last Updated: 2014-08-07
Results Overview
A confirmed SRS responder was defined as a patient who had at least 12 weeks of exposure to memantine, and a ≥ 10-point reduction in the SRS total raw score relative to baseline at 2 consecutive visits separated by at least 2 weeks. The SRS is a 65-item, caregiver-rated assessment scale that measures observable items on social behavior and social language use, as well as characteristics of autism in a naturalistic social setting. Each item is rated on a scale from 0 (never true) to 3 (almost always true). The SRS total raw score ranges from 0 to 195; a higher score indicates greater severity of social impairment.
COMPLETED
PHASE2
906 participants
Visit 1 (Baseline) to Visit 8 (week 48/Final Visit)
2014-08-07
Participant Flow
Patient recruitment occurred over an eleven month period, from June of 2012 to May of 2013, at 118 study sites, located in the United States and 17 other countries.
Enrolled patients went through a 2-week screening period.
Participant milestones
| Measure |
Memantine Hydrochloride (HCl)
Memantine Hydrochloride (HCl) extended-release 3-mg capsules once daily oral administration. Dosing was 3-mg, 6-mg, 9-mg, 12-mg, or 15-mg per day, based upon patient weight.
|
|---|---|
|
Overall Study
STARTED
|
903
|
|
Overall Study
COMPLETED
|
765
|
|
Overall Study
NOT COMPLETED
|
138
|
Reasons for withdrawal
| Measure |
Memantine Hydrochloride (HCl)
Memantine Hydrochloride (HCl) extended-release 3-mg capsules once daily oral administration. Dosing was 3-mg, 6-mg, 9-mg, 12-mg, or 15-mg per day, based upon patient weight.
|
|---|---|
|
Overall Study
Adverse Event
|
60
|
|
Overall Study
Lack of Efficacy
|
19
|
|
Overall Study
Protocol Violation
|
14
|
|
Overall Study
Withdrawal by Subject
|
23
|
|
Overall Study
Lost to Follow-up
|
19
|
|
Overall Study
Other
|
3
|
Baseline Characteristics
An Open-Label Safety Study of Memantine in Pediatric Patients With Autism, Asperger's Disorder, or Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS)
Baseline characteristics by cohort
| Measure |
Memantine Hydrochloride (HCl)
n=903 Participants
Memantine Hydrochloride (HCl) extended-release 3-mg capsules once daily, oral administration. Dosing was 3-mg, 6-mg, 9-mg, 12-mg, or 15-mg per day, based upon patient weight.
|
|---|---|
|
Age, Continuous
|
9.0 Years
STANDARD_DEVIATION 1.9 • n=5 Participants
|
|
Sex/Gender, Customized
Male
|
759 participants
n=5 Participants
|
|
Sex/Gender, Customized
Female
|
144 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
768 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
54 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
54 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
2 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
3 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
22 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
102 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
801 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
712 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
0 participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
11 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Colombia
|
8 participants
n=5 Participants
|
|
Region of Enrollment
Estonia
|
6 participants
n=5 Participants
|
|
Region of Enrollment
France
|
14 participants
n=5 Participants
|
|
Region of Enrollment
Hungary
|
20 participants
n=5 Participants
|
|
Region of Enrollment
Iceland
|
5 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Korea, Republic of
|
25 participants
n=5 Participants
|
|
Region of Enrollment
New Zealand
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
37 participants
n=5 Participants
|
|
Region of Enrollment
Serbia
|
21 participants
n=5 Participants
|
|
Region of Enrollment
Singapore
|
0 participants
n=5 Participants
|
|
Region of Enrollment
South Africa
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
17 participants
n=5 Participants
|
|
Region of Enrollment
Ukraine
|
16 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Visit 1 (Baseline) to Visit 8 (week 48/Final Visit)Population: Of the 906 patients who enrolled in the study 903 received at least 1 dose of open-label treatment to comprise the Safety Population. The Intent to Treat (ITT) population included the 868 patients in the Safety population who also had at least 1 post-Visit 1 assessment of the SRS total raw score.
A confirmed SRS responder was defined as a patient who had at least 12 weeks of exposure to memantine, and a ≥ 10-point reduction in the SRS total raw score relative to baseline at 2 consecutive visits separated by at least 2 weeks. The SRS is a 65-item, caregiver-rated assessment scale that measures observable items on social behavior and social language use, as well as characteristics of autism in a naturalistic social setting. Each item is rated on a scale from 0 (never true) to 3 (almost always true). The SRS total raw score ranges from 0 to 195; a higher score indicates greater severity of social impairment.
Outcome measures
| Measure |
Memantine Hydrochloride (HCl)
n=868 Participants
Memantine Hydrochloride (HCl) extended-release 3-mg capsules once daily, oral administration. Dosing was 3-mg, 6-mg, 9-mg, 12-mg or 15-mg per day, based upon patient weight.
|
|---|---|
|
Number of Confirmed Social Responsiveness Scale (SRS) Responders
Responders
|
517 participants
|
|
Number of Confirmed Social Responsiveness Scale (SRS) Responders
Non responders
|
351 participants
|
Adverse Events
Memantine Hydrochloride (HCl)
Serious adverse events
| Measure |
Memantine Hydrochloride (HCl)
n=903 participants at risk
Memantine Hydrochloride (HCl) extended-release 3-mg capsules, oral administration. Dosing was 3-mg, 6-mg, 9-mg, 12-mg or 15-mg, once per day, based upon patient weight.
|
|---|---|
|
Psychiatric disorders
Abnormal behaviour
|
0.22%
2/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
|
Injury, poisoning and procedural complications
Accidental exposure
|
0.11%
1/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
|
Gastrointestinal disorders
Constipation
|
0.11%
1/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
|
Psychiatric disorders
Disinhibition
|
0.11%
1/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
|
Gastrointestinal disorders
Gastroenteritis
|
0.11%
1/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
Other adverse events
| Measure |
Memantine Hydrochloride (HCl)
n=903 participants at risk
Memantine Hydrochloride (HCl) extended-release 3-mg capsules, oral administration. Dosing was 3-mg, 6-mg, 9-mg, 12-mg or 15-mg, once per day, based upon patient weight.
|
|---|---|
|
General disorders
Pyrexia
|
5.8%
52/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
|
General disorders
Irritability
|
5.4%
49/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
|
Infections and infestations
Nasopharyngitis
|
6.3%
57/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
|
Nervous system disorders
Headache
|
8.0%
72/903 • Adverse event data was collected over a 14 month period from June 2012 to August 2013 at 118 study sites in the US and 17 other countries.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee All data generated in this study are the property of Forest Research Institute, Inc. An integrated clinical and statistical report will be prepared at the completion of the study. Publication of the results by the Investigator will be subject to mutual agreement between the Investigator and Forest Research Institute, Inc.
- Publication restrictions are in place
Restriction type: OTHER