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Oral Irinotecan with Temozolomide for Recurrent Pediatric Solid Tumors

Pilot Pharmacokinetic Study of VAL-413 (Orotecan®) in Patients With Recurrent Pediatric Solid Tumors

Phase 1 Interventional Valent Technologies, LLC · NCT04337177

This study is testing a new flavored oral treatment combining irinotecan and temozolomide to see if it helps kids and young adults with recurring solid tumors like neuroblastoma and rhabdomyosarcoma.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment20 (estimated)
Ages1 Year to 30 Years
SexAll
SponsorValent Technologies, LLC Industry-sponsored
Drugs / interventionschemotherapy, immunotherapy, Radiation
Locations8 sites (San Francisco, California and 7 other locations)
Trial IDNCT04337177 on ClinicalTrials.gov

What this trial studies

This pilot trial evaluates the safety and efficacy of a flavored oral formulation of irinotecan (VAL-413) combined with temozolomide for treating recurrent pediatric solid tumors, including neuroblastoma and rhabdomyosarcoma. Up to 20 patients aged 1 to 30 years will receive a specific dosing regimen of VAL-413 and temozolomide over multiple treatment cycles. The study will monitor patient responses through medical histories, physical exams, and imaging evaluations to assess the treatment's effectiveness and safety. The goal is to gather data that could inform future treatment options for this vulnerable population.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients aged 1 to 30 years with recurrent solid tumors for which there is no known curative therapy.

Not a fit: Patients with solid tumors that have effective curative therapies or those who do not meet the age or performance status criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new oral therapy option for children with recurrent solid tumors, potentially improving their quality of life and treatment outcomes.

How similar studies have performed: While this approach is novel in its specific combination and formulation, similar studies have shown promise in treating pediatric solid tumors with irinotecan and temozolomide.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Patients must be 1 year of age to ≤ 30 years of age at the time of study entry.
2. Patients must have had histologic verification of a solid tumor or CNS tumor at either original diagnosis or relapse.
3. Measurable or evaluable disease is not required for enrollment on this safety/feasibility study.
4. Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life or for which irinotecan and/or temozolomide are acceptable therapeutic options based on existing standard of care available.
5. Karnofsky Performance Status ≥ 50% for patients \> 16 years of age and Lansky Performance Status ≥ 50 for patients ≤ 16 years of age. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
6. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraception method during and for 30 days after study treatment. (Abstinence is considered an acceptable method of effective contraception.)
7. Prior treatment with temozolomide, vincristine or irinotecan is allowed, although patients must not have had disease progression while receiving either irinotecan, vincristine or temozolomide.
8. Patients must have recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study, as described below:

   1. Myelosuppressive chemotherapy: patients must not have received myelosuppressive chemotherapy within 21 days of first study treatment, but nitrosourea within 8 weeks (42 days) of first study treatment
   2. Anti-cancer agents not known to be myelosuppressive (e.g., not associated with drops in platelet or neutrophil count): must not have received these therapies within 7 days of first study treatment, or at least 5 half-lives of the agent (whichever is longer)
   3. Antibody therapy: At least 4 weeks must have elapsed since last antibody dose prior to first study treatment
   4. Radiation therapy: At least two weeks must have elapsed since last local palliative radiation (small port) prior to first study treatment. At least 6 weeks must have elapsed if more substantial radiation was administered (e.g., \>50% pelvis, craniospinal, whole body), or therapeutic radiolabeled 131I MIBG or other radiopharmaceutical therapy.
   5. High-Dose Chemotherapy with Autologous Stem Cell Transplant/Rescue: At least two months must have elapsed since receiving autologous hematopoietic stem cells prior to first study treatment. Patients who have had allogeneic transplants are ineligible.
   6. Hematopoietic growth factors: must not have been received in the 14 days prior to first study treatment for a long-acting growth factor (e.g., pegfilgrastim), or 7 days prior to first study treatment for short-acting growth factor.
9. Peripheral absolute neutrophil count (ANC) ≥ 1,000/µL
10. Platelet count ≥100,000/µL (transfusion independent, defined as not receiving platelet transfusions within a 7-day period prior to first study treatment)
11. Hemoglobin ≥ 8.0 gm/dL (may receive RBC transfusions) NOTE: Patients with metastatic tumor in the bone marrow ARE eligible provided the above hematologic criteria are met.
12. Creatinine clearance or radioisotope GFR ≥ 70mL/min/1.73 m2 or Serum creatinine based on age/gender as follows:

    Age Maximum Serum Creatinine (mg/dL) Male Female 1 to \< 2 years 0.6 0.6 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4

    ≥ 16 years 1.7 1.4

    The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC.
13. Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age
14. SGPT (ALT) ≤ 5 x upper limit of normal (ULN) for age
15. Serum albumin ≥ 2 g/dL

Exclusion Criteria:

1. Patients with a history of severe allergic reaction (e.g., more than simple rash) to dacarbazine or third-generation cephalosporins are ineligible.
2. Pregnant or breast-feeding women will not be entered on this study due to potential risks of fetal and teratogenic adverse events. A pregnancy test must be obtained prior to starting chemotherapy in post-menarchal female patients.
3. Patients who are currently receiving investigational drugs, or who have received an investigational drug within the last 7 days prior to first study treatment, are ineligible.
4. Patients who are currently receiving other anti-cancer agents are ineligible.
5. Patients taking strong inducers of CYP3A4, including but not limited to phenobarbital, phenytoin, carbamazepine, oxcarbazepine (Trileptal), rifampin, voriconazole, itraconazole, ketoconazole or other systemically-administered azole antifungal drugs, aprepitant (Emend) or St. John's Wort, in the 2 weeks prior to first study treatment are ineligible.
6. Patients taking strong inhibitors of CYP3A4 or UGT1A1 in the 1 week prior to first study treatment are ineligible.
7. Patients must not be receiving medications known to inhibit platelet function or known to selectively inhibit cyclooxygenase activity. Medicines in this class are excluded, with the exception of acetaminophen.
8. Patients who have uncontrolled infections, require IV antibiotics at time of enrollment, or who are currently receiving treatment for Clostridium difficile infection are excluded.
9. Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.

Where this trial is running

San Francisco, California and 7 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Solid TumorsNeuroblastomaRhabdomyosarcomaEwing SarcomaHepatoblastomaMedulloblastomairinotecanpediatric
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.