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An Open-Label Safety Study of Memantine in Pediatric Patients With Autism, Asperger's Disorder, or Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS)

Phase 2
Completed
Conditions
Autism
Asperger's Disorder
Autistic Disorder
Pediatric Autism
Asperger's
Autism Spectrum Disorder (ASD)
Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS)
Interventions
Registration Number
NCT01592786
Lead Sponsor
Forest Laboratories
Brief Summary

The purpose of this study is to evaluate the safety and tolerability of memantine in pediatric (6-12 years old) patients with autism, Asperger's Disorder, or Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS) and to identify responders for participation in a follow-up randomized withdrawal study.

Detailed Description

This 50-week multicenter and multinational clinical study is comprised of a 2-week screening period, a 6-week open-label dose-titration period followed by a variable duration maintenance period (up to 42 weeks).

Patients with at least 12 weeks of investigational product exposure who meet the protocol specified responder criterion at two consecutive visits separated by at least two weeks are eligible to transition to a randomized withdrawal study. A responder is defined as a patient who demonstrates at least a 10 point improvement (reduction in score) in the Social Responsiveness Scale (SRS) total raw score relative to the Visit 1 total raw score.

Weight based dose limits were selected in this study to ensure that exposure in terms of area under the curve (AUC) was less than the predefined limit of 2100 ng·h/mL which represents a 10-fold lower exposure than observed at the No observed adverse effect level (NOAEL) of 15 mg/kg/day in juvenile rats.

The weight-based dose limits in this study were as follows:

* Group A: ≥ 60 kg; maximum 15 mg/day

* Group B: 40-59 kg; maximum 9 mg/day

* Group C: 20-39 kg; maximum 6 mg/day

* Group D: \< 20 kg; maximum 3 mg/day

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
906
Inclusion Criteria
  1. Male or female outpatients.
  2. Age of 6-12.
  3. Meet Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, Text Revision (DSM-IV-TR) diagnostic criteria for autism, Asperger's Disorder, or Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS)
  4. Have a family that is sufficiently organized and stable to guarantee adequate safety monitoring and continuous attendance to clinic visits for the duration of the study
Exclusion Criteria
  1. Have enrolled in Study MEM-MD-57A
  2. Medical conditions that might interfere with the conduct of the study, confound interpretation of the study results, or endanger the patient's well-being.
  3. Participation in any other clinical investigation using an experimental drug within 30 days of screening.
  4. Having any primary psychiatric (Axis I) diagnosis other than autism, Asperger's Disorder, and PDD-NOS.
  5. Meeting DSM-IV-TR criteria for bipolar I disorder, psychotic disorder not otherwise specified, posttraumatic stress disorder, schizophrenia, or major depressive disorder within the past 6 months

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Memantine Hydrochloride (HCl)Memantine Hydrochloride (HCl)Once daily oral administration of open-label memantine for up to 48 weeks: 6-week dose-titration period followed by up to 42-week maintenance period.
Primary Outcome Measures
NameTimeMethod
Number of Confirmed Social Responsiveness Scale (SRS) RespondersVisit 1 (Baseline) to Visit 8 (week 48/Final Visit)

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.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (118)

Forest Investigative Site 054

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Glendale, California, United States

Forest Investigative Site 109

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Imperial, California, United States

Forest Investigative Site 068

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Dothan, Alabama, United States

Forest Investigative Site 026

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Santa Ana, California, United States

Forest Investigative Site 077

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Little Rock, Arkansas, United States

Forest Investigative Site 102

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Libertyville, Illinois, United States

Forest Investigative Site 023

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Naperville, Illinois, United States

Forest Investigative Site 177

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Camperdown, New South Wales, Australia

Forest Investigative Site 081

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Albuquerque, New Mexico, United States

Forest Investigative Site 002

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Stanford, California, United States

Forest Investigative Site 075

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Bradenton, Florida, United States

Forest Investigative Site 078

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Boulder, Colorado, United States

Forest Investigative Site 082

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Evansville, Indiana, United States

Forest Investigative Site 071

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Bothell, Washington, United States

Forest Investigative Site 123

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Fort Wayne, Indiana, United States

Forest Investigative Site 098

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Bronx, New York, United States

Forest Investigative Site 069

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Avon Lake, Ohio, United States

Forest Investigative Site 070

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The Woodlands, Texas, United States

Forest Investigative Site 051

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Houston, Texas, United States

Forest Investigative Site 113

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Norfolk, Virginia, United States

Forest Investigative Site 382

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Gyula, Hungary

Forest Investigative Site 379

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Budapest, Hungary

Forest Investigative Site 727

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Madrid, Spain

Forest Investigative Site 329

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Bron, Rhone, France

Forest Investigative Site 729

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Barcelona, Spain

Forest Investigative Site 055

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Tucson, Arizona, United States

Forest Investigative Site 115

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Orange City, Florida, United States

Forest Investigative Site 101

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Wellington, Florida, United States

Forest Investigative Site 086

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Rockville, Maryland, United States

Forest Investigative Site 095

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Lake Charles, Louisiana, United States

Forest Investigative Site 116

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Lincoln, Nebraska, United States

Forest Investigative Site 059

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Newton, Massachusetts, United States

Forest Investigative Site 108

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Springfield, Massachusetts, United States

Forest Investigative Site 127

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Toms River, New Jersey, United States

Forest Investigative Site 130

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Henderson, Nevada, United States

Forest Investigative Site 136

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Neptune, New Jersey, United States

Forest Investigative Site 090

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Memphis, Tennessee, United States

Forest Investigative Site 141

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Ogden, Utah, United States

Forest Investigative Site 028

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Clinton, Utah, United States

Forest Investigative Site 073

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Centennial, Colorado, United States

Forest Investigative Site 155

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Toronto, Ontario, Canada

Forest Investigative Site 204

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Brussel, Belgium

Forest Investigative Site 226

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Bogota, Colombia

Forest Investigative Site 203

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Bruxelles, Belgium

Forest Investigative Site 228

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Bello, Colombia

Forest Investigative Site 202

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Hoboken, Belgium

Forest Investigative Site 201

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Leuven, Belgium

Forest Investigative Site 276

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Tallinn, Estonia

Forest Investigative Site 381

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Budapest, Hungary

Forest Investigative Site 702

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Seoul, Korea, Republic of

Forest Investigative Site 703

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Seoul, Korea, Republic of

Forest Investigative Site 526

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Wellington, New Zealand

Forest Investigative Site 579

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Gdansk, Poland

Forest Investigative Site 578

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Gdansk, Poland

Forest Investigative Site 676

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Cape Town, Western Cape, South Africa

Forest Investigative Site 802

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Kherson, Vil. Stepanivka, Ukraine

Forest Investigative Site 803

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Donetsk, Ukraine

Forest Investigative Site 804

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Kyiv, Ukraine

Forest Investigative Site 132

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Johnstown, Pennsylvania, United States

Forest Investigative Site 100

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Media, Pennsylvania, United States

Forest Investigative Site 052

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Washington, District of Columbia, United States

Forest Investigative Site 080

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Gainesville, Florida, United States

Forest Investigative Site 117

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Jacksonville, Florida, United States

Forest Investigative Site 065

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Maitland, Florida, United States

Forest Investigative Site 096

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Los Angeles, California, United States

Forest Investigative Site 066

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Irvine, California, United States

Forest Investigative Site 074

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Owensboro, Kentucky, United States

Forest Investigative Site 001

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Columbus, Ohio, United States

Forest Investigative Site 107

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Albuquerque, New Mexico, United States

Forest Investigative Site 064

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Charlottesville, Virginia, United States

Forest Investigative Site 106

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Wichita, Kansas, United States

Forest Investigative Site 131

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McMurray, Pennsylvania, United States

Forest Investigative Site 053

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Gresham, Oregon, United States

Forest Investigative Site 092

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Tulsa, Oklahoma, United States

Forest Investigative Site 119

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Charleston, West Virginia, United States

Forest Investigative Site 063

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Middleton, Wisconsin, United States

Forest Investigative Site 453

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Roma, Italy

Forest Investigative Site 577

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Warszawa, Poland

Forest Investigative Site 576

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Kobierzyce, Poland

Forest Investigative Site 628

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Novi Sad, Serbia

Forest Investigative Site 728

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Sabadell, Barcelona, Spain

Forest Investigative Site 326

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Paris, France

Forest Investigative Site 378

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Budapest, Hungary

Forest Investigative Site 062

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Orlando, Florida, United States

Forest Investigative Site 061

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Louisville, Kentucky, United States

Forest Investigative Site 005

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Phoenix, Arizona, United States

Forest Investigative Site 021

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San Francisco, California, United States

Forest Investigative Site 125

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Orlando, Florida, United States

Forest Investigative Site 114

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Orlando, Florida, United States

Forest Investigative Site 084

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Indianapolis, Indiana, United States

Forest Investigative Site 118

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Miami, Florida, United States

Forest Investigative Site 124

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Orlando, Florida, United States

Forest Investigative Site 067

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Tampa, Florida, United States

Forest Investigative Site 056

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Indianapolis, Indiana, United States

Forest Investigative Site 104

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Las Vegas, Nevada, United States

Forest Investigative Site 019

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Oklahoma City, Oklahoma, United States

Forest Investigative Site 057

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Nashville, Tennessee, United States

Forest Investigative Site 029

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Salt Lake City, Utah, United States

Forest Investigative Site 085

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Oakland Park, Florida, United States

Forest Investigative Site 227

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Barranquilla, Colombia

Forest Investigative Site 380

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Szeged, Hungary

Forest Investigative Site 452

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Siena, Italy

Forest Investigative Site 097

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Lincoln, Nebraska, United States

Forest Investigative Site 701

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Seoul, Korea, Republic of

Forest Investigative Site 401

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Kopavogur, Iceland

Forest Investigative Site 580

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Kielce, Poland

Forest Investigative Site 629

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Nis, Serbia

Forest Investigative Site 807

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Kharkiv, Ukraine

Forest Investigative Site 801

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Odesa, Ukraine

Forest Investigative Site 704

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Yangsan-si, Gyeongsangnam-do, Korea, Republic of

Forest Investigative Site 626

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Belgrade, Serbia

Forest Investigative Site 627

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Belgrade, Serbia

Forest Investigative Site 651

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Singapore, Singapore

Forest Investigative Site 730

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Torremolinos, Málaga, Spain

Forest Investigative Site 091

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New Orleans, Louisiana, United States

Forest Investigative Site 072

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Chapel Hill, North Carolina, United States

Forest Investigative Site 105

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Charleston, South Carolina, United States

Forest Investigative Site 376

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Budapest, Hungary

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