Infantile SpasmPhase 2NCT04289467
Treatment of Refractory Infantile Spasms With Fenfluramine
A Phase 2 study testing Fenfluramine in people with Infantile Spasm, run by Children's Hospital of Orange County at 2 sites in United States.
Trial summary
This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Read more about this study
- Who is it for?
- Ages 12 to 36 months
- Conditions the study is for
- Infantile Spasm
- What is being tested?
- Fenfluramine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- 16 Jun 2023to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 12 to 36 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children ages 12 to 36 months, inclusive
- Clinical diagnosis of infantile spasms
- Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin.
Who cannot join
- Significant preexisting cardiovascular disease
- Exposure to any cannabinoid product within 14 days of screening
- Initiation or dose-titration of any second-line treatment for infantile spasms in the 14 days prior to screening.
- Implantation of a vagal nerve simulator within 14 days of screening
- Initiation and maintenance of the ketogenic diet within 3 months of screening
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Virginia Allhusen, PhD contact
United States2 sites, 2 recruiting
- Not yet recruiting
UCLA Health
Los Angeles, California, 90095
Angela L. Martinez, (310) 206-7630, angelamartinez@mednet.ucla.edu
Shaun A Hussain, MD
- Recruiting
Children's Hospital of Orange County
Orange, California, 92868
Daniel Shrey, MD, dshrey@choc.org
Angelyque Lorenzana, 714-509-8972, alorenzana@choc.org
About this study
Official title: A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Patients with infantile spasms that have not responded to treatment with vigabatrin and ACTH we will be invited to participate. Study participants will undergo baseline video-EEG, receive treatment with fenfluramine for 21 days, and then undergo repeat video-EEG to determine effectiveness. Patients with favorable response will have the opportunity to continue treatment for up to 6 months.
- Conditions
- Infantile Spasm
- Being tested
- Fenfluramine
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FenfluramineDrug
Also called Fenfluramine Hydrochloride
Open-label
| Study group | Role | What they receive |
|---|---|---|
| Fenfluramine treatment | Experimental | Open label treatment with fenfluramine. Dosage will be titrated to 0.8 mg/kg/day, for an initial duration of 21 days. Patients with favorable response will have an option to continue treatment for up to 6 months. Receives: Drug: Fenfluramine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Electroclinical response (Efficacy)
Number of participants with resolution of epileptic spasms and hypsarrhythmia (if present at baseline) after 21 days of treatment, as determined by overnight video-electroencephalography (EEG) evaluation and caregiver seizure diary.
Measured: 12 months
Secondary outcomes (2)
Computational electroencephalography response (Efficacy)
Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden.
Measured: 12 months
Incidence of treatment emergent adverse events (Safety and tolerability)
Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2020
- Study start16 Jun 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile SpasmStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Orange County
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Daniel Shrey, Children's Hospital of Orange County
- Investigators
- Daniel Shrey, MD, Principal investigator, Children's Hospital of Orange County
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04289467
- Sponsor's protocol ID
- ZX008-IS
- Status last verified
- Apr 2026
- First submitted
- 21 Feb 2020
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
- ClinicalTrials.gov ID
- NCT04289467
- Acronym / sponsor ID
- ZX008-IS
- Conditions
- Infantile Spasm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Children's Hospital of Orange CountyUniversity, hospital or other
- Study start
- 16 Jun 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 28 Feb 2020 (actual)
- 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.
This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Patients with infantile spasms that have not responded to treatment with vigabatrin and ACTH we will be invited to participate. Study participants will undergo baseline video-EEG, receive treatment with fenfluramine for 21 days, and then undergo repeat video-EEG to determine effectiveness. Patients with favorable response will have the opportunity to continue treatment for up to 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fenfluramine treatment | Experimental | Drug: FenfluramineOpen label treatment with fenfluramine. Dosage will be titrated to 0.8 mg/kg/day, for an initial duration of 21 days. Patients with favorable response will have an option to continue treatment for up to 6 months. |
Who may qualify
- Ages
- 12 to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Children ages 12 to 36 months, inclusive
- Clinical diagnosis of infantile spasms
- Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin.
Key exclusion criteria (5 listed)
- Significant preexisting cardiovascular disease
- Exposure to any cannabinoid product within 14 days of screening
- Initiation or dose-titration of any second-line treatment for infantile spasms in the 14 days prior to screening.
- Implantation of a vagal nerve simulator within 14 days of screening
- Initiation and maintenance of the ketogenic diet within 3 months of screening
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 |
|---|---|---|
| FenfluramineAlso: Fenfluramine HydrochlorideOpen-label | Drug | Fenfluramine treatment |
Primary outcome
Electroclinical response (Efficacy)
Time frame: 12 months
Number of participants with resolution of epileptic spasms and hypsarrhythmia (if present at baseline) after 21 days of treatment, as determined by overnight video-electroencephalography (EEG) evaluation and caregiver seizure diary.
2 secondary outcomes
Computational electroencephalography response (Efficacy)
Time frame: 12 months
Incidence of treatment emergent adverse events (Safety and tolerability)
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- Individual participant dataSharing planned: No
Related trials for Infantile Spasm
Not yet recruitingObservational study
IBIS - Investigating Reliability of BIS and SEDLINE Monitoring in Children With Developmental and Epileptic Encephalopathies (DEE).
Epileptic Encephalopathy, Early Infantile Epileptic Encephalopathy
Active, not recruitingPhase 3
A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder.
CDKL5 Deficiency Disorder, Generalized Tonic Clonic Seizure
RecruitingObservational study
Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
Epilepsy, West Syndrome
RecruitingPhase 2
A Study to Prevent Infantile Spasms Relapse
Infantile Spasms, Infantile Epileptic Spasms Syndrome
Active, not recruitingPhase 4
Clinical Trial on 7-day Followed by Maintenance Therapy for 10 Weeks vs. 14-day and no Maintenance Course of Prednisolone for the Treatment of Infantile Epileptic Spasms Syndrome (IESS)
Spasms, Infantile, Prednisolone
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.
Virginia Allhusen, PhD contact
Daniel Shrey, MD principal investigator
Children's Hospital of Orange County
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fenfluramine in people with Infantile Spasm, run by Children's Hospital of Orange County at 2 sites 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 3 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kellie Bacon, MPH (714-509-8972, kellie.bacon@choc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Electroclinical response (Efficacy) (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Apr 2026.