Pediatric Brain TumorPhase 1NCT01861990
Valproic Acid in Childhood Progressive Brain Tumors
A Phase 1 study testing Valproic Acid in people with Pediatric Brain Tumor and Glioma, run by Medical University of South Carolina at 1 site in United States.
Trial summary
The study investigates valproic acid added to radiation and temozolomide therapy (standard of care) for progressive or recurrent pediatric brain tumors. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Pediatric Brain Tumor, Glioma, Anaplastic Astrocytoma
- What is being tested?
- Valproic Acid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- May 2013to Dec 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject, parent, or guardian willing and able to give informed consent
- Recurrent or progressive pediatric brain tumor, with MRI evidence of disease
- Age at first diagnosis of brain tumor 1-21 years old
- Lansky or Karnofsky performance score of at least 50 at diagnosis
Who cannot join
- Pregnancy
- Prior intolerance to valproic acid
- History of use of temozolomide
- Use of enzyme inducing anticonvulsant medications (see appendix B)
- Known urea cycle disorder (e.g. ornithine transcarbamylase deficiency)
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical University of South Carolina
Charleston, South Carolina, 29425
About this study
Official title: Valproic Acid for Children With Recurrent and Progressive Brain Tumors
The study investigates valproic acid added to radiation and temozolomide therapy (standard of care) for progressive or recurrent pediatric brain tumors.
Read the detailed description
Patients with progressive or recurrent pediatric brain tumors are administered valproic acid, an HDAC inhibitor, along with standard of care therapy (radiation and temozolomide) for induction therapy. Thereafter, patients will be able to continue on valproic acid and temozolomide therapy as long as the combination is well tolerated and the tumor is not progressing.
- Being tested
- Valproic Acid
- Keywords
- Glioma, Glioblastoma, Pediatric, Child, Brain tumor, Astrocytoma, Medulloblastoma, PNET
- Medical subject headings
- Glioma, Astrocytoma, Medulloblastoma, Glioblastoma, Brain Neoplasms, Neuroectodermal Tumors, Primitive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Valproic AcidDrug
Also called Valproate, VPA, Depakote, Depakote ER, Depakene
All participants enrolled on valproic acid arm.
| Study group | Role | What they receive |
|---|---|---|
| Treatment arm | Experimental | All participants will be enrolled on to one, open-label arm. Participants will be treated with valproic acid in addition to standard of care therapy. Receives: Drug: Valproic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Completion of protocol
Number of participants completing the protocol will be measured.
Measured: 2 months
Secondary outcomes (1)
Time to progression
Participants will be evaluated for progression every 2 months while on the study.
Measured: 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2013 (estimated)
- Study startMay 2013
- Primary completionDec 2013
- Study completionDec 2013
Why it stopped: Feasibility of the trial was proven to be absent.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Pediatric Brain TumorStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amy-Lee Bredlau, Medical University of South Carolina
- Investigators
- Amy L Bredlau, MD, Principal investigator, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01861990
- Sponsor's protocol ID
- VA2013
- Other IDs
- IRG-97-219-14
- Status last verified
- May 2015
- First submitted
- 22 May 2013
- Last updated
- 22 May 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Van Nifterik KA, Van den Berg J, Slotman BJ, Lafleur MV, Sminia P, Stalpers LJ. Valproic acid sensitizes human glioma cells for temozolomide and gamma-radiation. J Neurooncol. 2012 Mar;107(1):61-7. doi: 10.1007/s11060-011-0725-z. Epub 2011 Oct 26.
PubMed 22037799Asklund T, Kvarnbrink S, Holmlund C, Wibom C, Bergenheim T, Henriksson R, Hedman H. Synergistic killing of glioblastoma stem-like cells by bortezomib and HDAC inhibitors. Anticancer Res. 2012 Jul;32(7):2407-13.
PubMed 22753697Fu J, Shao CJ, Chen FR, Ng HK, Chen ZP. Autophagy induced by valproic acid is associated with oxidative stress in glioma cell lines. Neuro Oncol. 2010 Apr;12(4):328-40. doi: 10.1093/neuonc/nop005. Epub 2009 Oct 15.
PubMed 20308311Weller M, Gorlia T, Cairncross JG, van den Bent MJ, Mason W, Belanger K, Brandes AA, Bogdahn U, Macdonald DR, Forsyth P, Rossetti AO, Lacombe D, Mirimanoff RO, Vecht CJ, Stupp R. Prolonged survival with valproic acid use in the EORTC/NCIC temozolomide trial for glioblastoma. Neurology. 2011 Sep 20;77(12):1156-64. doi: 10.1212/WNL.0b013e31822f02e1. Epub 2011 Aug 31.
PubMed 21880994Masoudi A, Elopre M, Amini E, Nagel ME, Ater JL, Gopalakrishnan V, Wolff JE. Influence of valproic acid on outcome of high-grade gliomas in children. Anticancer Res. 2008 Jul-Aug;28(4C):2437-42.
PubMed 18751431
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Valproic Acid for Children With Recurrent and Progressive Brain Tumors
- ClinicalTrials.gov ID
- NCT01861990
- Acronym / sponsor ID
- VA2013
- Keywords
- Glioma, Glioblastoma, Pediatric, Child, Brain tumor, Astrocytoma, Medulloblastoma, PNET
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Feasibility of the trial was proven to be absent.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Study start
- May 2013
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 24 May 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study investigates valproic acid added to radiation and temozolomide therapy (standard of care) for progressive or recurrent pediatric brain tumors.
Detailed description
Patients with progressive or recurrent pediatric brain tumors are administered valproic acid, an HDAC inhibitor, along with standard of care therapy (radiation and temozolomide) for induction therapy. Thereafter, patients will be able to continue on valproic acid and temozolomide therapy as long as the combination is well tolerated and the tumor is not progressing.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment arm | Experimental | Drug: Valproic AcidAll participants will be enrolled on to one, open-label arm. Participants will be treated with valproic acid in addition to standard of care therapy. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Subject, parent, or guardian willing and able to give informed consent
- Recurrent or progressive pediatric brain tumor, with MRI evidence of disease
- Age at first diagnosis of brain tumor 1-21 years old
- Lansky or Karnofsky performance score of at least 50 at diagnosis
Key exclusion criteria (5 listed)
- Pregnancy
- Prior intolerance to valproic acid
- History of use of temozolomide
- Use of enzyme inducing anticonvulsant medications (see appendix B)
- Known urea cycle disorder (e.g. ornithine transcarbamylase deficiency)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Valproic AcidAlso: Valproate, VPA, Depakote, Depakote ER, DepakeneAll participants enrolled on valproic acid arm. | Drug | Treatment arm |
Primary outcome
Completion of protocol
Time frame: 2 months
Number of participants completing the protocol will be measured.
1 secondary outcome
Time to progression
Time frame: 2 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2015.
- Background: PubMed 22037799Van Nifterik KA, Van den Berg J, Slotman BJ, Lafleur MV, Sminia P, Stalpers LJ. Valproic acid sensitizes human glioma cells for temozolomide and gamma-radiation. J Neurooncol. 2012 Mar;107(1):61-7. doi: 10.1007/s11060-011-0725-z. Epub 2011 Oct 26.
- Background: PubMed 22753697Asklund T, Kvarnbrink S, Holmlund C, Wibom C, Bergenheim T, Henriksson R, Hedman H. Synergistic killing of glioblastoma stem-like cells by bortezomib and HDAC inhibitors. Anticancer Res. 2012 Jul;32(7):2407-13.
- Background: PubMed 20308311Fu J, Shao CJ, Chen FR, Ng HK, Chen ZP. Autophagy induced by valproic acid is associated with oxidative stress in glioma cell lines. Neuro Oncol. 2010 Apr;12(4):328-40. doi: 10.1093/neuonc/nop005. Epub 2009 Oct 15.
- Background: PubMed 21880994Weller M, Gorlia T, Cairncross JG, van den Bent MJ, Mason W, Belanger K, Brandes AA, Bogdahn U, Macdonald DR, Forsyth P, Rossetti AO, Lacombe D, Mirimanoff RO, Vecht CJ, Stupp R. Prolonged survival with valproic acid use in the EORTC/NCIC temozolomide trial for glioblastoma. Neurology. 2011 Sep 20;77(12):1156-64. doi: 10.1212/WNL.0b013e31822f02e1. Epub 2011 Aug 31.
- Background: PubMed 18751431Masoudi A, Elopre M, Amini E, Nagel ME, Ater JL, Gopalakrishnan V, Wolff JE. Influence of valproic acid on outcome of high-grade gliomas in children. Anticancer Res. 2008 Jul-Aug;28(4C):2437-42.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Amy L Bredlau, MD principal investigator
Medical University of South Carolina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Valproic Acid in people with Pediatric Brain Tumor and Glioma, run by Medical University of South Carolina 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 4 inclusion and 5 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: withdrawn, as of the sponsor's last update on 22 May 2015. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Completion of protocol (time frame: 2 months). 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 NCT01861990, which also lists 5 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 22 May 2015.