Minocycline to Treat Childhood Regressive Autism
Condition: Autism; Minocycline; Regressive Autism
MeSH terms: Autistic Disorder
Intervention: Minocycline
Intervention MeSH terms: Minocycline
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 11.0
Sex: All
Minimum age: 3 Years
Maximum age: 12 Years
Healthy volunteers: No
Keywords: Immunology; Microglia; NF-Kappa-B; Antibiotic; Autism; Minocycline; Regressive Autism

Summary:

      There is a subgroup of children with autism that appears to develop typically for a period of
      time, and then loses social or language skills, or regresses. A recent study by Vargas and
      co-workers at Johns Hopkins has demonstrated that this regressive type of autism is
      associated with chronic brain inflammation as shown by an abnormal production of inflammatory
      cytokines among other abnormalities.

      This present study will test the effectiveness of minocycline, an antibiotic with
      anti-inflammatory properties, in treating regressive autism. Although behavioral therapies
      have improved some symptoms of autism, there are no medical treatments for the disorder, and
      many children have ongoing behavioral difficulties. A medicine with anti-inflammatory
      properties may be beneficial for children with regressive autism.

      This will be an open-label trial, meaning all children in this study will receive
      minocycline. They will also receive vitamin B6 to reduce the possible chance of side effects
      of the minocycline.

      Children ages 3 to 12 with regressive autism may be eligible for this study. The children
      will take minocycline and vitamin B6 daily for 6 months. Prior to starting the medication and
      vitamin B6, children will receive a comprehensive diagnostic assessment for autism as well as
      a physical examination, medical history, and laboratory tests. Children will then receive
      ongoing assessments to monitor their behavior, communication, language skills, and medical
      issues at 2 weeks, and at 1, 2, 4, 6, and 12 months. Children who respond to the treatment
      will receive an additional 3 months of minocycline and vitamin B6.
    

Eligibility criteria:

        -  INCLUSION CRITERIA:

        The sample will be children with:

          -  Diagnosis of idiopathic autism and regression

          -  Age between 3 and 12 years

          -  Willingness to undergo lumbar puncture for evaluation of proinflammatory CSF cytokines

          -  Stable behavioral plus or minus medication therapies.

        EXCLUSION CRITERIA:

          -  Significant prematurity at birth (less than 32 weeks gestation); or birthweight
             significantly below normal for gestational age (SGA--small for gestational age).

          -  Neurologic disorders including cerebral palsy, uncontrolled epilepsy, and
             Landau-Kleffner syndrome.

          -  Evidence of renal insufficiency or hepatic disease (to reduce the incidence of
             side-effects, since minocycline is excreted by the kidneys following hepatic
             metabolism)

          -  Increased risk of developing lupus-like syndrome with minocycline administration
             (positive anti-double stranded DNA or anti-nucleosome antibody tests at baseline, or
             presence of a first degree relative with S.L.E.)

          -  Recent (less than two months prior to study entry) initiation of a behavioral therapy
             program or new psychotropic medication trial.

          -  Subjects on one of the medications/supplements listed as those with possible
             interactions or those on high dose B6 supplementation. For those families who are
             interested in the study but are on any of these medications/supplements at the time of
             intake, they will be instructed to wean the medication as appropriate (working with
             the prescribing MD), and they will be enrolled after a 6-week wash-out period.