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Treatment of Refractory Infantile Spasms With Fenfluramine

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04289467
Recruitment Status : Unknown
Verified February 2020 by Shaun Hussain, MD, University of California, Los Angeles.
Recruitment status was:  Not yet recruiting
First Posted : February 28, 2020
Last Update Posted : February 28, 2020
Sponsor:
Information provided by (Responsible Party):
Shaun Hussain, MD, University of California, Los Angeles

Study Description
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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.

Condition or disease Intervention/treatment Phase
Infantile Spasm Drug: Fenfluramine Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
Estimated Study Start Date : April 1, 2020
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : December 31, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Cramps

Arms and Interventions
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Arm Intervention/treatment
Experimental: Fenfluramine treatment
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.
 Drug: Fenfluramine
Open-label
Other Name: Fenfluramine Hydrochloride


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Computational electroencephalography response (Efficacy) [ Time Frame: 12 months ]
    Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden.

  2. Incidence of treatment emergent adverse events (Safety and tolerability) [ Time Frame: 12 months ]
    Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography.


Eligibility Criteria
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Ages Eligible for Study:   3 Months to 36 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04289467


Contacts
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Contact: Angela Martinez 310-206-4037 angelamartinez@mednet.ucla.edu
Contact: Shaun Hussain, MD 310-206-4037

Locations
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United States, California
University of California, Los Angeles
Los Angeles, California, United States, 90095-1752
Sponsors and Collaborators
University of California, Los Angeles
Investigators
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Principal Investigator: Shaun Hussain, MD Univeristy of California, Los Angeles
More Information
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Responsible Party: Shaun Hussain, MD, Associate Professor, University of California, Los Angeles
ClinicalTrials.gov Identifier: NCT04289467    
Other Study ID Numbers: ZX008-IS
First Posted: February 28, 2020    Key Record Dates
Last Update Posted: February 28, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Spasm
Spasms, Infantile
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
 Epileptic Syndromes
Fenfluramine
Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs