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Anti-metabolite
Combination Therapy for Brain Cancer
Phase 1
Waitlist Available
Led By Giles W. Robinson, MD
Research Sponsored by St. Jude Children's Research Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participants with recurrent, progressive, or refractory CNS tumors as confirmed through central pathology review and whose diagnosis is being treated on this study
Participants with recurrent, progressive, or refractory Non-WNT Non-SHH (NWNS) Medulloblastoma or Ependymoma as confirmed through central pathology review
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 month after start of therapy
Awards & highlights
Study Summary
This trial will help improve cure rates for children with brain tumors by studying how the tumors continue to resist modern advances and by testing new approaches to treatment.
Who is the study for?
This trial is for children and young adults aged 1 to less than 25 years with recurrent brain tumors, except those with specific low-grade gliomas or retinal vein occlusion. Participants must have completed previous cancer treatments at least 4 weeks prior, be able to swallow medication (or use a g-tube), not be pregnant or breastfeeding, and agree to use effective contraception. They should also have stable health without serious medical issues that could affect the study.Check my eligibility
What is being tested?
The SJDAWN trial tests combinations of drugs like Gemcitabine, Trametinib, Filgrastim, Sonidegib, and Ribociclib on various types of recurrent brain tumors in children and young adults. It aims to find safe dosages and see how well these drug pairs work together based on tumor type and molecular characteristics.See study design
What are the potential side effects?
Potential side effects may include reactions related to the immune system's response to treatment, blood cell count changes leading to increased infection risk or bleeding problems, liver function changes causing fatigue or yellowing skin/eyes (jaundice), heart rhythm irregularities (QTc prolongation), as well as general discomforts such as nausea or rash.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My brain tumor is growing or returning despite treatment.
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My tumor is a specific type of brain cancer that has not responded to treatment.
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I am between 1 and 24 years old.
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I am not currently taking medications that strongly affect liver enzymes or that can alter my heart rhythm.
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I am not pregnant, can take a pregnancy test, and will use effective birth control during and 8 months after the study.
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My medulloblastoma is worsening or not responding to treatment, and my tumor has specific genetic changes.
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I can swallow pills.
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My weight and body surface area meet the trial's requirements.
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I am between 10 and 40 years old.
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I am between 1 and 24 years old.
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My brain tumor is growing or has come back despite treatment.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 month after start of therapy
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 month after start of therapy
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Estimate the Maximum tolerated dose (MTD)/Recommended Phase 2 Dose (RP2D) of each doublet by stratum
Pharmacokinetics of combination treatment: Stratum A
Pharmacokinetics of combination treatment: Stratum B
+1 moreSecondary outcome measures
Duration of objective response by stratum
Response rate by stratum
Trial Design
3Treatment groups
Experimental Treatment
Group I: C: ribociclib + sonidegibExperimental Treatment2 Interventions
Stratum C participants with refractory or recurrent medulloblastoma (SHH-activated) >6 months off smoothened inhibitor, presence of 9q loss or PTCH1 mutant, skeletally mature. They received combination treatment with ribociclib and sonidegib.
Stratum C is being closed due to low accrual
Group II: B: ribociclib + trametinibExperimental Treatment2 Interventions
Stratum B participants with a diagnosis of one of the following refractory or recurrent CNS diseases: medulloblastoma, [sonic hedgehog (SHH)- or WNT-activated];; high grade glioma (including: high grade glioma, (NOS), WHO Grade III or IV; anaplastic astrocytoma, IDH mutant; glioblastoma, IDH-wildtype; glioblastoma, IDH-mutant; diffuse midline glioma, H3K27-mutant; anaplastic oligodendroglioma, IDH mutant and 1p/19q-codeleted; anaplastic pleomorphic xanthoastrocytoma); select CNS embryonal tumors (including: embryonal tumors with multilayered rosettes, C19MC-altered; embryonal tumors with multilayered rosettes, NOS; medulloepithelioma; CNS neuroblastoma; CNS ganglioneuroblastoma; CNS embryonal tumor, NOS; atypical teratoid/rhabdoid tumor; CNS embryonal tumor with rhabdoid features). They receive combination treatment with ribociclib and trametinib.
Stratum B has completed all the necessary accrual
Group III: A: ribociclib + gemcitabineExperimental Treatment3 Interventions
Stratum A participants with a diagnosis of refractory or recurrent medulloblastoma (Group 3/4) or refractory or recurrent ependymoma. (including: ependymoma, not otherwise specified (NOS), WHO Grade III; ependymoma, RELA fusion positive; anaplastic ependymoma; ependymoma, NOS, WHO grade II). They receive combination treatment with ribociclib and gemcitabine. They may also receive growth therapy support with filgrastim.
Stratum A has completed all the necessary accrual
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
filgrastim
1997
Completed Phase 3
~7260
Gemcitabine
2017
Completed Phase 3
~2070
ribociclib
2016
Completed Phase 3
~560
trametinib
2012
Completed Phase 2
~200
Find a Location
Who is running the clinical trial?
St. Jude Children's Research HospitalLead Sponsor
427 Previous Clinical Trials
5,306,509 Total Patients Enrolled
13 Trials studying Medulloblastoma
2,828 Patients Enrolled for Medulloblastoma
Novartis PharmaceuticalsIndustry Sponsor
2,855 Previous Clinical Trials
4,197,285 Total Patients Enrolled
6 Trials studying Medulloblastoma
503 Patients Enrolled for Medulloblastoma
Giles W. Robinson, MDPrincipal InvestigatorSt. Jude Children's Research Hospital
3 Previous Clinical Trials
273 Total Patients Enrolled
2 Trials studying Medulloblastoma
141 Patients Enrolled for Medulloblastoma
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My brain tumor is growing or returning despite treatment.I have not taken smoothened inhibitors in the last 6 months.I have a history of serious heart disease that is not under control.You have a history of prolonged QTc interval (more than 450 milliseconds).You have other important health issues that could make it hard for you to handle the treatment or affect the study results.Your heart's pumping ability is significantly lower than normal.My tumor is a specific type of brain cancer that has not responded to treatment.I am between 1 and 24 years old.I am not pregnant, can take a pregnancy test, and will use effective birth control during and after the study.My bones have fully developed according to my age.I am a man who can father children and agree to use contraception during and for 8 months after treatment.I have been on a stable or decreasing dose of corticosteroids for at least 1 week.I can do some activities and am expected to live at least 6 more weeks.My cancer can be measured or seen on scans.I received my last cancer treatment over 4 weeks ago.I finished my last radiation session at least 4 weeks ago, or 2 weeks ago if it was for symptom relief.I am not currently taking medications that strongly affect liver enzymes or that can alter my heart rhythm.I am not pregnant, can take a pregnancy test, and will use effective birth control during and 8 months after the study.I have been treated with a CDK4/6 inhibitor before.I am currently breastfeeding.I have been diagnosed with subependymoma or myxopapillary ependymoma.I have been diagnosed with Low Grade Glioma or Diffuse Intrinsic Pontine Glioma.I can swallow pills or have a g-tube for medication.My medulloblastoma is worsening or not responding to treatment, and my tumor has specific genetic changes.I have been treated with a MEK inhibitor before.I have been treated with a MEK inhibitor before.I can swallow pills.My weight and body surface area meet the trial's requirements.I am between 10 and 40 years old.Your heart's electrical activity (measured by ECG) shows a prolonged QTc interval (> 450 msec).I am between 1 and 24 years old, or up to 39 years old with specific brain cancer.I have a confirmed RB1 gene mutation.I have been diagnosed with retinal vein occlusion.I am a man willing to use contraception during and for 16 weeks after treatment.I am between 1 and 24 years old.My condition is a recurring or worsening low grade brain tumor.I can understand and am willing to sign the consent form.My brain tumor is growing or has come back despite treatment.My bone marrow and organs are functioning well.I have stopped taking any herbal or dietary supplements, except multivitamins, 7 days before joining the study.
Research Study Groups:
This trial has the following groups:- Group 1: A: ribociclib + gemcitabine
- Group 2: B: ribociclib + trametinib
- Group 3: C: ribociclib + sonidegib
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many persons have enrolled in this clinical inquiry?
"Yes, the data on clinicaltrials.gov shows that this medical study is currently in search of participants. This trial initially went live on March 5th 2018 and was last revised on April 1st 2022. There are 108 spots open across a single site for participation."
Answered by AI
What has ribociclib been demonstrated to be efficacious for?
"Primarily used to combat infection, ribociclib can also be employed for urinary bladder issues, stem cell mobilization therapy and small-cell lung cancer treatment."
Answered by AI
Are there any remaining slots available in this clinical trial?
"Clinicaltrials.gov demonstrates that this trial is still in search of patients; it was first published on the 5th March 2018 and last amended on 1st April 2022."
Answered by AI
What prior research has been conducted surrounding ribociclib's efficacy?
"At the moment, 737 clinical trials are being conducted for ribociclib. Of those active studies, 182 have been elevated to Phase 3. Most of these tests are located in Adelaide South Australia; however there is a total 36108 trial sites across the globe involved with this medication."
Answered by AI
Is it possible to join this clinical investigation?
"The eligibility requirements of this clinical trial necessitate candidates with ependymoma and aged between 1 year old to 39 years. A total of 108 patients must be recruited in order for the study's goals to be achieved."
Answered by AI
Is the usage of ribociclib associated with any known adverse effects?
"Whilst there is some evidence of efficacy, due to the trial being in Phase 1, limited data exists for ribociclib's safety. As a result our assessment rated it at level 1 on the scale from 1 to 3."
Answered by AI
Does this research encompass participants below the age of 20?
"To qualify for this clinical trial, the patient must fall between 1 Year and 39 Years of age. Additionally, 841 studies are available for those under 18 years old and 4485 exist for seniors above 65."
Answered by AI
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