LeukemiaPhase 1/2NCT01251965
Phase l/II Study of Ruxolitinib for Acute Leukemia
A Phase 1/2 study testing Ruxolitinib in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with acute leukemia and to learn if the study drug can help control the disease. The safety of the drug will also be studied. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2010to Jul 2012
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Must be >14 years of age
- Must be diagnosed with refractory or relapsed AML or ALL.
- Must have adequate organ function as demonstrated by the following: o Alanine Aminotransferase (ALT) (SGOT) and/or Aspartate Aminotransferase (AST) (SGPT) equal to or less than 1.5x upper limit of normal o Serum creatinine equal to or less than 2.5 mg/dL
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2
- At least 2 weeks from prior leukemia-directed treatment to starting treatment drug (except for hydroxyurea, which is allowed if clinically indicated but should be stopped after 2 weeks of receiving study drug, and glucocorticoids, which are allowed but should be stopped upon starting treatment drug).
- Treatment-related toxicities from prior therapies must have resolved to Grade equal to or less than 1 (except for peripheral neuropathy, which should resolve to grade equal to or less than 2)
- No active malignancies with the exception of basal cell or squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.
- Females of childbearing potential (FCBP)(A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months) must have negative pregnancy test. FCBP and males participating in the study must agree to use a reliable form of contraception or to practice complete abstinence from heterosexual intercourse while participating in the study and for at least 28 days after discontinuation from the study. If pregnancy or a positive pregnancy test does occur in a study subject, treatment with the study drug must be immediately discontinued.
Who cannot join
- Known positive status for HIV, or known active hepatitis A, B, or C infection.
- Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Pregnant or lactating females.
- Acute promyelocytic leukemia
- Concurrent use of strong inducers or strong inhibitors of cytochrome P450 3A4 (CYP3A4). Strong inducers are rifampin and St. John's Worth. Strong inhibitors are HIV-antivirals, clarythromycin, itraconazole, ketoconazole, nefazodone, and telithromycin.
- Participating in any other research trial.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Phase I/II Study to Determine the Safety and Efficacy of Ruxolitinib, a JAK1/JAK2 Inhibitor, in Subjects With Relapsed or Refractory Acute Leukemia
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with acute leukemia and to learn if the study drug can help control the disease. The safety of the drug will also be studied.
Read the detailed description
The Study Drug:
Ruxolitinib is designed to block a gene mutation that may be important in cancer cell growth and survival. By blocking the gene mutation, this may cause the cancer cells to die.
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study. Up to 30 participants will be enrolled in the Phase I portion of the study, and up to 136 participants will be enrolled in Phase II.
If you are enrolled in the Phase I portion, the dose of ruxolitinib you receive will depend on when you joined this study. The first group of participants will receive the lowest dose level of ruxolitinib. Each new group will receive a higher dose of ruxolitinib than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of ruxolitinib is found.
If you are enrolled in the Phase II portion, you will receive ruxolitinib at the highest dose that was tolerated in the Phase I portion or at a lower dose.
Study Drug Administration:
You will take ruxolitinib tablet(s) by mouth 2 times a day on Days 1-28 of each 28-day study cycle.
You will be asked to keep a diary to record the doses taken. You will be asked to bring your diary and any unused drug to your next visit.
Study Visits:
On Days 1, 7, 14, and 21 of Cycle 1:
- You will have a physical exam.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- On Day 14 only, you will have a bone marrow aspiration performed to check the status of the disease.
On Day 1 of Cycle 2:
- You will have a physical exam.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspiration performed to check the status of the disease.
During Cycles 2 and beyond, blood (about 2 teaspoons) will be drawn for routine tests at least every 1-2 weeks. This blood may be drawn at a clinic close to your home.
On Day 1 of Cycles 3, 6, 9, and beyond:
- You will have a physical exam.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspiration performed to check the status of the disease. On Day 1 of Cycle 3, this will only be done if your doctor thinks it is needed.
Length of Study:
You may continue taking the study drug for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse or intolerable side effects occur.
Your participation on the study will be over once you have completed the end-of-study visit and the follow-up call.
End-of-Study Visit:
After your last dose of study drug, you will have an end-of-study visit. At this visit, the following tests and procedures will be performed:
- You will have a physical exam.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspiration performed to check the status of the disease.
Follow-Up:
About one month after your end-of-study visit, the study staff will call and ask about any side effects you may be having. This call should last about 5 minutes.
This is an investigational study. Ruxolitinib is FDA approved and commercially available for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera (post-PV) myelofibrosis and post-essential thrombocythemia (post-ET) myelofibrosis. Its use to treat acute leukemia is investigational.
Up to 166 patients will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Ruxolitinib
- Keywords
- Relapsed Acute Leukemia, Acute myeloid leukemia, AML, Acute lymphocytic leukemia, ALL, Ruxolitinib, Jakafi, INC424, INCB018424
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Also called Jakafi, INCBO18424, INC424
Phase I - Starting dose of 50 mg by mouth twice a day for 28 day cycle.
Phase II - MTD reached in Phase I.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib 50 mg BID | Experimental | Phase I - Starting dose of Ruxolitinib 50 mg by mouth twice a day for 28 day cycle. Receives: Drug: Ruxolitinib |
| Ruxolitinib 100 mg BID | Experimental | Phase I dose of Ruxolitinib 100 mg by mouth twice a day for 28 day cycle. Receives: Drug: Ruxolitinib |
| Ruxolitinib 200 mg BID | Experimental | Phase I dose of Ruxolitinib 200 mg by mouth twice a day for 28 day cycle. Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Dose Limiting Toxicities (DLTs)
MTD defined as highest dose level at which no more than one out of six subject experiences dose limiting toxicity (DLT) during first cycle (28 days) of therapy. A non-hematologic DLT defined as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value (according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria) assessed as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during first 28 days on study. Participants who received at least 80% of the originally assigned doses in the first cycle were evaluable for DLT assessment of each cohort.
Measured: End of first 28 day cycle for toxicity
Maximum Tolerated Dose (MTD) of Ruxolitinib
The MTD is defined as the highest dose level at which no more than one out of six subject experiences DLT during the first cycle (28 days) of therapy.
Measured: End of first 28 day cycle
Secondary outcomes (1)
Participants With a Response
Response is defined as complete remission (CR) + complete remission with incomplete blood count (CRi) + Hematologic improvement (HI). Response was to be assessed for participants who were evaluated for the Phase II portion of this study. CR is absolute neutrophil count (ANC) >/= 1x109/L and platelet count >/= 100x109/L, absence of leukemia blast cells, normal marrow differential, and complete resolution of extramedullary disease. CRi is CR but platelets are < 100x109/L or ANC is <1x109/L. HI is described by the number of individual, positively affected cell lines without the use of growth factors and/or transfusions (lasting at least 4 weeks).
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2010 (estimated)
- Study startDec 2010
- Primary completionJul 2012
- Study completionJul 2012
Why it stopped: Study was stopped by the principal investigator due to nonsatisfactory clinical benefit even in patients treated at the highest dose (200 mg ).
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Phase I is a standard 3+3 design and will be used to determine the dose limiting toxicity (DLT) and maximum tolerated dose (MTD), and no formal evaluation of efficacy (response rate will be implemented. Patients in Phase I part who were treated at the MTD will be included toward patients assessed for response in the phase II stage.
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Incyte Corporation
- Responsible party
- Sponsor
- Investigators
- Srdan Verstovsek, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01251965
- Sponsor's protocol ID
- 2010-0450
- Other IDs
- NCI-2011-02439
- Status last verified
- May 2025
- First submitted
- 30 Nov 2010
- Last updated
- 8 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Pemmaraju N, Kantarjian H, Kadia T, Cortes J, Borthakur G, Newberry K, Garcia-Manero G, Ravandi F, Jabbour E, Dellasala S, Pierce S, Verstovsek S. A phase I/II study of the Janus kinase (JAK)1 and 2 inhibitor ruxolitinib in patients with relapsed or refractory acute myeloid leukemia. Clin Lymphoma Myeloma Leuk. 2015 Mar;15(3):171-6. doi: 10.1016/j.clml.2014.08.003. Epub 2014 Sep 17.
PubMed 25441108
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study to Determine the Safety and Efficacy of Ruxolitinib, a JAK1/JAK2 Inhibitor, in Subjects With Relapsed or Refractory Acute Leukemia
- ClinicalTrials.gov ID
- NCT01251965
- Acronym / sponsor ID
- 2010-0450
- Conditions
- Leukemia
- Keywords
- Relapsed Acute Leukemia, Acute myeloid leukemia, AML, Acute lymphocytic leukemia, ALL, Ruxolitinib, Jakafi, INC424
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was stopped by the principal investigator due to nonsatisfactory clinical benefit even in patients treated at the highest dose (200 mg ).
- Enrolment
- 27 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Incyte Corporation
- Study start
- Dec 2010
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 2 Dec 2010 (estimated)
- Results
- Posted 6 Sep 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with acute leukemia and to learn if the study drug can help control the disease. The safety of the drug will also be studied.
Detailed description
The Study Drug:
Ruxolitinib is designed to block a gene mutation that may be important in cancer cell growth and survival. By blocking the gene mutation, this may cause the cancer cells to die.
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study. Up to 30 participants will be enrolled in the Phase I portion of the study, and up to 136 participants will be enrolled in Phase II.
If you are enrolled in the Phase I portion, the dose of ruxolitinib you receive will depend on when you joined this study. The first group of participants will receive the lowest dose level of ruxolitinib. Each new group will receive a higher dose of ruxolitinib than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of ruxolitinib is found.
If you are enrolled in the Phase II portion, you will receive ruxolitinib at the highest dose that was tolerated in the Phase I portion or at a lower dose.
Study Drug Administration:
You will take ruxolitinib tablet(s) by mouth 2 times a day on Days 1-28 of each 28-day study cycle.
You will be asked to keep a diary to record the doses taken. You will be asked to bring your diary and any unused drug to your next visit.
Study Visits:
On Days 1, 7, 14, and 21 of Cycle 1:
- You will have a physical exam.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- On Day 14 only, you will have a bone marrow aspiration performed to check the status of the disease.
On Day 1 of Cycle 2:
- You will have a physical exam.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspiration performed to check the status of the disease.
During Cycles 2 and beyond, blood (about 2 teaspoons) will be drawn for routine tests at least every 1-2 weeks. This blood may be drawn at a clinic close to your home.
On Day 1 of Cycles 3, 6, 9, and beyond:
- You will have a physical exam.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspiration performed to check the status of the disease. On Day 1 of Cycle 3, this will only be done if your doctor thinks it is needed.
Length of Study:
You may continue taking the study drug for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse or intolerable side effects occur.
Your participation on the study will be over once you have completed the end-of-study visit and the follow-up call.
End-of-Study Visit:
After your last dose of study drug, you will have an end-of-study visit. At this visit, the following tests and procedures will be performed:
- You will have a physical exam.
- You will be asked about any treatments you may have had, any other drugs you may be taking, and any side effects you may be having.
- Blood (about 2 teaspoons) will be drawn for routine tests.
- You will have a bone marrow aspiration performed to check the status of the disease.
Follow-Up:
About one month after your end-of-study visit, the study staff will call and ask about any side effects you may be having. This call should last about 5 minutes.
This is an investigational study. Ruxolitinib is FDA approved and commercially available for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera (post-PV) myelofibrosis and post-essential thrombocythemia (post-ET) myelofibrosis. Its use to treat acute leukemia is investigational.
Up to 166 patients will take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Phase I is a standard 3+3 design and will be used to determine the dose limiting toxicity (DLT) and maximum tolerated dose (MTD), and no formal evaluation of efficacy (response rate will be implemented. Patients in Phase I part who were treated at the MTD will be included toward patients assessed for response in the phase II stage.
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib 50 mg BID | Experimental | Drug: RuxolitinibPhase I - Starting dose of Ruxolitinib 50 mg by mouth twice a day for 28 day cycle. |
| Ruxolitinib 100 mg BID | Experimental | Drug: RuxolitinibPhase I dose of Ruxolitinib 100 mg by mouth twice a day for 28 day cycle. |
| Ruxolitinib 200 mg BID | Experimental | Drug: RuxolitinibPhase I dose of Ruxolitinib 200 mg by mouth twice a day for 28 day cycle. |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Must be >14 years of age
- Must be diagnosed with refractory or relapsed AML or ALL.
- Must have adequate organ function as demonstrated by the following: o Alanine Aminotransferase (ALT) (SGOT) and/or Aspartate Aminotransferase (AST) (SGPT) equal to or less than 1.5x upper limit of normal o Serum creatinine equal to or less than 2.5 mg/dL
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2
- At least 2 weeks from prior leukemia-directed treatment to starting treatment drug (except for hydroxyurea, which is allowed if clinically indicated but should be stopped after 2 weeks of receiving study drug, and glucocorticoids, which are allowed but should be stopped upon starting treatment drug).
Key exclusion criteria (6 listed)
- Known positive status for HIV, or known active hepatitis A, B, or C infection.
- Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Pregnant or lactating females.
- Acute promyelocytic leukemia
- Concurrent use of strong inducers or strong inhibitors of cytochrome P450 3A4 (CYP3A4). Strong inducers are rifampin and St. John's Worth. Strong inhibitors are HIV-antivirals, clarythromycin, itraconazole, ketoconazole, nefazodone, and telithromycin.
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 |
|---|---|---|
| RuxolitinibAlso: Jakafi, INCBO18424, INC424Phase I - Starting dose of 50 mg by mouth twice a day for 28 day cycle. Phase II - MTD reached in Phase I. | Drug | Ruxolitinib 100 mg BID, Ruxolitinib 200 mg BID, Ruxolitinib 50 mg BID |
Primary outcome
Number of Participants With Dose Limiting Toxicities (DLTs)
Time frame: End of first 28 day cycle for toxicity
MTD defined as highest dose level at which no more than one out of six subject experiences dose limiting toxicity (DLT) during first cycle (28 days) of therapy. A non-hematologic DLT defined as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value (according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria) assessed as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during first 28 days on study. Participants who received at least 80% of the originally assigned doses in the first cycle were evaluable for DLT assessment of each cohort.
Maximum Tolerated Dose (MTD) of Ruxolitinib
Time frame: End of first 28 day cycle
The MTD is defined as the highest dose level at which no more than one out of six subject experiences DLT during the first cycle (28 days) of therapy.
1 secondary outcome
Participants With a Response
Time frame: Up to 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25441108Pemmaraju N, Kantarjian H, Kadia T, Cortes J, Borthakur G, Newberry K, Garcia-Manero G, Ravandi F, Jabbour E, Dellasala S, Pierce S, Verstovsek S. A phase I/II study of the Janus kinase (JAK)1 and 2 inhibitor ruxolitinib in patients with relapsed or refractory acute myeloid leukemia. Clin Lymphoma Myeloma Leuk. 2015 Mar;15(3):171-6. doi: 10.1016/j.clml.2014.08.003. Epub 2014 Sep 17.
- University of Texas MD Anderson Cancer Center WebsiteLink 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.
Srdan Verstovsek, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Ruxolitinib in people with Leukemia, run by M.D. Anderson Cancer Center 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 8 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: stopped early, as of the sponsor's last update on 8 Jun 2025. 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 combined 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.
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?
Yes. Results are posted in the registry (first posted 6 Sep 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Dose Limiting Toxicities (DLTs) (time frame: End of first 28 day cycle for toxicity). 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 NCT01251965, 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 2025.