West SyndromePREVENT-WSNCT01367964
Prevention of West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)
A study testing adrenocorticotropin hormone in people with West Syndrome, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 12 months
- Conditions the study is for
- West Syndrome
- What is being tested?
- adrenocorticotropin hormone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2011to 5 May 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Infants with pre-hypsarhythmia (Type 3 EEG) between 2 months to 12 months of age.
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
About this study
Official title: Early Treatment of Infants at High Risk of Developing West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)
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.
Read the 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.
- Conditions
- West Syndrome
- Being tested
- adrenocorticotropin hormone
- Keywords
- Hypsarrhythmia, Spasms, Infantile, West Syndrome
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
adrenocorticotropin hormoneDrug
Also called H.P. Acthar® Gel (repository corticotropin injection)
ACTH 16 units intramuscular injection once daily for 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| ACTH treatment | Experimental | Infants with a Type 3 EEG (pre-hypsarhythmia) will be treated with ACTH for 2 weeks. Receives: Drug: adrenocorticotropin hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evidence for improvement in the EEG one month following initiation of the 2 week course of low-dose ACTH.
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).
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2011 (estimated)
- Study startJul 2011
- Primary completion30 Aug 2016
- Study completion5 May 2020
Why it stopped: Staffing disruptions leading to incomplete study enrollment prior to PI departure.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for West SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- Thrasher Research Fund
- Responsible party
- Sponsor
- Investigators
- John J. Millichap, MD, Principal investigator, Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of MedicineSookyong Koh, MD, PhD, Principal investigator, Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of MedicineDoulgas R Nordli, Jr, MD, Principal investigator, Ann & Robert H. Lurie Children's Hospital of Chicago and Northwestern University Feinberg School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01367964
- Sponsor's protocol ID
- 2011-14518
- Acronym
- PREVENT-WS
- Status last verified
- Aug 2026
- First submitted
- 3 Jun 2011
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
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.
PubMed 13532578Okumura 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.
PubMed 11701242Suzuki 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.
PubMed 12614401Philippi 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.
PubMed 18631366Watanabe 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.
PubMed 4358106Simon 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.
PubMed 2702835
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Treatment of Infants at High Risk of Developing West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)
- ClinicalTrials.gov ID
- NCT01367964
- Acronym / sponsor ID
- PREVENT-WS / 2011-14518
- Conditions
- West Syndrome
- Keywords
- Hypsarrhythmia, Spasms, Infantile, West Syndrome
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Staffing disruptions leading to incomplete study enrollment prior to PI departure.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- Thrasher Research Fund
- Study start
- Jul 2011 (actual)
- Primary completion
- 30 Aug 2016 (actual)
- Study completion
- 5 May 2020 (actual)
- First posted
- 7 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ACTH treatment | Experimental | Drug: adrenocorticotropin hormoneInfants with a Type 3 EEG (pre-hypsarhythmia) will be treated with ACTH for 2 weeks. |
Who may qualify
- Ages
- 2 to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Infants with pre-hypsarhythmia (Type 3 EEG) between 2 months to 12 months of age.
Key exclusion criteria (4 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| adrenocorticotropin hormoneAlso: H.P. Acthar® Gel (repository corticotropin injection)ACTH 16 units intramuscular injection once daily for 2 weeks | Drug | ACTH treatment |
Primary outcome
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).
Results and safety
The study was stopped early (reason given: Staffing disruptions leading to incomplete study enrollment prior to PI departure.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2026.
- Background: PubMed 13532578SOREL 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.
- Background: PubMed 11701242Okumura 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.
- Background: PubMed 12614401Suzuki 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.
- Background: PubMed 18631366Philippi 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.
- Background: PubMed 4358106Watanabe 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.
- Background: PubMed 2702835Simon 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.
- Lurie Children's Epilepsy CenterLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing adrenocorticotropin hormone in people with West Syndrome, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Staffing disruptions leading to incomplete study enrollment prior to PI departure.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evidence for improvement in the EEG one month following initiation of the 2 week course of low-dose ACTH. (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01367964, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2026.