Simplified Patient Care Strategy in Decreasing Early Death in Patients With Acute Promyelocytic Leukemia

• ClinicalTrials.gov Identifier: NCT03253848
• Recruitment Status: Recruiting
• First Posted: August 18, 2017
• Last Update Posted: May 21, 2020
• Sponsor: ECOG-ACRIN Cancer Research Group
• Collaborator: National Cancer Institute (NCI)
• Information provided by (Responsible Party): Eastern Cooperative Oncology Group ( ECOG-ACRIN Cancer Research Group )

Tracking Information

• First Submitted Date: August 16, 2017
• First Posted Date: August 18, 2017
• Last Update Posted Date: May 21, 2020
• Actual Study Start Date: August 16, 2017
• Estimated Primary Completion Date: August 18, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 16, 2017)

Mortality rate [ Time Frame: At 1 month ]

Will be estimated by dividing total number of deaths within one month of study entry by total number of cases with at least one-month follow up data. 95% repeated confidence interval will be provided.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: August 16, 2017)

• Compliance with treatment guidelines [ Time Frame: Up to 1 year ]
  Will be assessed as binary outcome and the successful compliance rate will be described with 95% confidence interval. A regression model will be developed to evaluate the factors associated with outcome. Data items such as age, risk status, comorbidity will be collected and evaluated.
• Length of hospital stay [ Time Frame: Up to 1 year ]
  Will be associated with toxicity measures. Will be dichotomized as ?mild? versus (vs.) ?severe? and the length of hospital stay will be compared using the two-sample t-test.
• Mortality rate [ Time Frame: At 1 month ]
  Will be compared between patients with long time or short time from diagnosis to treatment using the Fisher?s exact test. Will also be compared between lead institutions and outlying centers.
• Overall survival [ Time Frame: At 1 month ]
  Will be compared between the lead institutions and outlying centers and two-sided p-values will be reported.
• Overall survival (OS) [ Time Frame: At 1 year ]
  Will be described using the method of Kaplan-Meier. The median OS and one-year OS rate will be estimated and 95% confidence interval (CI) will be provided.
• Severity and duration of coagulopathy, bleeding, infections, and differentiation syndrome [ Time Frame: Up to 1 year ]
  Will be measured based on the Common Terminology Criteria for Adverse Events version 4.0. Analysis of the safety data will be mostly descriptive. The proportion of patients with the worst degree toxicity of grade 3 or higher will be assessed and presented with 95% CI.
• Time from diagnosis and initiation of treatment [ Time Frame: Up to 1 year ]
  Will be assessed and dichotomized as ?short? vs. ?long? using the median value.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Simplified Patient Care Strategy in Decreasing Early Death in Patients With Acute Promyelocytic Leukemia
• Official Title: A Simplified Patient Care Strategy to Decrease Early Deaths in Acute Promyelocytic Leukemia (APL)
• Brief Summary: This clinical trial studies how well simplified patient care strategy works in decreasing early death in patients with acute promyelocytic leukemia. Implementing simplified acute promyelocytic leukemia guidelines along with support from acute promyelocytic leukemia experts may decrease deaths and improve survival.

Detailed Description

PRIMARY OBJECTIVES:

I. To evaluate if the proposed patient care strategy, that includes use of simplified guidelines along with acute promyelocytic leukemia (APL) expert support, decreases the one-month induction mortality rate from 30% to under 15%.

SECONDARY OBJECTIVES:

I. To assess the overall survival 1 year after accrual is completed. II. To assess incidence and severity of differentiation syndrome. III. To correlate outcomes with time to initiation of all-trans retinoic acid (ATRA) from diagnosis or suspicion of diagnosis.

IV. To compare outcomes between academic and community centers separately. V. To evaluate factors associated with outcome.

OUTLINE:

Patients receive standard of care treatment for APL. Patients? doctors regularly discuss with an APL expert to identify and mange treatment.

After diagnosis, patients are followed up for 1 year.

• Study Type: Observational
• Study Design: Observational Model: Case-Only; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Patients with confirmed APL 18 years and over.

Condition

• Acute Promyelocytic Leukemia With PML-RARA
• t(15;17)

Intervention

• Other: Best Practice
  Discussion between doctors and APL expert
  Other Names:

  • standard of care
  • standard therapy
• Other: Laboratory Biomarker Analysis
  Correlative studies
• Procedure: Supportive Care
  Receive simplified guidelines and support
  Other Names:

  • Supportive Therapy
  • Symptom Management
  • Therapy, Supportive

Study Groups/Cohorts

Observational (simplified guidelines and support)

Patients receive standard of care treatment for APL. Patients? doctors regularly discuss with an APL expert to identify and mange treatment.

Interventions:

• Other: Best Practice
• Other: Laboratory Biomarker Analysis
• Procedure: Supportive Care

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: August 16, 2017): 200
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: August 18, 2025
• Estimated Primary Completion Date: August 18, 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Confirmed to have a diagnosis of APL, which is defined as:

  • Positive t(15:17) by fluorescence in situ hybridization (FISH) or conventional karyotype
  • Positive promyelocytic leukemia/retinoic acid receptor PML/RAR alpha by polymerase chain reaction (PCR)
• Patients must accept treatment and supportive care guidelines
• Referrals must be made as early as possible but no later than 5 calendar days after ATRA therapy is initiated; consent can be obtained up till day 7 or earlier
• Co-management can be started as soon as referral is made including weekends; the physician at the outlying facility should make every effort to call the APL expert at the first suspicion of APL

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Listed Location Countries: Puerto Rico, United States

Administrative Information

• NCT Number: NCT03253848
• Other Study ID Numbers: EA9131; NCI-2016-01123 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) ); EA9131 ( Other Identifier: ECOG-ACRIN Cancer Research Group ); ECOG-ACRIN-EA9131 ( Other Identifier: DCP ); EA9131 ( Other Identifier: CTEP ); UG1CA189828 ( U.S. NIH Grant/Contract ); U10CA037403 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Eastern Cooperative Oncology Group ( ECOG-ACRIN Cancer Research Group )
• Study Sponsor: ECOG-ACRIN Cancer Research Group
• Collaborators: National Cancer Institute (NCI)

Investigators

• Principal Investigator: Anand Jillella; ECOG-ACRIN Cancer Research Group

• PRS Account: Eastern Cooperative Oncology Group
• Verification Date: May 2020