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Treatment of Refractory Infantile Spasms With Fenfluramine

Primary Purpose

Infantile Spasm

Status
Not yet recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Fenfluramine
Sponsored by
Children's Hospital of Orange County
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Infantile Spasm

Eligibility Criteria

12 Months - 36 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Children ages 3 to 36 months, inclusive
  • Clinical diagnosis of infantile spasms
  • Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin.

Exclusion Criteria:

  • 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

Sites / Locations

  • UCLA Health
  • Children's Hospital of Orange County

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Fenfluramine treatment

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.

Full Information

First Posted
February 21, 2020
Last Updated
April 14, 2023
Sponsor
Children's Hospital of Orange County
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1. Study Identification

Unique Protocol Identification Number
NCT04289467
Brief Title
Treatment of Refractory Infantile Spasms With Fenfluramine
Official Title
A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
September 1, 2023 (Anticipated)
Primary Completion Date
December 31, 2024 (Anticipated)
Study Completion Date
December 31, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Hospital of Orange County

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief 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. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Infantile Spasm

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Fenfluramine treatment
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Fenfluramine
Other Intervention Name(s)
Fenfluramine Hydrochloride
Intervention Description
Open-label
Primary Outcome Measure Information:
Title
Electroclinical response (Efficacy)
Description
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.
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Computational electroencephalography response (Efficacy)
Description
Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden.
Time Frame
12 months
Title
Incidence of treatment emergent adverse events (Safety and tolerability)
Description
Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography.
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
36 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Children ages 12 to 36 months, inclusive Clinical diagnosis of infantile spasms Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin. Exclusion Criteria: 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Angelyque Lorenzana, MA
Phone
714-509-8972
Email
alorenzana@choc.org
First Name & Middle Initial & Last Name or Official Title & Degree
Virginia Allhusen, PhD
Email
vallhusen@choc.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Daniel Shrey, MD
Organizational Affiliation
Children's Hospital of Orange County
Official's Role
Principal Investigator
Facility Information:
Facility Name
UCLA Health
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angela L. Martinez
Phone
310-206-7630
Email
angelamartinez@mednet.ucla.edu
First Name & Middle Initial & Last Name & Degree
Shaun A Hussain, MD
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Daniel Shrey, MD
Email
dshrey@choc.org
First Name & Middle Initial & Last Name & Degree
Angelyque Lorenzana
Phone
714-509-8972
Email
alorenzana@choc.org

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Treatment of Refractory Infantile Spasms With Fenfluramine

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