Clinical trial NCT03870750: 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
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Hematopoietic and Lymphoid Cell Neoplasm
Non-Neoplastic Hematologic and Lymphocytic Disorder
Lead sponsor: Fred Hutchinson Cancer Center
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
name: National Institutes of Health (NIH)
First posted: 2019-03-12
Enrollment: 458
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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.

Detailed description: 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.

Eligibility criteria: Inclusion Criteria:

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

  * Age 65 years or older
  * Having Hematopoietic Cell Transplantation - Comorbidity Index (HCT-CI) scores of \>= 3 (for patients that could be 20 years old and older)
  * Having frailty as determined by walk speed of \< 0.8 m/s using 4-meter walk test (for patients 50 years old and older)
* 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
* 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, specifically taking into consideration the rare circumstances where subjects are not able to exercise due to either birth deformity or prior traumatic injury that affects their gait
* Adequate cardiopulmonary reserve, as judged by data from the patient's electronic medical record as to whether a patient could 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)

Primary outcomes:
measure: Improvement in health-related quality of life (HRQOL) (Phase II)
timeFrame: First 90 days after HCT
measure: Survival after hematopoietic cell transplantation (HCT) (Phase III)
timeFrame: At 1 year after HCT
measure: Change in HRQOL (Phase III)
timeFrame: Baseline to 90 days post-HCT
Secondary outcomes:
measure: Rate of overall survival
timeFrame: Up to 1 year
measure: Non-relapse mortality
timeFrame: At 90 days and up to 1 year
measure: Cumulative incidence of relapse
timeFrame: Up to 1 year
measure: Relapse-free survival
timeFrame: Up to 1 year
measure: Cumulative incidence of frailty
timeFrame: Up to 1 year
measure: Cumulative incidence of disability
timeFrame: Up to 1 year
measure: Frequency of hospitalization
timeFrame: Up to 90 days after HCT
measure: Duration of each hospitalization
timeFrame: Up to 90 days after HCT
measure: Number of admissions to intensive care unit
timeFrame: Up to 90 days after HCT
measure: Duration of admissions to intensive care unit
timeFrame: Up to 90 days after HCT
measure: Days out of hospital alive
timeFrame: Up to 90 days after HCT
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford Cancer Institute Palo Alto
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Detroit
country: United States
facility: Wayne State University/Karmanos Cancer Institute
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota/Masonic Cancer Center
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: New Hyde Park
country: United States
facility: Northwell Health Cancer Institute
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
state: Texas
city: Seattle
country: United States
facility: Fred Hutch/University of Washington Cancer Consortium
state: Washington
Study officials:
affiliation: Fred Hutch/University of Washington Cancer Consortium
name: Mohamed Sorror, MD, MSc
role: PRINCIPAL_INVESTIGATOR
References: