Refractory or Relapsed RMS and Soft Tissue SarcomasPhase 2NCT01216839
Phase II Study of Everolimus in Children and Adolescents With Refractory or Relapsed Rhabdomyosarcoma and Other Soft Tissue Sarcomas
A Phase 2 study testing Everolimus in people with Refractory or Relapsed RMS and Soft Tissue Sarcomas, run by Sidnei Epelman at 1 site in Brazil.
Trial summary
The purpose of this study is to determine the Everolimus aim response in children and adolescents with refractory or relapsed Rhabdomyosarcoma and other soft tissue sarcomas Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Refractory or Relapsed RMS and Soft Tissue Sarcomas
- What is being tested?
- Everolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteBrazil
- Study dates
- Mar 2011to Dec 2013
- Phase
- Phase 2
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Rhabdomyosarcoma and other soft tissue sarcoma histological confirmation.
- No option of known curative treatment, neither approved treatment that increases survival with adequate quality of life.
- Karnofsky scale ≥ 50 for patients over 16 years and Lansky scale ≥ 50 for patients under 16 years old.
- Subjects shouldn't have received antineoplasic therapy < 4 weeks before study treatment initiation.
- Adequate hematological function: neutrophil count > 1.500/mm³ and hemoglobin > 8.0 mg/dL.
- Adequate renal function, as defined below:
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- Age Maximum serum creatinine (mg/dL) 0 - 29 days 0,4 - 0,7 1 month - 3 years 0,7 4 - 7 years 0,8 8 - 10 years 0,9 11 - 12 years 1,0 13 - 17 years 1,2
- ≥ 18 years 1,3
- Adequate hepatic function: total bilirubin ≤ 1.5 x ULN.
- Patient and/or legal responsible must sign ICF.
- Life expectation > 8 weeks.
- Measurable disease, according to RECIST criteria.
- For female patients of childbearing age: presence of a negative pregnancy test within 7 days prior to day 0.
- The patient agrees to use effective contraception if procreative potential exists. Use of reliable means of contraception (e.g. hormonal contraceptive, patch, vaginal ring, intrauterine device, physical barrier, abstinence)for subjects of reproductive potential (male and female) is required during study treatment and for 3 months following last dose of study drug.
Who cannot join
- History of myocardial infarction, angina and cerebrovascular accident related to atherosclerosis.
- Pulmonary disorder(e.g. FEV1 ou DLCO ≤ 70% from expected).
- Significant hematologic or hepatic abnormality (transaminases levels > 2.5 x ULN or serum bilirubin > 1.5 x ULN, hemoglobin < 8 g/dL, platelets < 100.000/mm³, ANC < 1.500/mm³.
- Has other existing serious medical conditions that could adversely affect the ability of the patient to be treated in accordance with the protocol.
- Any condition, therapy, or medical condition, which, in the opinion of the attending physician could represent a risk for the patient or adversely affect the study objectives.
- If female, is pregnant or lactating.
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- Active infection at the moment of recruitment.
- Previous history of organ transplantation.
- Recent surgery < 2 months before entering study.
- Concomitant antineoplasic therapy.
- Patient received more than one rescue treatment, previously.
- Previous treatment with mTor inhibitors (ex: sirolimus, temsirolimus, everolimus).
- Use of investigational drug < 30 days before entering study.
- Non-controlled hyperlipidaemia: serum cholesterol (fasting) > 300 mg/dL or 7,75 mmol/L and triglycerides (fasting) > 2,5 x ULN.
- Non-controlled Diabetes Mellitus defined as: glycemia (fasting) > 1,5 x ULN.
- Patient with hemorrhagic disorder or using oral anti-vitamin K (except Warfarin in low doses).
- Patient with HIV infection.
- Incapable to perform protocol visits.
- Another neoplasia for the last 2 years (except squamous or basocellular skin cancer).
- Hypersensitivity history to rapamycin analogs.
- Chronic treatment with corticoids (except per oral, topical or local treatment) or another immunosuppressor agent.
Trial locations and contacts
1 site in 1 country.
Brazil1 site, 1 recruiting
- Recruiting
Casa de Saúde Santa Marcelina
São Paulo, São Paulo, 08270070
Sidnei Epelman, MD, +55 11 2522-2472, pesquisaoncoped@santamarcelina.org
Sidnei Epelman, MD
About this study
The purpose of this study is to determine the Everolimus aim response in children and adolescents with refractory or relapsed Rhabdomyosarcoma and other soft tissue sarcomas
- Being tested
- Everolimus
- Medical subject headings
- Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EverolimusDrug
Also called Afinitor, RAD
Everolimus will be administered every day, initial dose 5 mg/m²/day, in 28 days cycle. Maximum dose: 10 mg/day. The cycles will be repeated till progression disease or untolerable toxicity.
| Study group | Role | What they receive |
|---|---|---|
| Everolimus | Experimental | Not described Receives: Drug: Everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the Everolimus aim response in children and adolescents with refractory and relapsed Rhabdomyosarcoma and other soft tissue sarcomas
Measured: Up to 2 years
Secondary outcomes (1)
Define Everolimus toxicity in this population
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2010 (estimated)
- Study startMar 2011
- Primary completionOct 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory or Relapsed RMS and Soft Tissue SarcomasStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sidnei Epelman
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Sidnei Epelman, Hospital Santa Marcelina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01216839
- Sponsor's protocol ID
- CRAD001CBR08T
- Status last verified
- Mar 2013
- First submitted
- 4 Oct 2010
- Last updated
- 26 Mar 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Everolimus in Children and Adolescents With Refractory or Relapsed Rhabdomyosarcoma and Other Soft Tissue Sarcomas
- ClinicalTrials.gov ID
- NCT01216839
- Acronym / sponsor ID
- CRAD001CBR08T
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Sidnei EpelmanUniversity, hospital or other
- Study start
- Mar 2011
- Primary completion
- Oct 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 7 Oct 2010 (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.
The purpose of this study is to determine the Everolimus aim response in children and adolescents with refractory or relapsed Rhabdomyosarcoma and other soft tissue sarcomas
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Everolimus | Experimental | Drug: Everolimus |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- Rhabdomyosarcoma and other soft tissue sarcoma histological confirmation.
- No option of known curative treatment, neither approved treatment that increases survival with adequate quality of life.
- Karnofsky scale ≥ 50 for patients over 16 years and Lansky scale ≥ 50 for patients under 16 years old.
- Subjects shouldn't have received antineoplasic therapy < 4 weeks before study treatment initiation.
- Adequate hematological function: neutrophil count > 1.500/mm³ and hemoglobin > 8.0 mg/dL.
Key exclusion criteria (21 listed)
- History of myocardial infarction, angina and cerebrovascular accident related to atherosclerosis.
- Pulmonary disorder(e.g. FEV1 ou DLCO ≤ 70% from expected).
- Significant hematologic or hepatic abnormality (transaminases levels > 2.5 x ULN or serum bilirubin > 1.5 x ULN, hemoglobin < 8 g/dL, platelets < 100.000/mm³, ANC < 1.500/mm³.
- Has other existing serious medical conditions that could adversely affect the ability of the patient to be treated in accordance with the protocol.
- Any condition, therapy, or medical condition, which, in the opinion of the attending physician could represent a risk for the patient or adversely affect the study objectives.
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 |
|---|---|---|
| EverolimusAlso: Afinitor, RADEverolimus will be administered every day, initial dose 5 mg/m²/day, in 28 days cycle. Maximum dose: 10 mg/day. The cycles will be repeated till progression disease or untolerable toxicity. | Drug | Everolimus |
Primary outcome
Determine the Everolimus aim response in children and adolescents with refractory and relapsed Rhabdomyosarcoma and other soft tissue sarcomas
Time frame: Up to 2 years
1 secondary outcome
Define Everolimus toxicity in this population
Time frame: Up to 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2013.
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Everolimus in people with Refractory or Relapsed RMS and Soft Tissue Sarcomas, run by Sidnei Epelman at 1 site in Brazil.
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 14 inclusion and 21 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: status not confirmed, as of the sponsor's last update on 26 Mar 2013. 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 Brazil. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sidnei Epelman, MD (+55 11 2522-2472, pesquisaoncoped@santamarcelina.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the Everolimus aim response in children and adolescents with refractory and relapsed Rhabdomyosarcoma and other soft tissue sarcomas (time frame: Up to 2 years). 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 26 Mar 2013.