Diffuse Intrinsic Pontine Glioma (DIPG)Phase 1NCT01688401
Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
A Phase 1 study testing Melphalan hydrochloride in people with Diffuse Intrinsic Pontine Glioma (DIPG), run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
Trial summary
The goal of this pilot study is to determine if intra-arterial (IA) chemotherapy is safe in the treatment of progressive diffuse intrinsic pontine gliomas (DIPG). IA administration of the chemotherapeutic agent enhances the regional distribution of the drug, thereby increasing the local delivered dose while minimizing… Read more about this study
- Who is it for?
- Ages 1 month to 17 years
- Conditions the study is for
- Diffuse Intrinsic Pontine Glioma (DIPG)
- What is being tested?
- Melphalan hydrochloride
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Mar 2013to 26 Nov 2018
- 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 month to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Pediatric patients of all ages with progressive DIPG.
- Consensus following presentation of the case at the multidisciplinary Pediatric Neuro-Oncology conference, which includes participation of neuro-oncology, neurosurgery, radiation oncology, interventional neuroradiology and neurology.
Who cannot join
- Documented hypercoagulable disorders or vasculopathies
- INR value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>1 - 1.5 x ULN; >1 - 1.5 times above baseline if on anticoagulation).
- APTT value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>ULN - 1.5 x ULN).
- Platelets less than 50 x 103/mm3
- Absolute neutrophil count less than 500/ mm3
- Pregnancy
- Documented severe allergic reaction to IV iodinated contrast, specifically bronchospasm and anaphylaxis.
Trial locations and contacts
1 site in 1 country.
United States1 site
The Johns Hopkins Hospital
Baltimore, Maryland, 21287
About this study
The goal of this pilot study is to determine if intra-arterial (IA) chemotherapy is safe in the treatment of progressive diffuse intrinsic pontine gliomas (DIPG). IA administration of the chemotherapeutic agent enhances the regional distribution of the drug, thereby increasing the local delivered dose while minimizing systemic toxicity. It also provides a treatment option for these patients at the time of tumor recurrence.
Read the detailed description
Delivering the chemotherapeutic agent directly to the tumor via the arterial system avoids the complications and adverse events associated with toxicity from systemic chemotherapy.
- Conditions
- Diffuse Intrinsic Pontine Glioma (DIPG)
- Being tested
- Melphalan hydrochloride
- Keywords
- diffuse intrinsic pontine glioma (DIPG), intra-arterial chemotherapy, angiography
- Medical subject headings
- Diffuse Intrinsic Pontine Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Melphalan hydrochlorideDrug
Also called Alkeran
Drug administered intra-arterially (injection in the artery).
Standard dose: Cycle 1: 1 mg, intra-arterial (IA) delivery. Cycle 2: 2 mg, intra-arterial (IA) delivery.
Duration of treatment: Eight weeks total - two cycles of IA chemotherapy, separated by four weeks.
| Study group | Role | What they receive |
|---|---|---|
| IA melphalan | Experimental | IA melphalan is administered via the basilar artery. Receives: Drug: Melphalan hydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Technical safety as determined by number of participants with toxicity
Number of participants with grades 3-5 intracranial hemorrhage, grades 3-5 stroke, as defined by the Nervous system disorder CTCAE, and requirement of blood transfusion.
Measured: 60 days
Secondary outcomes (1)
Long-term Efficacy as assessed by progression free survival
Number of months until disease progression.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2012 (estimated)
- Study start8 Mar 2013
- Primary completionMay 2017
- Study completion26 Nov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diffuse Intrinsic Pontine Glioma (DIPG)Studies of the same condition, open and under way.
- More trials run by Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsThe 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Solving Kids' Cancer
- Responsible party
- Sponsor
- Investigators
- Monica Pearl, M.D., Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01688401
- Sponsor's protocol ID
- J11164
- Other IDs
- NA_00069122
- Status last verified
- Mar 2019
- First submitted
- 10 Sep 2012
- Last updated
- 28 Mar 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
- ClinicalTrials.gov ID
- NCT01688401
- Acronym / sponsor ID
- J11164
- Conditions
- Diffuse Intrinsic Pontine Glioma (DIPG)
- Keywords
- diffuse intrinsic pontine glioma (DIPG), intra-arterial chemotherapy, angiography
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Collaborators
- Solving Kids' Cancer
- Study start
- 8 Mar 2013 (actual)
- Primary completion
- May 2017 (actual)
- Study completion
- 26 Nov 2018 (actual)
- First posted
- 19 Sep 2012 (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 goal of this pilot study is to determine if intra-arterial (IA) chemotherapy is safe in the treatment of progressive diffuse intrinsic pontine gliomas (DIPG). IA administration of the chemotherapeutic agent enhances the regional distribution of the drug, thereby increasing the local delivered dose while minimizing systemic toxicity. It also provides a treatment option for these patients at the time of tumor recurrence.
Detailed description
Delivering the chemotherapeutic agent directly to the tumor via the arterial system avoids the complications and adverse events associated with toxicity from systemic chemotherapy.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IA melphalan | Experimental | Drug: Melphalan hydrochlorideIA melphalan is administered via the basilar artery. |
Who may qualify
- Ages
- 1 month to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Pediatric patients of all ages with progressive DIPG.
- Consensus following presentation of the case at the multidisciplinary Pediatric Neuro-Oncology conference, which includes participation of neuro-oncology, neurosurgery, radiation oncology, interventional neuroradiology and neurology.
Key exclusion criteria (7 listed)
- Documented hypercoagulable disorders or vasculopathies
- Platelets less than 50 x 103/mm3
- Absolute neutrophil count less than 500/ mm3
- Pregnancy
- Documented severe allergic reaction to IV iodinated contrast, specifically bronchospasm and anaphylaxis.
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 |
|---|---|---|
| Melphalan hydrochlorideAlso: AlkeranDrug administered intra-arterially (injection in the artery). Standard dose: Cycle 1: 1 mg, intra-arterial (IA) delivery. Cycle 2: 2 mg, intra-arterial (IA) delivery. Duration of treatment: Eight weeks total - two cycles of IA chemotherapy, separated by four weeks. | Drug | IA melphalan |
Primary outcome
Technical safety as determined by number of participants with toxicity
Time frame: 60 days
Number of participants with grades 3-5 intracranial hemorrhage, grades 3-5 stroke, as defined by the Nervous system disorder CTCAE, and requirement of blood transfusion.
1 secondary outcome
Long-term Efficacy as assessed by progression free survival
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Mar 2019.
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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.
Monica Pearl, M.D. principal investigator
Johns Hopkins University
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 Melphalan hydrochloride in people with Diffuse Intrinsic Pontine Glioma (DIPG), run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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 2 inclusion and 7 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 28 Mar 2019. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Technical safety as determined by number of participants with toxicity (time frame: 60 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Mar 2019.