Verapamil as Therapy for Children and Young Adults With Dravet Syndrome
NCT ID: NCT01607073
Last Updated: 2021-04-13
Study Results
Results available
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View full resultsBasic Information
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Recruitment Status
COMPLETED
Clinical Phase
PHASE2
Total Enrollment
2 participants
Study Classification
INTERVENTIONAL
Study Start Date
2012-04-30
Study Completion Date
2015-01-31
Brief Summary
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This study will assess how well the drug verapamil can improve control of seizures and dysautonomia symptoms in children and young adults diagnosed with Dravet syndrome. The safety of verapamil when given with all concomitant medications will also be assessed.
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Detailed Description
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Dravet syndrome (DS) is a devastating form of pediatric seizure disorder (epilepsy), often related to abnormalities of one of the genes that controls sodium channel function in the brain (SCN1A). Most children with DS experience continued seizures even with optimal treatment of currently available anti-seizure therapies \[1\]. Many of these seizures are prolonged, and can be life threatening.
This pilot study will assess the efficacy of verapamil in improving control of seizures in children and young adults DS. This will be done by adding verapamil as open label adjunctive therapy to medications already being given. Investigators will assess the effect of verapamil therapy on seizure control and on signs of autonomic dysfunction observable to the parents/guardians. Signs of autonomic function include body temperature regulation, sweating, heart rate, pupil size, and flushing of the skin. Iannetti, et al reported treating 2 children with clinical DS (one with an SCN1A mutation) with verapamil as adjunctive therapy \[2\]. Both children had a positive clinical response persisting for a number of months. No adverse effects were noted. We have treated an additional 4 children with DS with verapamil. There have been no significant adverse effects; 3 of 4 have experienced improved seizure control for months also.
Verapamil has been shown to affect autonomic tone in patients with cardiac disorders (eg. high blood pressure, heart attack). It alters the balance between parts of the autonomic nervous system's function (called sympathetic and parasympathetic function) with a shift toward decreased sympathetic tone and increased parasympathetic (vagus nerve) tone \[8, 9, 10\]. Verapamil is used as an effective agent to treat certain types of autonomic headaches in both adults and children. In cluster headaches, autonomic symptoms (tearing, nasal congestion, facial sweating, papillary constriction) are prominent; verapamil is an accepted treatment \[11, 12\].
Intense emotion triggers seizures in a subset of children with DS. Modulation of autonomic function is likely an integral part of seizure threshold in those so affected. Children with DS have a higher rate of signs of abnormal autonomic function than do controls \[13\]. Cardiac autonomic control is also altered in these children, with a shift in the balance between sympathetic (relatively overactive) and parasympathetic (relatively less active) tone \[14\]. Similar findings have been identified in adults with intractable epilepsy and children with partial epilepsy \[15, 16, 17\]. Verapamil's action in stabilizing the balance of sympathetic and parasympathetic tone may play a role altering autonomic tone abnormalities in children with DS as well. This may be a part of the mechanism that leads to improved seizure control.
Verapamil has been in clinical use for \~ 25 years. The FDA has granted an Investigational New Drug approval for use of this medication in this population of children and young adults. Investigators propose to add it to the patient's existing medications, and evaluate potential improvement in seizure control. Potential side effects will be screened. Investigators will monitor liver function with blood tests as well as concentrations of anti-seizure medications. Verapamil and nor-verapamil levels will be assessed twice also. Testing of heart rhythm (EKG) will be done before the study starts and twice more during the study.
This pilot study will assess the efficacy of verapamil in improving control of seizures in children and young adults DS. This will be done by adding verapamil as open label adjunctive therapy to medications already being given. Investigators will assess the effect of verapamil therapy on seizure control and on signs of autonomic dysfunction observable to the parents/guardians. Signs of autonomic function include body temperature regulation, sweating, heart rate, pupil size, and flushing of the skin. Iannetti, et al reported treating 2 children with clinical DS (one with an SCN1A mutation) with verapamil as adjunctive therapy \[2\]. Both children had a positive clinical response persisting for a number of months. No adverse effects were noted. We have treated an additional 4 children with DS with verapamil. There have been no significant adverse effects; 3 of 4 have experienced improved seizure control for months also.
Verapamil has been shown to affect autonomic tone in patients with cardiac disorders (eg. high blood pressure, heart attack). It alters the balance between parts of the autonomic nervous system's function (called sympathetic and parasympathetic function) with a shift toward decreased sympathetic tone and increased parasympathetic (vagus nerve) tone \[8, 9, 10\]. Verapamil is used as an effective agent to treat certain types of autonomic headaches in both adults and children. In cluster headaches, autonomic symptoms (tearing, nasal congestion, facial sweating, papillary constriction) are prominent; verapamil is an accepted treatment \[11, 12\].
Intense emotion triggers seizures in a subset of children with DS. Modulation of autonomic function is likely an integral part of seizure threshold in those so affected. Children with DS have a higher rate of signs of abnormal autonomic function than do controls \[13\]. Cardiac autonomic control is also altered in these children, with a shift in the balance between sympathetic (relatively overactive) and parasympathetic (relatively less active) tone \[14\]. Similar findings have been identified in adults with intractable epilepsy and children with partial epilepsy \[15, 16, 17\]. Verapamil's action in stabilizing the balance of sympathetic and parasympathetic tone may play a role altering autonomic tone abnormalities in children with DS as well. This may be a part of the mechanism that leads to improved seizure control.
Verapamil has been in clinical use for \~ 25 years. The FDA has granted an Investigational New Drug approval for use of this medication in this population of children and young adults. Investigators propose to add it to the patient's existing medications, and evaluate potential improvement in seizure control. Potential side effects will be screened. Investigators will monitor liver function with blood tests as well as concentrations of anti-seizure medications. Verapamil and nor-verapamil levels will be assessed twice also. Testing of heart rhythm (EKG) will be done before the study starts and twice more during the study.
Conditions
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Dravet Syndrome
Study Design
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Allocation Method
NA
Intervention Model
SINGLE_GROUP
Primary Study Purpose
TREATMENT
Blinding Strategy
NONE
Study Groups
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open label adjunctive add on
open label adjunctive add on of verapamil to existing medications. dosing begins at 1 mg/kg/d and increases weekly to target of 4 mg/kg/d in divided doses (three times/day)
Group Type
OTHER
Verapamil
Intervention Type
DRUG
Verapamil will be prepared as a solution. A 50mg/ml oral suspension may be made with immediate release tablets and either a 1:1 mixture of Ora-Sweet and Ora-Plus or a 1:1 mixture of Ora-Sweet SF and Ora-Plus will be used.
Children will start on a 4 weeks titration period:
Week 1: 1mg/kg/day divided BID Week 2: 2mg/kg/day divided BID or TID Week 3: 3mg/kg/day divided BID or TID Week 4: 4mg/kg/day divided TID
In event of adverse events, and in consultation with the family and treating physician, the dosage may be decreased to 2mg/kg/day and remain at that dose for the remainder of the study.
Interventions
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Verapamil
Verapamil will be prepared as a solution. A 50mg/ml oral suspension may be made with immediate release tablets and either a 1:1 mixture of Ora-Sweet and Ora-Plus or a 1:1 mixture of Ora-Sweet SF and Ora-Plus will be used.
Children will start on a 4 weeks titration period:
Week 1: 1mg/kg/day divided BID Week 2: 2mg/kg/day divided BID or TID Week 3: 3mg/kg/day divided BID or TID Week 4: 4mg/kg/day divided TID
In event of adverse events, and in consultation with the family and treating physician, the dosage may be decreased to 2mg/kg/day and remain at that dose for the remainder of the study.
Intervention Type
DRUG
Other Intervention Names
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Calan
Covera
Isoptin
Verelan
Eligibility Criteria
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Inclusion Criteria
* 2 to 25 years old
* Onset of seizures in first year of life
* seizure type usually generalized tonic-clonic, clonic, or hemiclonic, often prolonged (\>10 minutes)
* myoclonic jerks/myoclonic seizures
* history of normal development at seizure onset with subsequent developmental delay or regression which occurs after seizure onset
* presence of documented abnormality on the SCN1A gene
* medically intractable epilepsy: must have been on at least 2 prior antiepileptic medications without adequate control of epilepsy
* subject is capable of giving informed consent (or assent if possible) or has an acceptable surrogate capable of giving informed consent on the subject's behalf
* Onset of seizures in first year of life
* seizure type usually generalized tonic-clonic, clonic, or hemiclonic, often prolonged (\>10 minutes)
* myoclonic jerks/myoclonic seizures
* history of normal development at seizure onset with subsequent developmental delay or regression which occurs after seizure onset
* presence of documented abnormality on the SCN1A gene
* medically intractable epilepsy: must have been on at least 2 prior antiepileptic medications without adequate control of epilepsy
* subject is capable of giving informed consent (or assent if possible) or has an acceptable surrogate capable of giving informed consent on the subject's behalf
Exclusion Criteria
* use of clonidine, propranolol, carbamazepine, oxcarbazine, stiripentol, lamotrigine, or cyclosporine
* Abnormalities of cardiac conduction or rhythm (excluding sinus arrhythmia) on screening EKG
* significant use of grapefruit juice
* ketogenic diet
* pregnancy
* Abnormalities of cardiac conduction or rhythm (excluding sinus arrhythmia) on screening EKG
* significant use of grapefruit juice
* ketogenic diet
* pregnancy
Minimum Eligible Age
2 Years
Maximum Eligible Age
25 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
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Mayo Clinic
OTHER
Sponsor Role
collaborator
Ann & Robert H Lurie Children's Hospital of Chicago
OTHER
Sponsor Role
collaborator
Dartmouth-Hitchcock Medical Center
OTHER
Sponsor Role
collaborator
Gillette Children's Specialty Healthcare
OTHER
Sponsor Role
lead
Responsible Party
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Responsibility Role
SPONSOR
Principal Investigators
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Beverly S Wical, MD
Role: PRINCIPAL_INVESTIGATOR
Gillette Children's Specialty Healthcare
Locations
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Children's Memorial Hospital
Chicago, Illinois, United States
Site Status
Mayo Clinic
Rochester, Minnesota, United States
Site Status
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States
Site Status
Mary Hitchcock Memorial Hospital
Lebanon, New Hampshire, United States
Site Status
Countries
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United States
References
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Skluzacek JV, Watts KP, Parsy O, Wical B, Camfield P. Dravet syndrome and parent associations: the IDEA League experience with comorbid conditions, mortality, management, adaptation, and grief. Epilepsia. 2011 Apr;52 Suppl 2:95-101. doi: 10.1111/j.1528-1167.2011.03012.x.
Reference Type
BACKGROUND
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Reference Type
BACKGROUND
Iannetti P, Parisi P, Spalice A, Ruggieri M, Zara F. Addition of verapamil in the treatment of severe myoclonic epilepsy in infancy. Epilepsy Res. 2009 Jul;85(1):89-95. doi: 10.1016/j.eplepsyres.2009.02.014. Epub 2009 Mar 20.
Reference Type
BACKGROUND
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Reference Type
BACKGROUND
Other Identifiers
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IND 113666
Identifier Type: -
Identifier Source: org_study_id
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