CNS Tumor, ChildhoodPRO-SACHANCT07738601
Patient Reported Outcome Measurement Within the Secured Access to Innovative Medicines for Children With cAncer (SACHA) Study
An observational study of CNS Tumor, Childhood, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- CNS Tumor, Childhood
- What is being tested?
- SSPedi - Symptom Screening in Pediatrics Questionnaire
- How many people
- 72 participants
- Where
- 1 siteFrance
- Study dates
- Sep 2026to Jul 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: 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
Who may be able to join
- 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
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- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site
Assistance Publique - Hôpitaux de Marseille
Marseille
Gabriel Revon-Rivière, Dr, 04 91 38 68 22 ext. 33, gabriel.revon-riviere@ap-hm.fr
About this study
Official title: PRO-SACHA: Patient Reported Outcome Measurement Within the Secured Access to Innovative Medicines for Children With cAncer (SACHA) Study
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.
Read more
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.
- Conditions
- CNS Tumor, Childhood
- Being tested
- SSPedi - Symptom Screening in Pediatrics Questionnaire
- Keywords
- Cancer, Patient Reported Outcomes, Pediatric malignancies, Off-label therapies, Innovative cancer therapies, Real-world evidence
- Medical subject headings
- Central Nervous System Neoplasms, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SSPedi - Symptom Screening in Pediatrics QuestionnaireOther
SSPedi responses (a self-report questionnaire capturing symptoms experienced by patients) are collected electronically through an online application
| Study group | Role | What they receive |
|---|---|---|
| Pediatric cancer cohort | Not described Receives: Other: SSPedi - Symptom Screening in Pediatrics Questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
Score of the Symptom Screening in Pediatrics Questionnaire (SSPedi)
Measure of the Symptom Screening in Pediatrics Questionnaire (SSPedi) scores for each patient, values ranging from 0 to 60, higher scores meaning worse outcome
Measured: Baseline and Day 1 of each Cycle until Cycle 6 (each Cycle is 22 days)
Secondary outcomes (1)
Adverse Events
Grade ≥2 clinical and Grade ≥3 laboratory adverse events assessed according to the CTCAE version 5 (Common Terminology Criteria for Adverse Events)
Measured: Baseline and Day 1 of each Cycle until Cycle 6 (each Cycle is 22 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study startSep 2026 (estimated)
- Primary completionJan 2029 (estimated)
- Study completionJul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CNS Tumor, ChildhoodStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07738601
- Sponsor's protocol ID
- RCAPHM25_0280
- Other IDs
- 2025-A02394-45
- Acronym
- PRO-SACHA
- Status last verified
- Jul 2026
- First submitted
- 22 Jul 2026
- Last updated
- 31 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PRO-SACHA: Patient Reported Outcome Measurement Within the Secured Access to Innovative Medicines for Children With cAncer (SACHA) Study
- ClinicalTrials.gov ID
- NCT07738601
- Acronym / sponsor ID
- PRO-SACHA / RCAPHM25_0280
- Conditions
- CNS Tumor, Childhood
- Keywords
- Cancer, Patient Reported Outcomes, Pediatric malignancies, Off-label therapies, Innovative cancer therapies, Real-world evidence
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Collaborators
- The Hospital for Sick Children, Gustave Roussy, Cancer Campus, Grand Paris
- Study start
- Sep 2026 (estimated)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Jul 2029 (estimated)
- First posted
- 31 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric cancer cohort | – | Other: SSPedi - Symptom Screening in Pediatrics Questionnaire |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 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
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SSPedi - Symptom Screening in Pediatrics QuestionnaireSSPedi responses (a self-report questionnaire capturing symptoms experienced by patients) are collected electronically through an online application | Other | Pediatric cancer cohort |
Primary outcome
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)
Measure of the Symptom Screening in Pediatrics Questionnaire (SSPedi) scores for each patient, values ranging from 0 to 60, higher scores meaning worse outcome
1 secondary outcome
Adverse Events
Time frame: Baseline and Day 1 of each Cycle until Cycle 6 (each Cycle is 22 days)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of CNS Tumor, Childhood, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Gabriel Revon-Rivière, Dr (04 91 38 68 22 ext. 33, gabriel.revon-riviere@ap-hm.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2026.