Acute Myeloid LeukemiaPhase 2UPCC04407NCT00615784
Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia
A Phase 2 study testing Bexarotene in people with Acute Myeloid Leukemia, run by Abramson Cancer Center at Penn Medicine at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the activity of bexarotene, a retinoic acid class drug, in patients with Acute Myeloid Leukemia (AML) that has returned after or is resistant to standard chemotherapy or are otherwise not eligible for conventional chemotherapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Bexarotene
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 25 May 2010to 8 Nov 2013
- Phase
- Phase 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age >= 18 years
- Confirmed diagnosis of AML as proven by bone marrow biopsy
- Must have received prior induction therapy with conventional chemotherapy and/or Mylotarg or otherwise not eligible for conventional chemotherapy
- ECOG performance status of 0-2
- Recovered from toxicities of prior chemotherapy
Who cannot join
- History of pancreatitis
- Active alcohol abuse
- Taken bexarotene in the past
- WBC > 10,000/uL at time of enrollment
- Cytotoxic therapy within the past 14 days other than hydrea, low dose cytarabine or low dose Mylotarg
- Significant organ disfunction: total bilirubin > 3x ULN, AST or ALT >3 x ULN, creatinine > 3 mg/dL, on blood pressure supporting medications or mechanical ventilation
Show all 13Show fewer
- Active participant in any other investigational treatment study for AML
- Life expectancy of less than 1 month
- Use of blood growth factors (G-CSF, GM-CSF, Aranesp, erythropoietin, or Neumega) within 1 week prior to treatment initiation
- Uncontrolled hyperlipidemia
- Known history of HIV
- Known active CNS involvement with AML
- Women of childbearing potential or active breast feeding
Trial locations and contacts
1 site in 1 country.
United States1 site
Abramson Cancer Center of University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia
The purpose of this study is to evaluate the activity of bexarotene, a retinoic acid class drug, in patients with Acute Myeloid Leukemia (AML) that has returned after or is resistant to standard chemotherapy or are otherwise not eligible for conventional chemotherapy. Retinoic acids are a class of drugs related to Vitamin A, and have a wide range of effects within normal and malignant cells that affect cell growth and cell death.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Bexarotene
- Keywords
- Acute Myeloid Leukemia, AML, Bexarotene
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BexaroteneDrug
Bexarotene given orally at a dose of 300mg/m2 until disease progression or unacceptable toxicities experienced by patient
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: Bexarotene |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematologic Response Rate of Bexarotene Monotherapy in Subjects With Relapsed/Refractory AML or Newly Diagnosed AML Who Are Unable to Receive Systemic Chemotherapy.
Hematologic response will be assessed according to modified criteria of an international working group defined by Cheson et al, Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood, 1 December 2000, Vol. 96, No. 12, pp. 3671-3674
Measured: Two months after 17th patient has started treatment with Bexarotene, for up to 1 year
Secondary outcomes (1)
Bone Marrow Response Rate of Bexarotene in Subjects With AML Unable/Unwilling to Receive Systemic Chemotherapy
A clinically significant result will be recorded if the patient's bone marrow blasts percentage decreased by 50% or more over pretreatment blast percentage.
Measured: Two months after 17th patient has started treatment with Bexarotene, 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 posted14 Feb 2008 (estimated)
- Study start25 May 2010
- Primary completion1 Oct 2013
- Study completion8 Nov 2013
Why it stopped: Study terminated. Eisai's Targretin acquired by another pharmaceutical company.
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.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Abramson Cancer Center at Penn MedicineThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abramson Cancer Center at Penn Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Donald E. Tsai, MD, PhD, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00615784
- Sponsor's protocol ID
- UPCC 04407
- Other IDs
- UPCC 04407
- Acronym
- UPCC04407
- Status last verified
- Nov 2020
- First submitted
- 1 Feb 2008
- Last updated
- 17 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Bexarotene in Patients With Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT00615784
- Acronym / sponsor ID
- UPCC04407 / UPCC 04407
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Acute Myeloid Leukemia, AML, Bexarotene
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated. Eisai's Targretin acquired by another pharmaceutical company.
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Abramson Cancer Center at Penn MedicineUniversity, hospital or other
- Study start
- 25 May 2010 (actual)
- Primary completion
- 1 Oct 2013 (actual)
- Study completion
- 8 Nov 2013 (actual)
- First posted
- 14 Feb 2008 (estimated)
- Results
- Posted 17 Nov 2020
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 evaluate the activity of bexarotene, a retinoic acid class drug, in patients with Acute Myeloid Leukemia (AML) that has returned after or is resistant to standard chemotherapy or are otherwise not eligible for conventional chemotherapy. Retinoic acids are a class of drugs related to Vitamin A, and have a wide range of effects within normal and malignant cells that affect cell growth and cell death.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: Bexarotene |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age >= 18 years
- Confirmed diagnosis of AML as proven by bone marrow biopsy
- Must have received prior induction therapy with conventional chemotherapy and/or Mylotarg or otherwise not eligible for conventional chemotherapy
- ECOG performance status of 0-2
- Recovered from toxicities of prior chemotherapy
Key exclusion criteria (13 listed)
- History of pancreatitis
- Active alcohol abuse
- Taken bexarotene in the past
- WBC > 10,000/uL at time of enrollment
- Cytotoxic therapy within the past 14 days other than hydrea, low dose cytarabine or low dose Mylotarg
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 |
|---|---|---|
| BexaroteneBexarotene given orally at a dose of 300mg/m2 until disease progression or unacceptable toxicities experienced by patient | Drug | A |
Primary outcome
Hematologic Response Rate of Bexarotene Monotherapy in Subjects With Relapsed/Refractory AML or Newly Diagnosed AML Who Are Unable to Receive Systemic Chemotherapy.
Time frame: Two months after 17th patient has started treatment with Bexarotene, for up to 1 year
Hematologic response will be assessed according to modified criteria of an international working group defined by Cheson et al, Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood, 1 December 2000, Vol. 96, No. 12, pp. 3671-3674
1 secondary outcome
Bone Marrow Response Rate of Bexarotene in Subjects With AML Unable/Unwilling to Receive Systemic Chemotherapy
Time frame: Two months after 17th patient has started treatment with Bexarotene, 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 17 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Donald E. Tsai, MD, PhD principal investigator
University of Pennsylvania
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 Bexarotene in people with Acute Myeloid Leukemia, run by Abramson Cancer Center at Penn Medicine 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 5 inclusion and 13 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 17 Dec 2020. 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 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 17 Nov 2020). 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?
Hematologic Response Rate of Bexarotene Monotherapy in Subjects With Relapsed/Refractory AML or Newly Diagnosed AML Who Are Unable to Receive Systemic Chemotherapy. (time frame: Two months after 17th patient has started treatment with Bexarotene, for up to 1 year). 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 17 Dec 2020.