Patient Reported Outcome Measurement Within the Secured Access to Innovative Medicines for Children With cAncer (SACHA) Study | NCT07738601

NCT07738601

Overview

NCT ID: NCT07738601
Lead Sponsor: Assistance Publique Hopitaux De MarseilleOTHER
Official Title: PRO-SACHA: Patient Reported Outcome Measurement Within the Secured Access to Innovative Medicines for Children With cAncer (SACHA) Study
Brief Summary: 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.

Status and Timeline

Status: Not yet recruiting
Last Update Posted: Jul 31, 2026Actual
Start Date: Sep 2026Estimated
Primary Completion Date: Jan 2029Estimated
Completion Date

Participation Criteria

Ages Eligible for Study: Child (0-17)Adult (18-64)
Sexes Eligible for Study: All
Accepts Healthy Volunteers?: No
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

Study Plan

Study Type: Observational
Phase: No data available
Enrollment: 72Estimated
Primary Purpose: No data available
Allocation: No data available
Interventional Model: No data available
Masking: No data available

Contacts and Locations

Central Contacts: Gabriel Revon-Rivière, DrContact
gabriel.revon-riviere@ap-hm.fr04 91 38 68 22 ext. 33

Overview

Status and Timeline

Participation Criteria

Study Plan

Contacts and Locations

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