TumorsPhase 1NCT00187174
Everolimus for Treating Pediatric Patients With Recurrent or Refractory Tumors
A Phase 1 study testing Everolimus in people with Tumors and Brain Tumors, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
Patients with recurrent or refractory solid tumors or brain tumors that are unresponsive to conventional therapy, or with no known effective therapy, will be treated. Experiments in the laboratory have shown the experimental drug RAD001C (RAD001, Everolimus) can prevent cells from multiplying. Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Tumors, Brain Tumors, Rhabdomyosarcoma
- What is being tested?
- Everolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2004to Oct 2008
- 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 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Adequate performance status
- Adequate bone marrow, kidney, heart, and liver function
Who cannot join
- Must not be receiving concomitant anti-cancer treatment
- Must not be pregnant
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Phase I Trial of RAD001C (Everolimus) in Pediatric Patients With Recurrent Refractory Solid Tumors or Brain Tumors
Patients with recurrent or refractory solid tumors or brain tumors that are unresponsive to conventional therapy, or with no known effective therapy, will be treated. Experiments in the laboratory have shown the experimental drug RAD001C (RAD001, Everolimus) can prevent cells from multiplying. RAD001 is now being tested in diseases such as cancer, in which excessive cell multiplication needs to be stopped. The drug has been tested in adult cancer patients and has been well tolerated by subjects in these studies. It is experimental and, therefore, available in clinical trials.
Read the detailed description
Although a Phase II portion of this study was planned, it was never initiated.
- Conditions
- Tumors, Brain Tumors, Rhabdomyosarcoma, Sarcoma, Soft Tissue
- Being tested
- Everolimus
- Keywords
- Brain tumors, Soft Tissue Sarcomas, Refractory solid tumors, Recurrent or Refractory Rhabdomyosarcomatous Soft Tissue and Non-Rhabdomyosarcoma Soft Tissue Sarcomas
- Medical subject headings
- Neoplasms, Brain Neoplasms, Rhabdomyosarcoma, Sarcoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EverolimusDrug
The drug is given orally in 28 day courses.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1 | Experimental | Not described Receives: Drug: Everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
To find the highest dose of RAD001 that can be given to children with refractory or relapsing solid tumors, leukemias, or brain tumors without causing severe side effects
Measured: Within 30 days per subject
Secondary outcomes (1)
To find out if there are changes in the body's blood cells and tumor cells after treatment with RAD001
Measured: Within 30 days per subject
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study startOct 2004
- Primary completionOct 2008
- Study completionOct 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for TumorsStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Sponsor
- Investigators
- Wayne L Furman, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00187174
- Sponsor's protocol ID
- RAD001
- Status last verified
- Dec 2009
- First submitted
- 13 Sep 2005
- Last updated
- 4 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Trial of RAD001C (Everolimus) in Pediatric Patients With Recurrent Refractory Solid Tumors or Brain Tumors
- ClinicalTrials.gov ID
- NCT00187174
- Acronym / sponsor ID
- RAD001
- Conditions
- Tumors, Brain Tumors, Rhabdomyosarcoma, Sarcoma, Soft Tissue
- Keywords
- Brain tumors, Soft Tissue Sarcomas, Refractory solid tumors, Recurrent or Refractory Rhabdomyosarcomatous Soft Tissue and Non-Rhabdomyosarcoma Soft Tissue Sarcomas
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Oct 2004
- Primary completion
- Oct 2008 (actual)
- Study completion
- Oct 2008 (actual)
- First posted
- 16 Sep 2005 (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.
Patients with recurrent or refractory solid tumors or brain tumors that are unresponsive to conventional therapy, or with no known effective therapy, will be treated. Experiments in the laboratory have shown the experimental drug RAD001C (RAD001, Everolimus) can prevent cells from multiplying. RAD001 is now being tested in diseases such as cancer, in which excessive cell multiplication needs to be stopped. The drug has been tested in adult cancer patients and has been well tolerated by subjects in these studies. It is experimental and, therefore, available in clinical trials.
Detailed description
Although a Phase II portion of this study was planned, it was never initiated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Phase 1 | Experimental | Drug: Everolimus |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Adequate performance status
- Adequate bone marrow, kidney, heart, and liver function
Key exclusion criteria (2 listed)
- Must not be receiving concomitant anti-cancer treatment
- Must not be pregnant
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 |
|---|---|---|
| EverolimusThe drug is given orally in 28 day courses. | Drug | Phase 1 |
Primary outcome
To find the highest dose of RAD001 that can be given to children with refractory or relapsing solid tumors, leukemias, or brain tumors without causing severe side effects
Time frame: Within 30 days per subject
1 secondary outcome
To find out if there are changes in the body's blood cells and tumor cells after treatment with RAD001
Time frame: Within 30 days per subject
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 4 Mar 2015.
- St. Jude Children's Research HospitalLink provided by the sponsor.
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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.
Wayne L Furman, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Everolimus in people with Tumors and Brain Tumors, run by St. Jude Children's Research Hospital at 1 site in United States.
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 2 inclusion and 2 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 4 Mar 2015. 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 United States. 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?
To find the highest dose of RAD001 that can be given to children with refractory or relapsing solid tumors, leukemias, or brain tumors without causing severe side effects (time frame: Within 30 days per subject). 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 4 Mar 2015.