ACSE-ESMART

Phase 1

• Conditions: Relapsed or refractory tumors in children; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2016-000133-40-NL
• Lead Sponsor: Gustave Roussy

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-12-07
• Last Update Posted: 2 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 460

Inclusion Criteria

1.Patients must be diagnosed with hematologic or solid tumor malignancy that has progressed or relapsed despite standard therapy, or for which no effective standard therapy exists.
2.Age <18 years at inclusion.
3.Patient must have had advanced molecular profiling (i.e. WES/WGS +/- RNAseq) of their recurrent or refractory tumor i.e. at the time of disease progression/relapse; exceptionally patients with advanced molecular profiling at diagnosis may be allowed.
4.Evaluable or measurable disease as defined by standard imaging criteria for the patient’s tumor type.
5.Performance status: Karnofsky performance status (for patients >12 years of age) or Lansky Play score (for patients =12 years of age) =70%.
6.Life expectancy =3 months.
7.Adequate organ function
8.Able to comply with scheduled follow-up and with management of toxicity.
9.Females of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to initiation of treatment.
10.For all oral medications patients must be able to comfortably swallow capsules or tablets
11.Written informed consent from parents/legal representative, patient, and age-appropriate assent before any study-specific screening procedures are conducted according to local, regional or national guidelines.
12.Patient affiliated to a social security regimen or beneficiary of the same according to local requirements.

Are the trial subjects under 18? yes
Number of subjects for this age range: 460
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 81
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Patients with symptomatic central nervous system (CNS) metastases who are neurologically unstable or require increasing doses of corticosteroids or local CNS-directed therapy to control their CNS disease.
2.Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, or malabsorption syndrome).
3.Clinically significant, uncontrolled heart disease (including history of any cardiac arrhythmias).
4.Active viral hepatitis or known human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
5.Presence of any =CTCAE grade 2 treatment-related toxicity with the exception of alopecia, ototoxicity or peripheral neuropathy.
6.Systemic anticancer therapy within 21 days of the first study dose or 5 times its half-life, whichever is less.
7.Previous myeloablative therapy with autologous hematopoietic stem cell rescue within 8 weeks of the first study drug dose
8.Allogeneic stem cell transplant within 3 months prior to the first study drug dose. Patients receiving any agent to treat or prevent graft-versus host disease (GVHD) post bone marrow transplant are not eligible for this trial.
9.Radiotherapy (non-palliative) within 21 days prior to the first dose of drug (or within 6 weeks for therapeutic doses of MIBG or craniospinal irradiation).
10.Major surgery within 21 days of the first dose.
11.Currently taking medications with a known risk of prolonging the QT interval or inducing Torsades de Pointes.
12.Known hypersensitivity to any study drug or component of the formulation.
13.Pregnant or nursing (lactating) females.
14.Vaccinated with live, attenuated vaccines within 4 weeks of the first dose of study drug.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified