Trial Outcomes & Findings for Denosumab in Treating Patients With Bone Loss Due to Donor Stem Cell Transplant (NCT NCT03925532)

Results Overview

Day 0 dual x-ray absorptiometry (DXA) scan and day 465 DXA scan will be compared based on the percent change in BMD in the Total hip in allogeneic HSCT patients who have experienced either at least 5% BMD loss between baseline (pre- HSCT) and day + 100 post-HSCT, or who have osteopenia or osteoporosis at either the pre-bone marrow transplant or day + 100 DXA scan.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

At baseline and 465 days post-HSCT

Results posted on

2024-03-13

Participant Flow

Participant milestones

Participant milestones
Measure	Supportive Care (Denosumab) Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Overall Study STARTED	30
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Supportive Care (Denosumab) Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Overall Study Lost to Follow-up	1

Baseline Characteristics

Denosumab in Treating Patients With Bone Loss Due to Donor Stem Cell Transplant

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Supportive Care (Denosumab) n=30 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=5 Participants
Age, Categorical >=65 years	10 Participants n=5 Participants
Age, Continuous	60.38 years STANDARD_DEVIATION 9.65 • n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants
Race (NIH/OMB) White	26 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: At baseline and 465 days post-HSCT

Population: All treated and eligible patients that had measures at day 0 and day 465

Day 0 dual x-ray absorptiometry (DXA) scan and day 465 DXA scan will be compared based on the percent change in BMD in the Total hip in allogeneic HSCT patients who have experienced either at least 5% BMD loss between baseline (pre- HSCT) and day + 100 post-HSCT, or who have osteopenia or osteoporosis at either the pre-bone marrow transplant or day + 100 DXA scan.

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=21 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Mean Total Hip Percent Change in Bone Mineral Density (BMD)	-4.28 percent change Standard Deviation 7.13

PRIMARY outcome

Timeframe: From the time of enrollment up to 465 days post-HSCT

Population: All treated and eligible patients with measures collected

Parameter estimate of the slope of the regression model of the hip bone mineral density change at day 465 post-HSCT regressed on the enrollment BMD levels.

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=21 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Slope in Hip Bone Mineral Density (g/cm^2 Per Day) Regressed on Time in Dual	0.04026 g/cm^2 per day Interval -0.11686 to 0.1973

PRIMARY outcome

Timeframe: At baseline and 465 days post-HSCT

Population: All treated and eligible patients that had measure taken at day 0 and day 465

Day 0 dual x-ray absorptiometry (DXA) scan and day 465 DXA scan will be compared based on the percent change in BMD in lumbar spine in allogeneic HSCT patients who have experienced either at least 5% BMD loss between baseline (pre- HSCT) and day + 100 post-HSCT, or who have osteopenia or osteoporosis at either the pre-bone marrow transplant or day + 100 DXA scan.

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=21 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Mean Lumbar Spine Percent Change in Bone Mineral Density (BMD)	0.77 percentage change Standard Deviation 5.85

PRIMARY outcome

Timeframe: At baseline, at time of enrollment (day 100 post-hematopoietic stem cell transplantation [HSCT])

Population: All treated and eligible patients with measure at day 0 and day 100

Day 100 dual x-ray absorptiometry (DXA) scan will be compared based on the percent change in BMD in the Total hip in allogeneic HSCT patients who have experienced either at least 5% BMD loss between baseline (pre- HSCT) and day + 100 post-HSCT, or who have osteopenia or osteoporosis at either the pre-bone marrow transplant or day + 100 DXA scan.

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=29 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Mean Total Hip Percent Change in Bone Mineral Density (BMD)	-7.61 percentage change Standard Deviation 4.54

PRIMARY outcome

Timeframe: From the time of enrollment up to 465 days post-HSCT

Population: All treated and eligible patients with measures collected

Parameter estimate of the slope of the regression model of the lumbar spine bone mineral density change at day 465 post-HSCT regressed on the enrollment BMD levels.

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=21 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Slope in Lumbar Spine Bone Mineral Density (g/cm^2 Per Day) Regressed on Time in Dual	-0.09690 g/cm^2 per day Interval -0.24883 to 0.05503

SECONDARY outcome

Timeframe: Baseline up to 100 days post-HSCT

Population: All treated and eligible patients with measures at day 0 and day 100

Day 100 dual x-ray absorptiometry (DXA) scan will be compared based on the percent change in BMD in the lumbar spine in allogeneic HSCT patients who have experienced either at least 5% BMD loss between baseline (pre- HSCT) and day + 100 post-HSCT, or who have osteopenia or osteoporosis at either the pre-bone marrow transplant or day + 100 DXA scan.

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=29 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Mean Lumbar Spine Percent Change in BMD	-4.04 percentage change Standard Deviation 5.46

SECONDARY outcome

Timeframe: Up to 1 year post-HSCT

Population: All treated and eligible patients

The number of participants with bone fractures tabulated overall

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=21 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Frequency of Bone Fractures	0 Participants

SECONDARY outcome

Timeframe: Up to 30 days

Population: All treated and eligible patients

Including but not limited to the following: Injection/hypersensitivity related reactions; osteonecrosis of the jaw; graft versus host disease. Will be assessed according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5 and tabulated by grade.

Outcome measures

Outcome measures
Measure	Supportive Care (Denosumab) n=30 Participants Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Number of Participants That Experienced Any AE	7 Participants

Adverse Events

Supportive Care (Denosumab)

Serious events: 5 serious events

Other events: 2 other events

Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure	Supportive Care (Denosumab) n=30 participants at risk Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Injury, poisoning and procedural complications Wrist fracture	3.3% 1/30 • Number of events 1 • AE Time frame - Baseline, weekly until 30 day follow-up.
General disorders General disorders and administration site conditions - Other, specify	3.3% 1/30 • Number of events 1 • AE Time frame - Baseline, weekly until 30 day follow-up.
Injury, poisoning and procedural complications Fracture	6.7% 2/30 • Number of events 2 • AE Time frame - Baseline, weekly until 30 day follow-up.
Injury, poisoning and procedural complications Hip fracture	3.3% 1/30 • Number of events 1 • AE Time frame - Baseline, weekly until 30 day follow-up.

Other adverse events

Other adverse events
Measure	Supportive Care (Denosumab) n=30 participants at risk Patients receive 2 doses of denosumab SC between days 70-130 and days 250-310 after allogeneic hematopoietic stem cell transplant in the absence of disease progression or unacceptable toxicity. Denosumab: Given SC
Investigations Neutrophil count decreased	3.3% 1/30 • Number of events 1 • AE Time frame - Baseline, weekly until 30 day follow-up.
Musculoskeletal and connective tissue disorders Back pain	3.3% 1/30 • Number of events 1 • AE Time frame - Baseline, weekly until 30 day follow-up.

Additional Information

Senior Administrator, Compliance - Clinical Research Services

Roswell Park Cancer Institute

Phone: 7168451300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place