Patient Reported Outcome Measurement Within the Secured Access to Innovative Medicines for Children With cAncer (SACHA) Study

Official title

PRO-SACHA: Patient Reported Outcome Measurement Within the Secured Access to Innovative Medicines for Children With cAncer (SACHA) Study

Overview

Patient-Reported Outcomes (PROs) are patient-centered measures used to assess health status, track changes over time, and evaluate the impact of treatment on the patient's perceived health. The SACHA study is a French prospective observational study developed by the Société Française de lutte contre les Cancers de l'Enfant et de l'adolescent (SFCE). It prospectively collects real-world safety and activity data on novel therapies given to patients aged 25 or younger with pediatric malignancies (solid tumors or hematologic malignancies) or related conditions, outside of a clinical trial. The Symptom Screening in Pediatrics Tool (SSPedi) is a validated questionnaire for measuring patient-reported symptoms in pediatric oncology. It includes 15 questions covering common symptoms in pediatric cancer patients and one open-ended question allowing patients to report any other bothersome symptoms. Patients complete the questionnaire through an online application. The PRO-SACHA study aims to describe the symptoms reported by participants receiving novel therapies in pediatric oncology and to examine the concordance between participant-reported symptoms and symptomatic adverse events (AEs) reported by investigators. This prospective observational study evaluates patient-reported symptoms in patients aged 2 to 18 years enrolled in the SACHA study. Participation is voluntary, based on an opt-out consent model (French category 3 interventional research involving the human person). Planned enrollment: 72 participants over 18 months, with each participant followed for 7 months (a 6-month follow-up period, with a 7th-month window for questionnaire completion). Participants are enrolled in PRO-SACHA at the same time as their enrollment in SACHA (two separate studies with separate enrollment). SSPedi responses (a self-report questionnaire capturing symptoms experienced by patients) are collected electronically through an online application. The extracted, anonymized data are then correlated with adverse events reported by investigators. The concordance between participant-reported symptoms and adverse events recorded in SACHA (CTCAE grading) will be analyzed.

Interventions

• Other SSPedi - Symptom Screening in Pediatrics Questionnaire
  SSPedi responses (a self-report questionnaire capturing symptoms experienced by patients) are collected electronically through an online application

Primary outcome measures

• Score of the Symptom Screening in Pediatrics Questionnaire (SSPedi) [Time frame: Baseline and Day 1 of each Cycle until Cycle 6 (each Cycle is 22 days)]

Secondary outcome measures (1)

• Adverse Events [Time frame: Baseline and Day 1 of each Cycle until Cycle 6 (each Cycle is 22 days)]

Eligibility criteria

Inclusion criteria

• Aged 2 to 18 years
• Patients aged 8 to 18 years who can read French, English, or Spanish fluently OR patients aged 6 to 7 years who can read French, English, or Spanish fluently, OR patients aged 2 to 7 years who cannot read fluently but whose parents can read French, English, or Spanish fluently
• Patients included in the SACHA study before starting treatment
• Patients with a pediatric tumor or leukemia who have failed treatment or relapsed and have no standard treatment options available OR patients receiving first-line treatment with no standard treatment options available (e.g., inoperable plexiform neurofibroma and MEK inhibitors, infantile fibrosarcoma and NTRK inhibitors)
• Not eligible for an open early-phase clinical trial in France, or declining participation
• Treated with a new innovative medicinal product and discussed at a RCCPI meeting under a granted early access authorization or compassionate use authorization issued by the ANSM, or receiving off-label use of a medicinal product already authorized in adults
• Treated in one of the SFCE centers authorized to prescribe chemotherapy
• The holders of parental authority have stated their non-opposition to their child's participation in the study after being informed. Minors will receive age-appropriate information adapted to their level of understanding. If the minor is emancipated, they will provide non-opposition directly
• A patient who is prescribed another innovative therapy and is re-included in SACHA may be included again in PRO-SACHA

Exclusion criteria

• Patient who has already started the innovative treatment
• Patients aged 8 years and older who cannot read
• Patients whose clinical condition does not allow them to report symptoms themselves (i.e., non-communicative patient; judgment left to the investigator's discretion)
• Patients currently enrolled in an early-phase trial
• Refusal of participation by the patient or their legal representatives
• Patient unable to express consent to participate in the study

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

France · 1 center

• Assistance Publique - Hôpitaux de Marseille — Marseille

Identifiers

NCT: NCT07738601 · RCAPHM25_0280 · 2025-A02394-45

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗