Adult Acute Megakaryoblastic Leukemia (M7)NCT01872819

Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay

A study testing antitumor drug screening assay, chemotherapy and biological therapy in people with Adult Acute Megakaryoblastic Leukemia (M7) and Adult Acute Minimally Differentiated Myeloid Leukemia (M0), run by University of Washington at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a)
What is being tested?: antitumor drug screening assay, chemotherapy and biological therapy
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 16 participants
Where: 1 siteUnited States
Study dates: 2 Aug 2013to 17 Nov 2014
Phase: Interventional
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

Diagnosis of acute myeloid leukemia by World Health Organization (WHO) criteria (except acute promyelocytic leukemia), acute leukemias of ambiguous lineage by WHO criteria, or myelodysplastic syndrome refractory anemia with excess blasts (RAEB)-2 by WHO classification or advanced myeloproliferative neoplasm with >= 10% blasts in the bone marrow or peripheral blood, including chronic myelomonocytic leukemia (CMML)-2 by WHO classification who have failed 2 inductions at initial diagnosis or failed >= 2 salvage regimens for relapsed acute myeloid leukemia (AML)
Patients who have had a 1st remission for >= 1 year must have received cytotoxic chemotherapy as a salvage regimen
Eastern Cooperative Oncology Group (ECOG) performance status 0 - 3
Expectation that we can obtain about 100 million blasts from blood and/or marrow (for example, circulating blast count of 5,000 or greater)
Bilirubin =< 1.5 x institutional upper limit of normal (IULN) unless elevation is thought to be due to Gilbert's syndrome, hemolysis, or hepatic infiltration by the hematologic malignancy
Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) and serum pyruvate glutamate transaminase (SPGT) (alanine aminotransferase [ALT]) =< 2.5 x IULN, unless elevation in thought to be due to hepatic infiltration by the hematologic malignancy

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Alkaline phosphatase =< 2.5 X ULN
Serum creatinine =< 2.0 mg/dL
Stable or improving on appropriate antimicrobial therapy for infection, without ongoing fever
Informed consent
Willing to use contraception

Who cannot join

No other concomitant treatment for leukemia
No other active cancer that requires systemic chemotherapy or radiation
Significant organ compromise that will increase risk of toxicity or mortality
Pregnancy or lactation

Trial locations and contacts

1 site in 1 country.

United States1 site

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
Seattle, Washington, 98109

Other trials in United States

About this study

This clinical trial uses a laboratory test called a high throughput sensitivity assay in planning treatment for patients with relapsed or refractory acute myeloid leukemia. The aim is to try to identify drugs that may be effective in killing leukemia cells for those patients who will not be cured with conventional chemotherapy. This assay will test multiple drugs simultaneously against a patient's own donated blood sample. The goal is to use this laboratory assay to best match a drug to a patient's disease.

Read the detailed description

PRIMARY OBJECTIVES:

To obtain results from a high throughput drug sensitivity assay within 10 days, procure drug within 14 days and initiate treatment within 21 days.

SECONDARY OBJECTIVES:

To achieve a response (cytoreduction or at least partial response) greater that than expected for comparable refractory patient populations with other salvage regimens.

OUTLINE:

A patient receives a drug intervention based on the results of a high throughput sensitivity assay. This assay best matches a drug to the patient's disease.

Conditions: Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1)
Being tested: antitumor drug screening assay, chemotherapy, biological therapy
Medical subject headings: Leukemia, Megakaryoblastic, Acute, Leukemia, Monocytic, Acute, Leukemia, Myeloid, Acute, Congenital Abnormalities, Leukemia, Myelomonocytic, Acute, Leukemia, Erythroblastic, Acute, Leukemia, Myelomonocytic, Chronic, Myeloproliferative Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

antitumor drug screening assayOther
Also called antitumor drug screening assays, drug screening assays, antitumor
Undergo high throughput drug sensitivity assay
chemotherapyDrug
Also called chemo
Patients receive 1 of 160 possible interventions
biological therapyBiological
Patients receive 1 of 160 possible interventions

Study groups in NCT01872819
Study group	Role	What they receive
Treatment (chemotherapy, biological therapy)	Experimental	Patients receive 1 of 160 possible interventions based on high throughput drug sensitivity assay. Receives: Other: antitumor drug screening assay; Drug: chemotherapy; Biological: biological therapy

What is being measured

The primary outcome is the main question the study is designed to answer.

Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML
Whether treatment was administered in the time frame based on the high throughput drug screen. Time from sample procurement to assay results.
Measured: Up to 21 days

Secondary outcomes (1)

Rate of Complete Response, Defined by Criteria of Cheson et al.
Number of patients who achieved a Complete Response (CR) with Minimal Residual Disease (MRD), a Complete Response with incomplete hematologic recovery (CRi), or showed reduced blasts in their bone marrow by flow cytometry (Cytoreduction).
Cheson et al. defines a CR as: Bone Marrow blasts <5%, absence of circulating blasts and blasts with Auer rods, absence of extramedullary disease, absolute neutrophil count >1.0 x 10^9/L, and platelet count >100 x 10^9/L. Cheson et al. defines a CRi as: all CR criteria except for residual neutropenia (<1.0 x 10^9/L) or thrombocytopenia (<100 x 10^9/L).
Measured: Baseline 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.

2 Aug 201317 Nov 2014

First posted7 Jun 2013 (estimated)
Study start2 Aug 2013
Primary completionNov 2014
Study completion17 Nov 2014

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 Adult Acute Megakaryoblastic Leukemia (M7)Studies of the same condition, open and under way.
More trials run by University of WashingtonThe 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 N/A
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01872819
Sponsor's protocol ID: 8003
Other IDs: NCI-2013-01070, 8003, P30CA015704
Status last verified: Jun 2018
First submitted: 4 Jun 2013
Last updated: 11 Jul 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Study at a glance

From the registry record, last updated by the sponsor on 11 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay
ClinicalTrials.gov ID: NCT01872819
Acronym / sponsor ID: 8003
Conditions: Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1)
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: University of WashingtonUniversity, hospital or other
Collaborators: National Cancer Institute (NCI)
Study start: 2 Aug 2013 (actual)
Primary completion: Nov 2014 (actual)
Study completion: 17 Nov 2014 (actual)
First posted: 7 Jun 2013 (estimated)
Results: Posted 17 Apr 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

PRIMARY OBJECTIVES:

To obtain results from a high throughput drug sensitivity assay within 10 days, procure drug within 14 days and initiate treatment within 21 days.

SECONDARY OBJECTIVES:

To achieve a response (cytoreduction or at least partial response) greater that than expected for comparable refractory patient populations with other salvage regimens.

OUTLINE:

A patient receives a drug intervention based on the results of a high throughput sensitivity assay. This assay best matches a drug to the patient's disease.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treatment (chemotherapy, biological therapy)	Experimental	Other: antitumor drug screening assay, Drug: chemotherapy, Biological: biological therapyPatients receive 1 of 160 possible interventions based on high throughput drug sensitivity assay.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (11 listed)

Diagnosis of acute myeloid leukemia by World Health Organization (WHO) criteria (except acute promyelocytic leukemia), acute leukemias of ambiguous lineage by WHO criteria, or myelodysplastic syndrome refractory anemia with excess blasts (RAEB)-2 by WHO classification or advanced myeloproliferative neoplasm with >= 10% blasts in the bone marrow or peripheral blood, including chronic myelomonocytic leukemia (CMML)-2 by WHO classification who have failed 2 inductions at initial diagnosis or failed >= 2 salvage regimens for relapsed acute myeloid leukemia (AML)
Patients who have had a 1st remission for >= 1 year must have received cytotoxic chemotherapy as a salvage regimen
Eastern Cooperative Oncology Group (ECOG) performance status 0 - 3
Expectation that we can obtain about 100 million blasts from blood and/or marrow (for example, circulating blast count of 5,000 or greater)
Bilirubin =< 1.5 x institutional upper limit of normal (IULN) unless elevation is thought to be due to Gilbert's syndrome, hemolysis, or hepatic infiltration by the hematologic malignancy

Key exclusion criteria (4 listed)

No other concomitant treatment for leukemia
No other active cancer that requires systemic chemotherapy or radiation
Significant organ compromise that will increase risk of toxicity or mortality
Pregnancy or lactation

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
antitumor drug screening assayAlso: antitumor drug screening assays, drug screening assays, antitumorUndergo high throughput drug sensitivity assay	Other	Treatment (chemotherapy, biological therapy)
chemotherapyAlso: chemoPatients receive 1 of 160 possible interventions	Drug	Treatment (chemotherapy, biological therapy)
biological therapyPatients receive 1 of 160 possible interventions	Biological	Treatment (chemotherapy, biological therapy)

Primary outcome

Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML
Time frame: Up to 21 days
Whether treatment was administered in the time frame based on the high throughput drug screen. Time from sample procurement to assay results.

1 secondary outcome

Rate of Complete Response, Defined by Criteria of Cheson et al.
Time frame: Baseline up to 2 years

Results and safety

Results posted to the registry on 17 Apr 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Treatment (Chemotherapy, Biological Therapy)	14	14

Primary outcome: Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML

Time frame: Up to 21 days. Median (Full Range). Unit: days

Measure	Treated Patients n = 14
Value	5.1 (4 to 8)

Population analysed: 14 patients were treated.

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 2 years. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Treatment (Chemotherapy, Biological Therapy)	11 of 14 (78.6%)	0 of 0	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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.

Pamela Becker principal investigator

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 11 inclusion and 4 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 11 Jul 2018. 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Apr 2017). 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?

Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML (time frame: Up to 21 days). 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 NCT01872819.

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 11 Jul 2018; AzTrials last refreshed its copy on 26 Oct 2026.