Refractory Solid Tumors in ChildrenPhase 1RAPIRINCT01282697
Clinical Trial of Rapamycin and Irinotecan in Pediatric Patients With Refractory Solid Tumors
A Phase 1 study testing Combined administration of irinotecan and rapamycin in people with Refractory Solid Tumors in Children, run by University Hospital, Strasbourg, France at 9 sites in France.
Trial summary
Therapeutic solutions to treat solid tumors that are resistant to conventional treatments are now limited. Laboratory data in animals (on pediatric tumors such as brain tumors, sarcomas and neuroblastomas) have shown that the combination of irinotecan (HIF1alpha inhibitor) and rapamycin (mTOR inhibitor) allowed to… Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Refractory Solid Tumors in Children
- What is being tested?
- Combined administration of irinotecan and rapamycin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 9 sitesFrance
- Study dates
- 22 Apr 2011to 20 Feb 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age >= 1 year old and =< 21 years old;
- Refractory solid tumors, histologically proven at diagnosis (no additional biopsy needs to be performed for the purpose of the study);
- Relapsed or refractory solid tumors after standard treatment or phase II, III-IV clinical trials treatment have failed;
- Karnofsky or Lansky status >= 70%;
- Life expectancy >= 8 weeks;
- No chemotherapy / radiotherapy within 4 weeks before entry into the study;
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- Adequate biological parameters :
- Absolute neutrophil count >= 1.0 x 109/L;
- Platelet count >= 100 x 109/L;
- Hemoglobin >= 8 mg/dL;
- Total bilirubine =< 1.5 ULN;
- Transaminases =< 2.5 ULN (=< 5 ULN in case of liver metastases);
- Creatinine clearance (Cockroft) >= 70 mL/min/1.73 m2;
- Normal coagulation profile with prothrombin >= 70%, TCA =< 35 and fibrinogen >= 2 g/L;
- Patients with 1 to 3 previous therapeutic lines are eligible;
- No current grade >= 2 organ toxicity based on NCI-CTCAE version 3.0;
- All patients with reproductive potential must have an effective method of birth control while on study;
- Negative pregnancy test in females when indicated;
- Informed written consent signed by patients or their parents or legal guardians;
- Patient who was informed of the results of prior medical consultation;
- Patient having a social insurance.
Who cannot join
- Patient with a constitutional anomaly of coagulation and/or of hemostasis (type hemophilia, von Willebrand disease, congenital clotting factor deficit, platelet disorder), exposing them to increased risk of bleeding;
- Pre-treatment with a mTOR inhibitor;
- Other simultaneous malignancy;
- Concurrent administration of any other anti-tumour therapy;
- Known hypersensitivity or contraindication to study drugs or ingredients;
- Severe concomitant disease (e.g. infection disease);
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- Patient unable for medical follow-up;
- Pregnancy and/or lactation;
- Patient included in another clinical drug trial;
- Patient taking drugs interfering with pharmacology of rapamycin and/or irinotecan (e.g. drugs interfering with CYP3A4);
- Patient under judicial protection.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
France9 sites
Hôpital des Enfants - Groupe Hospitalier Pellegrin
Bordeaux, 33076
Centre Oscar Lambret
Lille, 59020
Institut Hémato-Oncologie Pédiatrique (IHOP)
Lyon, 67008
CHU La Timone
Marseille, 13005
CHU Mère-Enfants
Nantes, 44093
Institut Curie
Paris, 75005
Hôpitaux Universitaires de Strasbourg
Strasbourg, 67098
Hôpital des Enfants
Toulouse, 31059
Institut Gustave Roussy
Villejuif, 94805
About this study
Official title: Phase I Clinical Trial of Rapamycin and Irinotecan in Pediatric Patients With Refractory Solid Tumors
Therapeutic solutions to treat solid tumors that are resistant to conventional treatments are now limited. Laboratory data in animals (on pediatric tumors such as brain tumors, sarcomas and neuroblastomas) have shown that the combination of irinotecan (HIF1alpha inhibitor) and rapamycin (mTOR inhibitor) allowed to block development of blood vessels in the tumor and could, in some cases, stop its progression. This drug combination has already been tested in adult patients with refractory tumors and seems to give encouraging results with stabilization of the tumor. The dose and toxicity of irinotecan and rapamycin are known when these drugs are administered separately and in a context different from that of refractory tumors. RAPIRI is a phase I clinical trial whose principal objectives are to determine the maximum dose at which these two molecules may be administered and to assess the safety of this new combination of drugs.
- Conditions
- Refractory Solid Tumors in Children
- Being tested
- Combined administration of irinotecan and rapamycin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Combined administration of irinotecan and rapamycinDrug
This phase I trial is a dose escalation study of irinotecan + rapamycin with a 3+3 statistical design.
| Study group | Role | What they receive |
|---|---|---|
| rapamycin+irinotecan at a given dose | Experimental | Not described Receives: Drug: Combined administration of irinotecan and rapamycin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the maximum tolerated dose (MTD) of irinotecan and rapamycin combination in children with refractory solid tumors.
The Dose-Limiting Toxicity (DLT) of the drug combination is determined during the first cycle (J1 to J28) of treatment. MTD will be defined as the dose level immediately below the dose level at which 2 patients in a cohort of 3 to 6 patients will have experienced a DLT.
Measured: 28 days
Characterize the pharmacokinetics of rapamycin and irinotecan during the first cycle of treatment.
Pharmacokinetic parameters for rapamycin will be evaluated at days 1 and 8 of the first cycle of treatment. Pharmacokinetic parameters for irinotecan will be evaluated at day 1 of the first cycle of treatment. Pharmacokinetic profile will be modelized for each patient.
Measured: Day1 + day8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2011 (estimated)
- Study start22 Apr 2011
- Primary completion20 Feb 2013
- Study completion20 Feb 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Refractory Solid Tumors in ChildrenStudies of the same condition, open and under way.
- More trials run by University Hospital, Strasbourg, FranceThe 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Strasbourg, France
- Sponsor type
- University, hospital or other
- Collaborators
- Gustave Roussy, Cancer Campus, Grand Paris
- Responsible party
- Sponsor
- Investigators
- Natacha ENTZ-WERLE, MD, PhD, Principal investigator, Hôpitaux Universitaires de Strasbourg
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01282697
- Sponsor's protocol ID
- 4791
- Other IDs
- 2010-022329-13
- Acronym
- RAPIRI
- Status last verified
- Dec 2025
- First submitted
- 12 Nov 2010
- Last updated
- 8 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Jannier S, Kemmel V, Sebastia Sancho C, Chammas A, Sabo AN, Pencreach E, Farace F, Chenard MP, Lhermitte B, Geoerger B, Aerts I, Frappaz D, Leblond P, Andre N, Ducassou S, Corradini N, Bertozzi AI, Guerin E, Vincent F, Velten M, Entz-Werle N. SFCE-RAPIRI Phase I Study of Rapamycin Plus Irinotecan: A New Way to Target Intra-Tumor Hypoxia in Pediatric Refractory Cancers. Cancers (Basel). 2020 Oct 20;12(10):3051. doi: 10.3390/cancers12103051.
PubMed 33092063
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Clinical Trial of Rapamycin and Irinotecan in Pediatric Patients With Refractory Solid Tumors
- ClinicalTrials.gov ID
- NCT01282697
- Acronym / sponsor ID
- RAPIRI / 4791
- Conditions
- Refractory Solid Tumors in Children
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- University Hospital, Strasbourg, FranceUniversity, hospital or other
- Collaborators
- Gustave Roussy, Cancer Campus, Grand Paris
- Study start
- 22 Apr 2011 (actual)
- Primary completion
- 20 Feb 2013 (actual)
- Study completion
- 20 Feb 2013 (actual)
- First posted
- 25 Jan 2011 (estimated)
- 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.
Therapeutic solutions to treat solid tumors that are resistant to conventional treatments are now limited. Laboratory data in animals (on pediatric tumors such as brain tumors, sarcomas and neuroblastomas) have shown that the combination of irinotecan (HIF1alpha inhibitor) and rapamycin (mTOR inhibitor) allowed to block development of blood vessels in the tumor and could, in some cases, stop its progression. This drug combination has already been tested in adult patients with refractory tumors and seems to give encouraging results with stabilization of the tumor. The dose and toxicity of irinotecan and rapamycin are known when these drugs are administered separately and in a context different from that of refractory tumors. RAPIRI is a phase I clinical trial whose principal objectives are to determine the maximum dose at which these two molecules may be administered and to assess the safety of this new combination of drugs.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rapamycin+irinotecan at a given dose | Experimental | Drug: Combined administration of irinotecan and rapamycin |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (21 listed)
- Age >= 1 year old and =< 21 years old;
- Refractory solid tumors, histologically proven at diagnosis (no additional biopsy needs to be performed for the purpose of the study);
- Relapsed or refractory solid tumors after standard treatment or phase II, III-IV clinical trials treatment have failed;
- Karnofsky or Lansky status >= 70%;
- Life expectancy >= 8 weeks;
Key exclusion criteria (11 listed)
- Patient with a constitutional anomaly of coagulation and/or of hemostasis (type hemophilia, von Willebrand disease, congenital clotting factor deficit, platelet disorder), exposing them to increased risk of bleeding;
- Pre-treatment with a mTOR inhibitor;
- Other simultaneous malignancy;
- Concurrent administration of any other anti-tumour therapy;
- Known hypersensitivity or contraindication to study drugs or ingredients;
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 |
|---|---|---|
| Combined administration of irinotecan and rapamycinThis phase I trial is a dose escalation study of irinotecan + rapamycin with a 3+3 statistical design. | Drug | rapamycin+irinotecan at a given dose |
Primary outcome
Determine the maximum tolerated dose (MTD) of irinotecan and rapamycin combination in children with refractory solid tumors.
Time frame: 28 days
The Dose-Limiting Toxicity (DLT) of the drug combination is determined during the first cycle (J1 to J28) of treatment. MTD will be defined as the dose level immediately below the dose level at which 2 patients in a cohort of 3 to 6 patients will have experienced a DLT.
Characterize the pharmacokinetics of rapamycin and irinotecan during the first cycle of treatment.
Time frame: Day1 + day8
Pharmacokinetic parameters for rapamycin will be evaluated at days 1 and 8 of the first cycle of treatment. Pharmacokinetic parameters for irinotecan will be evaluated at day 1 of the first cycle of treatment. Pharmacokinetic profile will be modelized for each patient.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2026.
- Results publication: PubMed 33092063Jannier S, Kemmel V, Sebastia Sancho C, Chammas A, Sabo AN, Pencreach E, Farace F, Chenard MP, Lhermitte B, Geoerger B, Aerts I, Frappaz D, Leblond P, Andre N, Ducassou S, Corradini N, Bertozzi AI, Guerin E, Vincent F, Velten M, Entz-Werle N. SFCE-RAPIRI Phase I Study of Rapamycin Plus Irinotecan: A New Way to Target Intra-Tumor Hypoxia in Pediatric Refractory Cancers. Cancers (Basel). 2020 Oct 20;12(10):3051. doi: 10.3390/cancers12103051.
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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. This study is not recruiting at the moment.
Natacha ENTZ-WERLE, MD, PhD principal investigator
Hôpitaux Universitaires de Strasbourg
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Combined administration of irinotecan and rapamycin in people with Refractory Solid Tumors in Children, run by University Hospital, Strasbourg, France at 9 sites 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 21 inclusion and 11 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: finished, as of the sponsor's last update on 8 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the maximum tolerated dose (MTD) of irinotecan and rapamycin combination in children with refractory solid tumors. (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01282697, which also lists 1 linked publication.
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 8 Jun 2026.