Prevention of West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)

Overview

West syndrome (WS) is a specific type of epilepsy (or seizure disorder) that has three features: infantile spasms (type of seizure), loss of milestones, and a specific pattern on electroencephalogram (EEG or brain wave test) called hypsarhythmia. The purpose of this study is to detect pre-hypsarhythmia in infants at high-risk for WS and determine whether treatment with ACTH will prevent WS.

Full Title of Study: “Early Treatment of Infants at High Risk of Developing West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Prevention
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 2018

Detailed Description

Hypothesis: Preemptive ACTH will halt the evolution of hypsarhythmia and improve the EEG patterns in infants with pre-hypsarhythmic EEG. Aim. To determine whether a low dose ACTH improves EEG, we will repeat EEG one month after a 2 week course of daily ACTH.

Interventions

  • Drug: adrenocorticotropin hormone
    • ACTH 16 units intramuscular injection once daily for 2 weeks

Arms, Groups and Cohorts

  • Experimental: ACTH treatment
    • Infants with a Type 3 EEG (pre-hypsarhythmia) will be treated with ACTH for 2 weeks.

Clinical Trial Outcome Measures

Primary Measures

  • Evidence for improvement in the EEG one month following initiation of the 2 week course of low-dose ACTH.
    • Time Frame: 1 month
    • If pre-hypsarhythmia (Type 3) is detected, ACTH treatment is given for 2 weeks and an EEG is performed one month later. Primary outcome is improvement in EEG (as defined by assigned type).

Participating in This Clinical Trial

Inclusion Criteria

  • Infants with pre-hypsarhythmia (Type 3 EEG) between 2 months to 12 months of age. Exclusion criteria:
  • Infants with any of the following diagnoses: – A previous history of infantile spasms; – Known inborn error of metabolism; – Other symptomatic epileptic encephalopathy (e.g. Ohtahara syndrome).
  • Gender Eligibility: All

    Minimum Age: 2 Months

    Maximum Age: 12 Months

    Are Healthy Volunteers Accepted: No

    Investigator Details

    • Lead Sponsor
      • Ann & Robert H Lurie Children’s Hospital of Chicago
    • Collaborator
      • Thrasher Research Fund
    • Provider of Information About this Clinical Study
      • Principal Investigator: John J Millichap, MD, Attending Physician, Division of Neurology – Ann & Robert H Lurie Children’s Hospital of Chicago
    • Overall Official(s)
      • John J. Millichap, MD, Principal Investigator, Ann & Robert H. Lurie Children’s Hospital of Chicago and Northwestern University Feinberg School of Medicine
      • Sookyong Koh, MD, PhD, Principal Investigator, Ann & Robert H. Lurie Children’s Hospital of Chicago and Northwestern University Feinberg School of Medicine
      • Doulgas R Nordli, Jr, MD, Principal Investigator, Ann & Robert H. Lurie Children’s Hospital of Chicago and Northwestern University Feinberg School of Medicine

    References

    SOREL L, DUSAUCY-BAULOYE A. [Findings in 21 cases of Gibbs' hypsarrhythmia; spectacular effectiveness of ACTH]. Acta Neurol Psychiatr Belg. 1958 Feb;58(2):130-41. French.

    Okumura A, Watanabe K. Clinico-electrical evolution in pre-hypsarrhythmic stage: towards prediction and prevention of West syndrome. Brain Dev. 2001 Nov;23(7):482-7.

    Suzuki M, Okumura A, Watanabe K, Negoro T, Hayakawa F, Kato T, Itomi K, Kubota T, Maruyama K. The predictive value of electroencephalogram during early infancy for later development of West syndrome in infants with cystic periventricular leukomalacia. Epilepsia. 2003 Mar;44(3):443-6.

    Philippi H, Wohlrab G, Bettendorf U, Borusiak P, Kluger G, Strobl K, Bast T. Electroencephalographic evolution of hypsarrhythmia: toward an early treatment option. Epilepsia. 2008 Nov;49(11):1859-64. doi: 10.1111/j.1528-1167.2008.01715.x. Epub 2008 Jul 9.

    Watanabe K, Iwase K, Hara K. The evolution of EEG features in infantile spasms: a prospective study. Dev Med Child Neurol. 1973 Oct;15(5):584-96.

    Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials. 1989 Mar;10(1):1-10.

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