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Identifying Best Approach in Improving Quality of Life and Survival After a Donor Stem Cell Transplant in Older, Medically Infirm, or Frail Patients With Blood Diseases

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ClinicalTrials.gov Identifier: NCT03870750
Recruitment Status : Not yet recruiting
First Posted : March 12, 2019
Last Update Posted : July 17, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Fred Hutchinson Cancer Research Center

Brief Summary:
This phase II/III trial studies the best approach in improving quality of life and survival after a donor stem cell transplant in older, weak, or frail patients with blood diseases. Patients who have undergone a transplant often experience increases in disease and death. One approach, supportive and palliative care (SPC), focuses on relieving symptoms of stress from serious illness and care through physical, cultural, psychological, social, spiritual, and ethical aspects. While a second approach, clinical management of comorbidities (CMC) focuses on managing multiple diseases, other than cancer, such as heart or lung diseases through physical exercise, strength training, stress reduction, medication management, dietary recommendations, and education. Giving SPC, CMC, or a combination of both may work better in improving quality of life and survival after a donor stem cell transplant compared to standard of care in patients with blood diseases.

Condition or disease Intervention/treatment Phase
Allogeneic Hematopoietic Stem Cell Transplantation Recipient Hematopoietic and Lymphoid Cell Neoplasm Non-Neoplastic Hematologic and Lymphocytic Disorder Other: Supportive Palliative Care Other: Clinical Management Other: Best Practice Procedure: Allogeneic Hematopoietic Stem Cell Transplantation Other: Questionnaire Administration Other: Quality-of-Life Assessment Other: Survey Administration Phase 2 Phase 3

Detailed Description:

PRIMARY OBJECTIVES:

I. Compare in a randomized phase II study the effectiveness of supportive and palliative care, a clinical multi-modal program, or a combined approach versus usual care only (UCO) to determine the winning arm in improving health-related quality of life (HRQOL) (Day-90 Functional Assessment of Cancer Therapy-Bone Marrow Transplant [FACT-BMT] scores) for vulnerable recipients of allogeneic hematopoietic cell transplantation (HCT). (Phase II) II. Compare in a randomized phase III study the effectiveness of the winning arm from the preceding phase II study versus UCO in improving HRQOL (Day-90 FACT-BMT scores) and/or overall survival at 1-year for vulnerable recipients of allogeneic HCT. (Phase III)

SECONDARY OBJECTIVES:

I. Rates of overall survival. (Phase II and III) II. Cumulative incidences of non-relapse mortality (NRM). (Phase II and III) III. Cumulative incidences of relapse. (Phase II and III) IV. Rates of relapse-free survival (RFS). (Phase II and III) V. Cumulative incidence of frailty. (Phase II and III) VI. Cumulative incidence of disability. (Phase II and III) VII. Cumulative incidence of grades III-IV cardiac, hepatic, pulmonary and renal toxicities according to the Common Toxicity Criteria (CTC) version 4. (Phase II) VIII. Use of resources within first 90 days after HCT: Frequency of hospitalization. (Phase II and III) IX. Use of resources within first 90 days after HCT: Duration of each hospitalization. (Phase II and III) X. Use of resources within first 90 days after HCT: Number and duration of admissions to intensive care unit. (Phase II and III) XI. Use of resources within first 90 days after HCT: Days out of hospital alive (DOHA). (Phase II and III)

OUTLINE: Patients are randomized to 1 of 4 arms.

ARM I: Patients undergo SPC on days -15 before to +56 after transplant.

ARM II: Patients undergo a CMC program on days -15 before to +56 after transplant.

ARM III: Patients undergo interventions as outlined in Arm I and Arm II.

ARM IV: Patients receive standard of care.

In all arms, patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment and 30, 90, 180, and 365 days post HCT. In all arms patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180 and 365 days post HCT.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 600 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Investigator)
Primary Purpose: Supportive Care
Official Title: Seamless Phase II-Phase III Randomized Clinical Trial to Identify and Confirm the Most Promising Novel Intervention to Alleviate Morbidity and Mortality After Allogeneic Hematopoietic Cell Transplantation Among Older, Medically Infirm, or Frail Patients With Hematological Diseases
Estimated Study Start Date : August 15, 2019
Estimated Primary Completion Date : June 30, 2024
Estimated Study Completion Date : June 30, 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Blood Disorders

Arm Intervention/treatment
Experimental: Arm I (SPC)
Patients undergo SPC on days -15 before to +56 after transplant. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment, 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180 and 365 days post HCT.
Other: Supportive Palliative Care
focuses on relieving symptoms of stress from serious illness and care through physical, cultural, psychological, social, spiritual, and ethical aspects
Other Names:
  • Comfort Care
  • palliation
  • palliative care
  • palliative therapy
  • Palliative Treatment
  • Symptom Management
  • Symptoms Management

Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo HCT
Other Names:
  • Allogeneic Hematopoietic Cell Transplantation
  • Allogeneic Stem Cell Transplantation
  • HSC
  • HSCT

Other: Questionnaire Administration
Ancillary studies

Other: Quality-of-Life Assessment
Ancillary studies
Other Name: Quality of Life Assessment

Other: Survey Administration
Ancillary studies

Experimental: Arm II (CMC)
Patients undergo a CMC program on days -15 to 56. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment, 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180 and 365 days post HCT..
Other: Clinical Management
physical exercise, strength training, stress reduction, medication management, dietary recommendations, and education

Other: Best Practice
Given standard of care
Other Names:
  • standard of care
  • standard therapy

Other: Questionnaire Administration
Ancillary studies

Other: Quality-of-Life Assessment
Ancillary studies
Other Name: Quality of Life Assessment

Other: Survey Administration
Ancillary studies

Experimental: Arm III (SPC and CMC)
Patients undergo interventions as outlined in Arm I and Arm II. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180 and 365 days post HCT..
Other: Supportive Palliative Care
focuses on relieving symptoms of stress from serious illness and care through physical, cultural, psychological, social, spiritual, and ethical aspects
Other Names:
  • Comfort Care
  • palliation
  • palliative care
  • palliative therapy
  • Palliative Treatment
  • Symptom Management
  • Symptoms Management

Other: Clinical Management
physical exercise, strength training, stress reduction, medication management, dietary recommendations, and education

Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo HCT
Other Names:
  • Allogeneic Hematopoietic Cell Transplantation
  • Allogeneic Stem Cell Transplantation
  • HSC
  • HSCT

Other: Questionnaire Administration
Ancillary studies

Other: Quality-of-Life Assessment
Ancillary studies
Other Name: Quality of Life Assessment

Other: Survey Administration
Ancillary studies

Active Comparator: Arm IV (standard of care)
Patients receive standard of care. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment, 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180 and 365 days post HCT.
Other: Best Practice
Given standard of care
Other Names:
  • standard of care
  • standard therapy

Other: Questionnaire Administration
Ancillary studies

Other: Quality-of-Life Assessment
Ancillary studies
Other Name: Quality of Life Assessment

Other: Survey Administration
Ancillary studies




Primary Outcome Measures :
  1. Improvement in HRQOL (Phase II) [ Time Frame: First 90 days after HCT ]
    The arm with the largest mean change in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) from baseline to day 90. The Wilcoxon rank-sum test will be used to compare change in FACT-BMT between arms, and this will also be the test to be used in computation of the conditional power at the end of phase II.

  2. Survival after hematopoietic cell transplantation (HCT) (Phase III) [ Time Frame: At 1 year after HCT ]
  3. Change in health-related quality of life (HRQOL) (Phase III) [ Time Frame: Baseline to 90 days post-HCT ]
    Will be measured by the FACT-BMT.


Secondary Outcome Measures :
  1. Rate of overall survival [ Time Frame: Up to 1 year ]
    Overall survival will be compared between each of the experimental arms and the usual care only (UCO) arm using the log-rank test. Arms that do not survive the screening phase will also be included for comparison.

  2. Non-relapse mortality [ Time Frame: At 90 days and up to 1 year ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; analysis of variance (ANOVA) or Kruskal-Wallis test for comparisons involving more than two groups). Will use generalized estimating equations (GEEs) approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  3. Cumulative incidence of relapse [ Time Frame: Up to 1 year ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  4. Relapse-free survival [ Time Frame: Up to 1 year ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  5. Cumulative incidence of frailty [ Time Frame: Up to 1 year ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  6. Cumulative incidence of disability [ Time Frame: Up to 1 year ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  7. Frequency of hospitalization [ Time Frame: Up to 90 days after HCT ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  8. Duration of each hospitalization [ Time Frame: Up to 90 days after HCT ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  9. Number of admissions to intensive care unit [ Time Frame: Up to 90 days after HCT ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  10. Duration of admissions to intensive care unit [ Time Frame: Up to 90 days after HCT ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.

  11. Days out of hospital alive [ Time Frame: Up to 90 days after HCT ]
    Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Vulnerable patients as defined by one or more of the following criteria

    • Aged 65 years or older
    • Having Hematopoietic Cell Transplantation - Comorbidity Index (HCT-CI) scores of >= 3
    • Having frailty as determined by walk speed of < 0.8 m/s using 4-meter walk test
  • Patients considered or referred for allogeneic HCT to treat a hematological malignant or non-malignant disease
  • Able to speak and read English - interaction with the interventionist trainer and endpoint measurement must occur in English
  • Willing and able to provide informed consent
  • Stated willingness to comply with study procedures and reporting requirements
  • Planned allogeneic HCT within 3 weeks - all types of donors and all sorts of conditioning regimens are allowed. Patients with suspected active disease (relatively old disease staging or relatively old intervention) or significant comorbidity (e.g. suspicious untreated pulmonary nodules) based on prior evaluations, that could delay the transplant would be considered for enrollment within a tighter window (10-14 days before allogeneic HCT) to allow for completed pre-HCT work-up evaluations that would confirm readiness to proceed with transplant
  • Able to exercise at low to moderate intensity
  • Adequate cardiopulmonary reserve, as judged by self-reported ability to walk up one flight of stairs, no need for supplemental oxygen, and/or physician judgment

Exclusion Criteria:

  • Orthopedic, neurologic or other problems which prevent safe ambulation and protocol adherence. Information on prior falls and other recent orthopedic or neurologic problems will be used to make judgment about protocol eligibility
  • Participation in another intervention clinical trial with HRQOL as a primary endpoint
  • Planned donor lymphocyte infusion (DLI) within 90 days post-transplant
  • Planned anti-cytotoxic therapies, other than tyrosine kinase inhibitors or single-agent monoclonal antibody, or FLT-3 inhibitors within 90 days of post-transplant unless pre-approved by the protocol principal investigator (PI)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03870750


Contacts
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Contact: Mohamed Sorror 206-667-6298 msorror@fredhutch.org

Locations
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United States, California
Stanford Cancer Center Palo Alto Not yet recruiting
Palo Alto, California, United States, 94304
Contact: Laura Johnston    650-723-0822      
Principal Investigator: Laura Johnston         
United States, Florida
Moffitt Cancer Center Not yet recruiting
Tampa, Florida, United States, 33612
Contact: Joseph Pidala    813-745-2557      
Principal Investigator: Joseph Pidala         
United States, Minnesota
University of Minnesota/Masonic Cancer Center Not yet recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Mukta Arora    612-626-4105      
Principal Investigator: Daniel Weisdorf         
United States, Missouri
Washington University School of Medicine Not yet recruiting
Saint Louis, Missouri, United States, 63110
Contact: Iskra Pusic    314-747-8465      
Principal Investigator: Iskra Pusic         
United States, Washington
Fred Hutch/University of Washington Cancer Consortium Not yet recruiting
Seattle, Washington, United States, 98109
Contact: Mohamed Sorror    206-667-6298      
Principal Investigator: Mohamed Sorror         
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Mohamed Sorror Fred Hutch/University of Washington Cancer Consortium

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Responsible Party: Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT03870750     History of Changes
Other Study ID Numbers: RG1004746
NCI-2019-01097 ( Registry Identifier: NCI / CTRP )
9885 ( Other Identifier: Fred Hutch/University of Washington Cancer Consortium )
First Posted: March 12, 2019    Key Record Dates
Last Update Posted: July 17, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Hematologic Neoplasms
Hematologic Diseases
Neoplasms by Site
Neoplasms