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Alternating Hemiplegia of ChildhoodPhase 1/2AHC-SONCT00931164

Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO)

A Phase 1/2 study testing Sodium Oxybate in people with Alternating Hemiplegia of Childhood, run by University of Utah at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

WHO: The investigators are recruiting children and young adults to participate in a research study who: 1. Have been diagnosed with Alternating Hemiplegia of Childhood (AHC) 2. Are between the ages of 6 months - 25 years old 3. Have at least three 10-minute-long AHC episodes during a typical week 4. Read more about this study

Who is it for?
Ages 6 months to 25 years
Conditions the study is for
Alternating Hemiplegia of Childhood
What is being tested?
Sodium Oxybate
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
Aug 2009to Sep 2011
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 6 months to 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Subjects must meet all of the following inclusion criteria.
  • AHC classic criteria:
  • Onset of symptomatology prior to 18 months of age
  • Repeated attacks of hemiplegia involving either side of the body
  • Paroxysmal disturbances, including tonic or dystonic spells, oculomotor abnormalities, and various autonomic phenomena, during hemiplegic bouts or in isolation
  • Episodes of bilateral hemiplegia or quadriplegia starting either as generalization of a hemiplegic episode or bilateral from the beginning
Show all 17Show fewer
  • Immediate disappearance of symptoms upon sleeping, with possible resumption 10-20 minutes after waking
  • Evidence of developmental delay and neurologic abnormalities including choreoathetosis, dystonia, or ataxia
  • In addition:
  • Provision of a complete daily event log for 6 weeks prior to initiation of sodium oxybate therapy, including the: (1) frequency, (2) duration, (3) type, (4) severity of episodes, and (5) therapies, if any, used in an attempt to abort the episode
  • Ages 6 months to 25 years at the time of study enrollment
  • Minimum of 3 AHC episodes weekly, lasting at least 10 minutes each, on average prior to study enrollment
  • Brain neuroimaging studies excluding alternative etiology for symptoms
  • Documented absence of epileptiform features on EEG during typical ictal events
  • Ability of primary caregivers of study participants to submit online daily AHC Episode Logs indicating frequency and duration of ictal episodes over the entire study period
  • Written informed consent from parents/guardians and assent from children 7 years or older
  • Girls/women > 12 years of age will be required to be on birth control continuously if they are considered at risk to become pregnant. Those not on birth control will be required to have a screening pregnancy test at baseline, and to confirm their willingness to practice birth control or abstain from sexual activity for the duration of treatment with study medication.

Who cannot join

  • Subjects who meet any of the following criteria will be excluded from participation.
  • History of hepatic insufficiency, renal insufficiency, significant respiratory disease, cardiac arrhythmia, congenital heart defect, hypertension, or ischemic stroke
  • History of allergy/sensitivity to sodium oxybate
  • Use of sodium oxybate within 30 days of study enrollment
  • Serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study enrollment
  • Change in neurologic medication regimen within 30 days of study enrollment.
Show all 11Show fewer
  • Inability to stay at the Center for Clinical and Translational Science (CCTS) for 5 days due to behavioral issues
  • Unwillingness or inability to travel to study site during the necessary 1 week titration period to determine the most appropriate dose of GHB for subsequent administration
  • Noncompliance with AHC Episode Log or study visit requirements
  • Sleep apnea not adequately treated with C-PAP and oxygen saturation monitoring prior to drug therapy initiation
  • Succinic semialdehyde dehydrogenase deficiency

Trial locations and contacts

1 site in 1 country.

United States1 site
  • University of Utah Hospital

    Salt Lake City, Utah, 84132

Other trials in United States

About this study

Official title: Single-center Phase I/II Trial of Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO Trial)

WHO: The investigators are recruiting children and young adults to participate in a research study who:

  1. Have been diagnosed with Alternating Hemiplegia of Childhood (AHC)
  2. Are between the ages of 6 months - 25 years old
  3. Have at least three 10-minute-long AHC episodes during a typical week
  4. Can commit to 12 weeks of completing of a daily log describing AHC episodes and to a multi-night hospital stay at the University of Utah Center for Clinical and Translational Science (CCTS)
Read more

WHY: The goal of this study is to evaluate whether the study drug can safely and effectively decrease AHC episodes and improve the quality of life of individuals with AHC.

WHAT and WHERE: This study involves at least 12 weeks of completing and submitting daily AHC Episode Logs and a five-day hospital stay at the University of Utah CCTS.

There are 4 phases of the study, and they include:

  1. Six weeks of daily log completion prior to starting study drug
  2. Five day stay at the University of Utah CCTS
  3. Six additional weeks of daily log completion while using study drug at home
  4. One day clinic visit to the University of Utah for follow up

COMPENSATION:

There is no fee to participate. All procedures performed specifically for this study will be covered by the study and will not be billed to you or your insurance company.

The study drug will be dispensed to you free of charge. Meals and lodging at the study site will be provided free of charge for the study participant and one care provider. Additionally, the research team will assist subjects with booking and obtaining free transportation, such as an airline tickets, to and from the study site.

Being tested
Sodium Oxybate
Keywords
phase I/II study, effects of sodium oxybate, cohort of 6 children and young adults, AHC
Medical subject headings
Alternating hemiplegia of childhood

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Sodium OxybateDrug

    dosage is by weight

Study groups in NCT00931164
Study groupRoleWhat they receive
Sodium oxybateExperimental* The study is an open-label, Phase I/II trial designed to obtain additional safety and pharmacokinetic parameters for use of sodium oxybate in children and adolescents afflicted with AHC. * Given the limited number of children carrying the diagnosis of AHC, typical controls will not be available for our study. In lieu of this, the subjective recording of ictal episodes in the 6 week period prior to drug initiation will serve as reference in determining drug efficacy.
Receives: Drug: Sodium Oxybate

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Time Duration of AHC Episodes

    Measured: Week 7

  2. Observed Safety Data During 5-day Hospitalization for Drug Dose Identification

    Measured: Week 7

  3. Number of Participants Who Reported Side Effects During Home Drug Maintenance Phase

    Measured: 1 year

Secondary outcomes (5)
  1. Mean Daily Percentage of Time That Functional Status is Affected Due to Episodes

    Measured: Week 14

  2. Quality of Life Questionnaires

    Measured: Week 14

  3. Mood and Behavior Questionnaires

    Measured: Week 14

  4. Functional Skills Questionnaires

    Measured: Week 14

  5. Neuropsychological Tests

    Measured: Week 14

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Jul 2009 (estimated)
  2. Study startAug 2009
  3. Primary completionJan 2011
  4. Study completionSep 2011

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00931164
Sponsor's protocol ID
32164
Other IDs
103932
Acronym
AHC-SO
Status last verified
Mar 2014
First submitted
30 Jun 2009
Last updated
31 Mar 2014
Data monitoring committee
Yes

Publications

  • Addolorato G, Cibin M, Capristo E, Beghe F, Gessa G, Stefanini GF, Gasbarrini G. Maintaining abstinence from alcohol with gamma-hydroxybutyric acid. Lancet. 1998 Jan 3;351(9095):38. doi: 10.1016/s0140-6736(05)78088-0. No abstract available.

    PubMed 9433435
  • A 12-month, open-label, multicenter extension trial of orally administered sodium oxybate for the treatment of narcolepsy. Sleep. 2003 Feb 1;26(1):31-5.

    PubMed 12627729
  • Arnold DL, Silver K, Andermann F. Evidence for mitochondrial dysfunction in patients with alternating hemiplegia of childhood. Ann Neurol. 1993 Jun;33(6):604-7. doi: 10.1002/ana.410330608.

    PubMed 8498840
  • Bourgeois M, Aicardi J, Goutieres F. Alternating hemiplegia of childhood. J Pediatr. 1993 May;122(5 Pt 1):673-9. doi: 10.1016/s0022-3476(06)80003-x.

    PubMed 8496742
  • Bourgeois M, Aicardi J. The treatment of alternating hemiplegia of childhood with flunarizine: experience with 17 patients. Alternating Hemiplegia of Childhood, edited by F Andermann, J Aicardi, and F Vigevano. 1995;191-194.

  • Caputo F, Addolorato G, Stoppo M, Francini S, Vignoli T, Lorenzini F, Del Re A, Comaschi C, Andreone P, Trevisani F, Bernardi M; Alcohol Treatment Study Group. Comparing and combining gamma-hydroxybutyric acid (GHB) and naltrexone in maintaining abstinence from alcohol: an open randomised comparative study. Eur Neuropsychopharmacol. 2007 Dec;17(12):781-9. doi: 10.1016/j.euroneuro.2007.04.008. Epub 2007 Jul 3.

    PubMed 17611081
  • Casaer P. Flunarizine in alternating hemiplegia in childhood. An international study in 12 children. Neuropediatrics. 1987 Nov;18(4):191-5. doi: 10.1055/s-2008-1052478.

    PubMed 3320807
  • Casaer P, Azou M. Flunarizine in alternating hemiplegia in childhood. Lancet. 1984 Sep 8;2(8402):579. doi: 10.1016/s0140-6736(84)90794-3. No abstract available.

    PubMed 6147627
  • Defossez M, Le Bec G, Sancier A, Manelfe C, Espagno MT, Bouzat A, Sevely A. [Neuro-sedation and cranial C.-T. scan in infants and children (author's transl)]. Anesth Analg (Paris). 1979;36(11-12):525-9. French.

    PubMed 554477
  • Ferrara SD, Giorgetti R, Zancaner S, Orlando R, Tagliabracci A, Cavarzeran F, Palatini P. Effects of single dose of gamma-hydroxybutyric acid and lorazepam on psychomotor performance and subjective feelings in healthy volunteers. Eur J Clin Pharmacol. 1999 Jan;54(11):821-7. doi: 10.1007/s002280050560.

    PubMed 10027654
  • Frucht SJ, Bordelon Y, Houghton WH, Reardan D. A pilot tolerability and efficacy trial of sodium oxybate in ethanol-responsive movement disorders. Mov Disord. 2005 Oct;20(10):1330-7. doi: 10.1002/mds.20605.

    PubMed 15986420
  • Haan J, Kors EE, Terwindt GM, Vermeulen FL, Vergouwe MN, van den Maagdenberg AM, Gill DS, Pascual J, Ophoff RA, Frants RR, Ferrari. Alternating hemiplegia of childhood: no mutations in the familial hemiplegic migraine CACNA1A gene. Cephalalgia. 2000 Oct;20(8):696-700. doi: 10.1046/j.0333-1024.2000.00095.x.

    PubMed 11167897
  • Hunter AS, Long WJ, Ryrie CG. An evaluation of gamma-hydroxybutyric acid in paediatric practice. Br J Anaesth. 1971 Jun;43(6):620-8. doi: 10.1093/bja/43.6.620. No abstract available.

    PubMed 5089942
  • Jiang W, Chi Z, Ma L, Du B, Shang W, Guo H, Wu W. Topiramate: a new agent for patients with alternating hemiplegia of childhood. Neuropediatrics. 2006 Aug;37(4):229-33. doi: 10.1055/s-2006-924721.

    PubMed 17177149
  • Jen J, Yue Q, Nelson SF, Yu H, Litt M, Nutt J, Baloh RW. A novel nonsense mutation in CACNA1A causes episodic ataxia and hemiplegia. Neurology. 1999 Jul 13;53(1):34-7. doi: 10.1212/wnl.53.1.34.

    PubMed 10408533

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Sodium Oxybate in people with Alternating Hemiplegia of Childhood, run by University of Utah at 1 site 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 17 inclusion and 11 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: finished, as of the sponsor's last update on 31 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Yes. Results are posted in the registry (first posted 31 Mar 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time Duration of AHC Episodes (time frame: Week 7). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00931164, which also lists 43 linked publications.

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 31 Mar 2014.

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