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

• ClinicalTrials.gov Identifier: NCT01367964
• Recruitment Status: Unknown
• First Posted: June 7, 2011
• Last Update Posted: November 16, 2018
• Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
• Collaborator: Thrasher Research Fund
• Information provided by (Responsible Party): John J Millichap, MD, Ann & Robert H Lurie Children's Hospital of Chicago

Study Description

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.

Condition or disease	Intervention/treatment	Phase
West Syndrome	Drug: adrenocorticotropin hormone	Not Applicable

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 28 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Prevention
• Official Title: Early Treatment of Infants at High Risk of Developing West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)
• Study Start Date: July 2011
• Estimated Primary Completion Date: December 2018
• Estimated Study Completion Date: December 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: ACTH treatment; Infants with a Type 3 EEG (pre-hypsarhythmia) will be treated with ACTH for 2 weeks.	Drug: adrenocorticotropin hormone; ACTH 16 units intramuscular injection once daily for 2 weeks; Other Name: H.P. Acthar® Gel (repository corticotropin injection)

Outcome Measures

Primary Outcome Measures :

1. 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).

Eligibility Criteria

• Ages Eligible for Study: 2 Months to 12 Months (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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).

Contacts and Locations

Locations

United States, Illinois

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States, 60611

Sponsors and Collaborators

Ann & Robert H Lurie Children's Hospital of Chicago

Thrasher Research Fund

Investigators

• Principal Investigator: John J. Millichap, MD; Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of Medicine
• Principal Investigator: Sookyong Koh, MD, PhD; Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of Medicine
• Principal Investigator: Doulgas R Nordli, Jr, MD; Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of Medicine

More Information

Additional Information:

Lurie Children's Epilepsy Center 

Publications:

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.

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.

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.

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. doi: 10.1111/j.1469-8749.1973.tb05169.x. No abstract available.

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.

• Responsible Party: John J Millichap, MD, Attending Physician, Division of Neurology, Ann & Robert H Lurie Children's Hospital of Chicago
• ClinicalTrials.gov Identifier: NCT01367964
• Other Study ID Numbers: 2011-14518
• First Posted: June 7, 2011
• Last Update Posted: November 16, 2018
• Last Verified: November 2018

Keywords provided by John J Millichap, MD, Ann & Robert H Lurie Children's Hospital of Chicago:

Hypsarrhythmia; Spasms, Infantile; West Syndrome

Additional relevant MeSH terms:

Spasms, Infantile; Syndrome; Disease; Pathologic Processes; Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Hormones; Adrenocorticotropic Hormone; Melanocyte-Stimulating Hormones; beta-Endorphin; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action