Your session is about to expire
← Back to Search
Anti-tumor antibiotic
PEN-866 + Chemotherapy for Sarcoma
Phase 1 & 2
Recruiting
Led By Christine M Heske, M.D.
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
For phase 2, participants must have histologically or cytologically confirmed recurrent or refractory Ewing sarcoma (Cohort 2) or embryonal or alveolar rhabdomyosarcoma (Cohort 3). Participants with Ewing sarcoma (Cohort 2 only) should have evidence of EWS translocation by FISH or RT-PCR.
Phase 2: Histologically or cytologically confirmed recurrent or refractory Ewing sarcoma (Cohort 2) or embryonal or alveolar rhabdomyosarcoma (Cohort 3)
Must not have
Requirement of medication with any of the inhibitors of UGT1A1, substrates of CYP1A2 or substrates of the P-gp, BCRP, OATP1B1, OATP1B3, or OCT1 transporters, or any prohibited medications
Known bone marrow metastatic disease causing decreased blood counts below the hematologic parameters for phase 1
Timeline
Screening 3 weeks
Treatment Varies
Follow Up pre/post infusion on cycle 1 day 1, 24 hr post infusion, cycle 1 day 4, cycle 1 day 8. cycle 3 day 1 and cycle 3 day 8
Awards & highlights
No Placebo-Only Group
Summary
This trial is studying the combination of 3 drugs to see if it can be used to treat adolescents and young adults with solid tumors, Ewing sarcoma, or rhabdomyosarcoma that returned after or did not respond to standard treatments.
Who is the study for?
Adolescents and young adults aged 12-39 with solid tumors, Ewing sarcoma, or rhabdomyosarcoma that have not responded to standard treatments are eligible. They must have adequate organ function, no severe prior treatment toxicity unresolved, and agree to use contraception if applicable. Those with certain genetic conditions or on prohibited medications cannot join.
What is being tested?
The trial tests a combination of PEN-866 with vincristine and temozolomide in patients who've had relapsed or refractory solid tumors. Over up to 18 cycles (21 days each), participants receive these drugs intravenously and orally while their health is monitored through exams, questionnaires, and lifelong follow-ups.
What are the potential side effects?
Potential side effects include reactions related to the infusion process, possible organ inflammation due to drug interactions, fatigue from treatment regimens, digestive issues from oral medication intake as well as blood disorders stemming from the chemotherapy agents.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have confirmed recurrent or refractory Ewing sarcoma or rhabdomyosarcoma.
Select...
I have a confirmed diagnosis of Ewing sarcoma or rhabdomyosarcoma that has come back or did not respond to treatment.
Select...
I have a confirmed solid tumor that has come back or didn't respond to treatment, but it's not in my brain or lymph nodes.
Select...
I can take care of myself but might not be able to do any heavy work.
Select...
I have had treatment for brain metastases.
Select...
It's been over 3 weeks since my last chemotherapy that affects my bone marrow.
Select...
I have a confirmed solid tumor that has come back or did not respond to treatment, excluding brain tumors and lymphoma.
Select...
I am between 12 and 39 years old.
Select...
My cancer can be seen on scans or confirmed through tests.
Select...
I am willing to have a central line placed if I don't already have one.
Select...
I have new or worsening brain metastases or leptomeningeal disease.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I am not taking any medications that interfere with specific body enzymes or transporters.
Select...
My cancer has spread to my bone marrow, affecting my blood counts.
Select...
I have not had major surgery in the last 28 days.
Select...
I stopped taking vincristine, temozolomide, or irinotecan due to severe side effects.
Select...
I have had severe nerve damage or constipation from vincristine.
Select...
I do not have any unmanaged ongoing illnesses.
Select...
My genetic test shows I have the UGT1A1 28/28 genotype.
Select...
I am not currently taking any experimental drugs or other cancer treatments.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from start of treatment to time of progression or death
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from start of treatment to time of progression or death
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Phase 1: Maximum tolerated/recommended phase 2 dose
Phase 2: Objective response rate
Secondary study objectives
Phase 1: Plasma and tumor pharmacokinetics
Phase 1: Toxicity
Phase 2: Duration of response
+2 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: 2/MTD/RP2DExperimental Treatment3 Interventions
PEN-866 at the MTD or RP2D from phase 1 plus vincristine and temozolomide
Group II: 1/Dose EscalationExperimental Treatment3 Interventions
Dose escalation of PEN-866 along with fixed doses of vincristine and temozolomide
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vincristine
2003
Completed Phase 4
~2970
Temozolomide
2010
Completed Phase 3
~1880
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,956 Previous Clinical Trials
41,112,033 Total Patients Enrolled
88 Trials studying Rhabdomyosarcoma
13,134 Patients Enrolled for Rhabdomyosarcoma
Christine M Heske, M.D.Principal InvestigatorNational Cancer Institute (NCI)
1 Previous Clinical Trials
14 Total Patients Enrolled
1 Trials studying Rhabdomyosarcoma
14 Patients Enrolled for Rhabdomyosarcoma
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have confirmed recurrent or refractory Ewing sarcoma or rhabdomyosarcoma.I have had multiple treatments for my condition.I am not taking any medications that interfere with specific body enzymes or transporters.You are currently pregnant or breastfeeding.My cancer diagnosis or relapse has been confirmed with a tissue sample.For phase 2, you must have a disease that can be measured by a specific standard.I have a confirmed diagnosis of Ewing sarcoma or rhabdomyosarcoma that has come back or did not respond to treatment.I have a confirmed solid tumor that has come back or didn't respond to treatment, but it's not in my brain or lymph nodes.I can take care of myself but might not be able to do any heavy work.I have recovered from the major side effects of my previous cancer treatments.I am HIV positive, on treatment, and my viral load has been undetectable for the last 6 months.I have chronic hepatitis B but my viral load is undetectable due to ongoing treatment.My cancer has spread to my bone marrow, affecting my blood counts.I have not had major surgery in the last 28 days.Side effects from my previous treatments are mild, except for nerve issues and hair loss.I stopped taking vincristine, temozolomide, or irinotecan due to severe side effects.My Ewing sarcoma has a specific genetic change.I am willing to have a tumor biopsy and I am 18 or older.I am between 12-17 years old and can have a biopsy with little risk.I have had multiple treatments for my condition.I have had severe nerve damage or constipation from vincristine.I haven't had treatment with both irinotecan and temozolomide together.I have had treatment for brain metastases.I can provide a tissue sample or slides of my tumor for testing.I had hepatitis C but it's either cured or currently undetectable.I am eligible for new treatments after a certain time since my last therapy.It's been over 3 weeks since my last chemotherapy that affects my bone marrow.I have recovered from the side effects of my previous cancer treatments.It's been over a week since my last non-myelosuppressive cancer treatment.My cancer can be seen on scans or has signs like high tumor markers.I do not have any unmanaged ongoing illnesses.My genetic test shows I have the UGT1A1 28/28 genotype.I have a confirmed solid tumor that has come back or did not respond to treatment, excluding brain tumors and lymphoma.I am between 12 and 39 years old.My cancer can be seen on scans or confirmed through tests.I am willing to have a central line placed if I don't already have one.My organs and bone marrow are working well.I am not currently taking any experimental drugs or other cancer treatments.I have new or worsening brain metastases or leptomeningeal disease.I have not been treated with both irinotecan and temozolomide together before.You have had allergic reactions to drugs with similar ingredients as PEN-866 or other drugs used in the study.
Research Study Groups:
This trial has the following groups:- Group 1: 1/Dose Escalation
- Group 2: 2/MTD/RP2D
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.