Acute Lymphoblastic LeukemiaPhase 1NCT00874562
Rapamycin in Relapsed Acute Lymphoblastic Leukemia
A Phase 1 study testing Corticosteroid and Rapamycin in people with Acute Lymphoblastic Leukemia, run by Dana-Farber Cancer Institute at 1 site in United States.
Trial summary
This is a research study designed to look at the biological effects of two drugs on leukemia cells. In this study, we are comparing the effects of drugs called corticosteroids when used alone or with another drug called rapamycin. Rapamycin is a drug that prevents the body's immune system from working normally. Read more about this study
- Who is it for?
- Ages 365 days and older
- Conditions the study is for
- Acute Lymphoblastic Leukemia
- What is being tested?
- Corticosteroid and Rapamycin
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2007to Apr 2016
- 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 365 days and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documented acute lymphoblastic leukemia (L1 or L2 subtypes)
- First or subsequent relapse
- 365 days of age or older
- Greater than 7 days from any chemotherapy or immunotherapy, with the exception of intrathecal chemotherapy
- Absolute peripheral leukemia blast count of 1000 cells/ul or greater
- Patient (or parent/guardian if patient is less than 18 years of age) must sign informed consent
Who cannot join
- Burkitts leukemia (acute lymphoblastic leukemia L3 subtype)
- Uncontrolled active infection
- Pregnancy or mothers who are nursing
- Patient currently taking rapamycin
- Patients with significant liver dysfunction as outlined in protocol
- Severe concurrent disease, which in the judgment of the investigator, would make the patient inappropriate for entry into the study
- Active psychiatric disease, substance abuse, or mental illness that would interfere with cooperation with the requirements of the trial
Trial locations and contacts
1 site in 1 country.
United States1 site
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
About this study
This is a research study designed to look at the biological effects of two drugs on leukemia cells. In this study, we are comparing the effects of drugs called corticosteroids when used alone or with another drug called rapamycin. Rapamycin is a drug that prevents the body's immune system from working normally. It has been used for many years after kidney transplants to prevent rejection of the organ. Recent work suggests that rapamycin may also help treat leukemia and other cancers.
Read the detailed description
- Participants will be randomized into two groups; one group will receive corticosteroid alone, and the other group will receive rapamycin and corticosteroid.
- The length of treatment will be 5 days, during which time we will collect blood samples to measure the biologic effects of these drugs. Because these drugs will be given for a short period of time only, this study is not designed to treat or cure the participants leukemia. After the 5-day period, participants may resume other cancer-directed therapies.
- Conditions
- Acute Lymphoblastic Leukemia
- Being tested
- Corticosteroid, Rapamycin
- Keywords
- rapamycin, corticosteroid, ALL
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CorticosteroidDrug
Initial dose of corticosteroid given intravenously. After the first dose, corticosteroid will be taken orally every 8 hours for the duration of the five-day period
RapamycinDrug
Also called sirolimus
Taken orally mixed with water or orange juice
| Study group | Role | What they receive |
|---|---|---|
| Steroid Only | Active comparator | Corticosteroid Alone Receives: Drug: Corticosteroid |
| Steroid plus Rapamycin | Active comparator | Corticosteroid plus Rapamycin Receives: Drug: Corticosteroid; Drug: Rapamycin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare changes in gene expression signatures in steroid alone versus steroid plus rapamycin treated patients.
Measured: 3 years
Compare biologic measures of "pro-apoptotic state" in steroid alone versus steroid plus rapamycin treated patients
Measured: 3 years
Secondary outcomes (1)
Assess clinical response by percent reduction of peripheral leukemia blasts at day 2 of therapy and at completion of the 5-day investigational window
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Apr 2009 (estimated)
- Study startJul 2007
- Primary completionNov 2009
- Study completionApr 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Brigham and Women's Hospital, Boston Children's Hospital, National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Lewis B. Silverman, M.D., Dana-Farber Cancer Institute
- Investigators
- Lewis Silverman, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00874562
- Sponsor's protocol ID
- 06-429
- Status last verified
- Nov 2016
- First submitted
- 1 Apr 2009
- Last updated
- 23 Nov 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rapamycin in Relapsed Acute Lymphoblastic Leukemia
- ClinicalTrials.gov ID
- NCT00874562
- Acronym / sponsor ID
- 06-429
- Conditions
- Acute Lymphoblastic Leukemia
- Keywords
- rapamycin, corticosteroid, ALL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Brigham and Women's Hospital, Boston Children's Hospital, National Cancer Institute (NCI)
- Study start
- Jul 2007
- Primary completion
- Nov 2009 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 2 Apr 2009 (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.
This is a research study designed to look at the biological effects of two drugs on leukemia cells. In this study, we are comparing the effects of drugs called corticosteroids when used alone or with another drug called rapamycin. Rapamycin is a drug that prevents the body's immune system from working normally. It has been used for many years after kidney transplants to prevent rejection of the organ. Recent work suggests that rapamycin may also help treat leukemia and other cancers.
Detailed description
- Participants will be randomized into two groups; one group will receive corticosteroid alone, and the other group will receive rapamycin and corticosteroid.
- The length of treatment will be 5 days, during which time we will collect blood samples to measure the biologic effects of these drugs. Because these drugs will be given for a short period of time only, this study is not designed to treat or cure the participants leukemia. After the 5-day period, participants may resume other cancer-directed therapies.
Study design
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Steroid Only | Active comparator | Drug: CorticosteroidCorticosteroid Alone |
| Steroid plus Rapamycin | Active comparator | Drug: Corticosteroid, Drug: RapamycinCorticosteroid plus Rapamycin |
Who may qualify
- Ages
- 365 days and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Documented acute lymphoblastic leukemia (L1 or L2 subtypes)
- First or subsequent relapse
- 365 days of age or older
- Greater than 7 days from any chemotherapy or immunotherapy, with the exception of intrathecal chemotherapy
- Absolute peripheral leukemia blast count of 1000 cells/ul or greater
Key exclusion criteria (7 listed)
- Burkitts leukemia (acute lymphoblastic leukemia L3 subtype)
- Uncontrolled active infection
- Pregnancy or mothers who are nursing
- Patient currently taking rapamycin
- Patients with significant liver dysfunction as outlined in protocol
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 |
|---|---|---|
| CorticosteroidInitial dose of corticosteroid given intravenously. After the first dose, corticosteroid will be taken orally every 8 hours for the duration of the five-day period | Drug | Steroid Only, Steroid plus Rapamycin |
| RapamycinAlso: sirolimusTaken orally mixed with water or orange juice | Drug | Steroid plus Rapamycin |
Primary outcome
Compare changes in gene expression signatures in steroid alone versus steroid plus rapamycin treated patients.
Time frame: 3 years
Compare biologic measures of "pro-apoptotic state" in steroid alone versus steroid plus rapamycin treated patients
Time frame: 3 years
1 secondary outcome
Assess clinical response by percent reduction of peripheral leukemia blasts at day 2 of therapy and at completion of the 5-day investigational window
Time frame: 3 years
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 23 Nov 2016.
- 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. This study is not recruiting at the moment.
Lewis Silverman, MD principal investigator
Dana-Farber Cancer Institute
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 Corticosteroid and Rapamycin in people with Acute Lymphoblastic Leukemia, run by Dana-Farber Cancer Institute 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 6 inclusion and 7 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 23 Nov 2016. 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?
Compare changes in gene expression signatures in steroid alone versus steroid plus rapamycin treated patients. (time frame: 3 years). 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?
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 23 Nov 2016.