A Novel Approach to Infantile Spasms

A Novel Approach to Infantile Spasms: Combined Cosyntropin Injectable Suspension, 1 mg/mL and Vigabatrin Induction Therapy

This study proposes to assess the efficacy of Cosyntropin Injectable Suspension, 1 mg/mL compared to vigabatrin. Additionally, this study proposes to determine efficacy of combination therapy of Cosyntropin Injectable Suspension, 1 mg/mL and vigabatrin to monotherapy Cosyntropin Injectable Suspension, 1mg/mL in children with new onset infantile spasms (IS).

Study Overview

• Status: Suspended
• Conditions: Infantile Spasm
• Intervention / Treatment: Drug: Cosyntropin Injectable Suspension 1 MG/ML + vigabatrin; Drug: Cosyntropin Injectable Suspension, 1 mg/mL; Drug: Vigabatrin

Detailed Description

This is a prospective randomized trial comparing 3 treatment arms:

1. Cosyntropin Injectable Suspension, 1 mg/mL
2. Vigabatrin
3. Combination of both these therapies, Cosyntropin Injectable Suspension, 1 mg/mL and vigabatrin for treatment of new onset infantile spasms. Outcome measures include resolution of clinical spasms, improvement of hypsarrhythmia as well as longer term outcomes of development and subsequent seizures.

The data is expected to demonstrate greater efficacy with Cosyntropin Injectable Suspension, 1 mg/mL than vigabatrin for the treatment of IS.

• Study Type: Interventional
• Enrollment (Anticipated): 394
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles
    • San Francisco, California, United States, 94158
      • UCSF Medical Center
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Anschutz Medical Campus
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Children's National Medical Center
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Children's Healthcare of Atlanta at Scottish Rite
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • C.S. Mott Children's Hospital
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • New York
    • Bronx, New York, United States, 10461
      • Montefiore Medical Center
    • New York, New York, United States, 10032
      • Columbia University Medical Center
  • North Carolina
    • Charlotte, North Carolina, United States, 28204
      • Atrium Health
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 months to 2 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• New onset infantile spasms
• Age > 2 months
• Age< 2 years
• Hypsarrhythmia on video-EEG
• Normal renal function

Exclusion Criteria:

• Prior treatment given for infantile spasms
• Diagnosis of Ohtahara syndrome or Early Myoclonic Epilepsy
• Absence of hypsarrhythmia
• Inability for the parent or caregiver to provide consent
• Inability for the parent or caregiver to complete seizure diary

• Diagnosis of:

  • scleroderma,
  • osteoporosis,
  • recent systemic fungal infections,
  • ocular herpes simplex,
  • recent surgery,
  • history of or the presence of a peptic ulcer,
  • congestive heart failure,
  • uncontrolled hypertension

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cosyntropin Injectable Suspension, 1mg/mL + vigabatrin	Drug: Cosyntropin Injectable Suspension 1 MG/ML + vigabatrin; Injectable + oral solution
Experimental: Cosyntropin Injectable Suspension, 1 mg/mL	Drug: Cosyntropin Injectable Suspension, 1 mg/mL; Injectable
Active Comparator: Vigabatrin	Drug: Vigabatrin; oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
A comparison of Cosyntropin Injectable Suspension and Vigabatrin on the proportion of subjects who become spasm-free as defined by a) and b).	a) Resolution of hypsarrhythmia via video electroencephalogram (EEG), and b) Resolution of clinical spasms via video EEG.	2 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
A comparison of combination therapy and Cosyntropin Injectable Suspension alone (monotherapy) on the proportion of subjects who become spasm-free as defined by a) and b)	a) Resolution of clinical spasms for 48 hours at 2 weeks sustained until day 42, and b) Resolution of hypsarrhythmia via video EEG at 2 weeks.	Day 14-42
A comparison between combination therapy and Cosyntropin Injectable Suspension alone (monotherapy) on the proportion of subjects who become seizure free at 6 months.	Difference in proportion of subjects who are seizure free between 5 and 6 months after treatment.	6 months
Comparison between combination therapy and Cosyntropin Injectable Suspension alone (monotherapy).	A comparison between combination therapy and Cosyntropin Injectable Suspension alone (monotherapy) on developmental scores as measured by the Adaptive Behavior Assessment System (ABAS III) at 18 months chronological age.	18 months chronological age
Comparison between combination therapy and Cosyntropin Injectable Suspension alone (monotherapy).	A comparison between combination therapy and Cosyntropin Injectable Suspension alone (monotherapy) on developmental scores as measured by the Vineland II at 18 months chronological age.	18 months chronological age

Collaborators and Investigators

• Sponsor: University of Colorado, Denver
• Collaborators: Pediatric Epilepsy Research Foundation; West Therapuetics, Inc
• Investigators: Principal Investigator: Kelly G. Knupp, MD, University of Colorado - Anschutz Medical Campus

Study record dates

Study Major Dates

• Study Start (Actual): April 19, 2018
• Primary Completion (Anticipated): June 1, 2021
• Study Completion (Anticipated): August 1, 2021

Study Registration Dates

• First Submitted: November 15, 2017
• First Submitted That Met QC Criteria: November 17, 2017
• First Posted (Actual): November 20, 2017

Study Record Updates

• Last Update Posted (Actual): May 27, 2021
• Last Update Submitted That Met QC Criteria: May 26, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Keywords: Vigabatrin; Cosyntropin
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy, Generalized; Epileptic Syndromes; Neurologic Manifestations; Neuromuscular Manifestations; Epilepsy; Spasms, Infantile; Spasm; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; GABA Agents; Anticonvulsants; Cosyntropin; Vigabatrin
• Other Study ID Numbers: 17-0222

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No