Safety of L1-79 in Autism
Condition: Autism
MeSH terms: Autistic Disorder
Study type: Interventional
Phase: Phase 2

Summary: 
      This is a five-arm designed to assess the safety of L1-79 that incorporates 15 prospectively
      randomized, placebo controlled patients and 5 open label patients at either 100 tid (three
      times daily) or 200 tid dosing for 28 days. The open label patients will be assessed for the
      purpose of understanding PK/PD and to determine if there are any EKG changes associated with
      the administration of L1-79. Additional safety information will be provided by the 30
      patients randomized 2:1 active:placebo.
    

Detailed description: 
      Protocol Number: HT 02-121

      Protocol Title: Phase 2 Safety Study of L1-79 for the Treatment of Autism Study Phase: 2

      The first cohort of 20 patients to be enrolled will all receive L1-79 100 mg t.i.d., and will
      be comprised of 3 groups of patients. The first group of patients to receive 100 mg will
      differ from the others in that they will get blood samples drawn for PK analysis and EKGs
      will be taken. The safety and PK data from this group will be submitted for FDA review and
      acceptance before the 200 mg t.i.d. cohort will be enrolled. The remaining 15 patients in
      this cohort will be randomized to receive either L1-79 100 mg t.i.d. or placebo on a 2:1
      basis (2 L1-79 patients for each placebo patient). While the FDA is reviewing the data from
      the first 5 patients all 100 mg t.i.d. patients will continue to be treated.

      The second cohort is identical to the first. The initial 5 patients to be enrolled will
      differ from the others in that they will get blood samples drawn for PK analysis and EKGs
      will be taken. The remaining 15 patients in this cohort will be randomized to receive either
      L1-79 200 mg t.i.d. or placebo on a 2:1 active:placebo.

      Sample Size: N=40

        -  Group 1 (n=5) open100mg L1-79 (1x100mg capsule+1 placebo capsule)

        -  Group 2 (n=10) blind100mg L1-79 (1x100mg capsule+1 placebo capsule)

        -  Group 3 (n=5) open200 mg L1-79 (2x100 mg capsules)

        -  Group 4 (n=10) blind200 mg L1-79 (2x100 mg capsules)

        -  Group 5 (n=10) Placebo (2 placebo capsules) All Groups will receive the assigned study
           drug three-times daily

      Study Population: Male subjects with autism between the ages of 13 and 21 years of age who
      meet the entry criteria and who are able to complete standardized measures allowing them to
      participate in this study.

      Evaluation Schedule: Subjects will be evaluated within one week prior to study accession, and
      weekly throughout the dosing period, and again 4 weeks after the cessation of treatment. The
      Assigned Dosage Groups (Groups 1 and 3) will have PK blood draws and EKG the randomized group
      will not have.

      Safety Measures: All Groups will have regularly scheduled complete history and physical
      examination that includes orthostatic blood pressure measurements, vital signs, CBC,
      differential, platelet counts, urine analysis, and serum analytes including: total protein,
      albumin, glucose, BUN, creatinine, direct and total bilirubin, alkaline phosphatase,
      phosphorous, calcium, AST, ALT, sodium, potassium, chloride, bicarbonate, T4, TSH, and
      adverse events assessments. The Assigned Groups (1 and 3) will also have electrocardiograms
      taken at the study screening visit and weekly throughout the treatment interval.
    

Intervention name(s): L1-79; Placebo
Intervention type(s): Drug; Drug
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment