Clinical trial NCT01367964: Prevention of West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)
Official title: Early Treatment of Infants at High Risk of Developing West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: West Syndrome
Keywords: Hypsarrhythmia
Spasms, Infantile
West Syndrome
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
Collaborators: name: Thrasher Research Fund
First posted: 2011-06-07
Enrollment: 28
Sex: ALL
Minimum age (years): 2
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: 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.

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.

Eligibility criteria: 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).

Primary outcomes:
measure: Evidence for improvement in the EEG one month following initiation of the 2 week course of low-dose ACTH.
timeFrame: 1 month
Secondary outcomes:
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
Study officials:
affiliation: Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of Medicine
name: John J. Millichap, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of Medicine
name: Sookyong Koh, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of Medicine
name: Doulgas R Nordli, Jr, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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. No abstract available. French.
citation: 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. doi: 10.1016/s0387-7604(01)00291-1.
citation: 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. doi: 10.1046/j.1528-1157.2003.29202.x.
citation: 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.
citation: 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. doi: 10.1111/j.1469-8749.1973.tb05169.x. No abstract available.
citation: Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials. 1989 Mar;10(1):1-10. doi: 10.1016/0197-2456(89)90015-9.