Trial Outcomes & Findings for Collection of Bone Marrow From Donors Treated With or Without Filgrastim (NCT NCT01149096)

Last Updated: 2020-02-26

Results Overview

The Kaplan-Meier method will be used to estimate the cumulative incidence of short term adverse events defined by having any of the following events: 1) death due to a cause that is unknown or possibly related to G-CSF, 2) development of a malignancy, 3) development of a splenic rupture, or 4) development of a severe acute lung injury possibly related to GCSF therapy.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

13 participants

Primary outcome timeframe

Up to 1 year after donation

Results posted on

2020-02-26

Participant Flow

Participant milestones

Participant milestones
Measure	Arm I (Conventional Bone Marrow Harvest) Donors undergo conventional (i.e., unstimulated) bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies
Overall Study STARTED	6	7
Overall Study COMPLETED	6	6
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Arm I (Conventional Bone Marrow Harvest) Donors undergo conventional (i.e., unstimulated) bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies
Overall Study Lost to Follow-up	0	1

Baseline Characteristics

Collection of Bone Marrow From Donors Treated With or Without Filgrastim

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm I (Conventional Bone Marrow Harvest) n=6 Participants Donors undergo conventional (i.e., unstimulated) bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) n=7 Participants Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies	Total n=13 Participants Total of all reporting groups
Age, Categorical <=18 years	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	6.5 years STANDARD_DEVIATION 5.2 • n=5 Participants	14.4 years STANDARD_DEVIATION 5.9 • n=7 Participants	10.8 years STANDARD_DEVIATION 6.7 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants	6 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants	7 participants n=7 Participants	13 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 1 year after donation

Population: Analysis conducted within G-BM donors only, therefore, no report for the control arm (Conventional bone marrow transplant).

Outcome measures

Outcome measures
Measure	Arm II (Filgrastim, Bone Marrow Harvest) n=7 Participants Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies
Percentage of Participants With Short-term Adverse Events in G-CSF (Filgrastim) Stimulated Bone Marrow (G-BM) Donors	0 Percentage of patients	—

PRIMARY outcome

Timeframe: Up to 1 year after donation

Population: Analysis conducted for stimulated-bone marrow (G-BM) donors only, therefore, no report on the control arm (Conventional bone marrow transplant).

The Kaplan-Meier method will be used to estimate the cumulative incidence of death event in G-BM donors only.

Outcome measures

Outcome measures
Measure	Arm II (Filgrastim, Bone Marrow Harvest) n=7 Participants Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies
Percentage of Participants Who Experienced Death in G-CSF Stimulated-bone Marrow (G-BM) Donors	0 Percentage of patients	—

PRIMARY outcome

Timeframe: Up to 1 year after donation

Population: Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.

Estimate the percentage of patients having non-fatal complications of CTCAE Grades 1 or 2 in standard BM and G-CSF stimulated-bone marrow (G-BM) donors.

Outcome measures

Outcome measures
Measure	Arm II (Filgrastim, Bone Marrow Harvest) n=6 Participants Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) n=7 Participants Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies
Percentage of Participants With Grade 1 or 2 Toxicities	67 percentage of patients	86 percentage of patients

PRIMARY outcome

Timeframe: Up to 1 year after donation

Population: All patients included in analysis. No incidence of grades 3 or 4 toxicities.

Estimate the percentage of patients having non-fatal complications of Grade 3 other than pain (consider Grade 4 for pain only), or any of Grade 4 in standard BM and G-CSF stimulated-bone marrow (G-BM) donors. Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.

Outcome measures

Outcome measures
Measure	Arm II (Filgrastim, Bone Marrow Harvest) n=6 Participants Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) n=7 Participants Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies
Percentage of Participants With Grade 3 or 4 Toxicities	0 percentage of patients	0 percentage of patients

Adverse Events

Arm I (Conventional Bone Marrow Harvest)

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Arm II (Filgrastim, Bone Marrow Harvest)

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 626-447-0064

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Must obtain prior approval.
Publication restrictions are in place

Restriction type: OTHER

Measure	Arm I (Conventional Bone Marrow Harvest) n=6 participants at risk Donors undergo conventional (i.e., unstimulated) bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Laboratory Biomarker Analysis: Optional correlative studies	Arm II (Filgrastim, Bone Marrow Harvest) n=7 participants at risk Donors receive filgrastim subcutaneously on days -4 through 0. Donors then undergo bone marrow harvest on day 0. Bone Marrow Donation: Undergo bone marrow harvest Filgrastim: Given subcutaneously Laboratory Biomarker Analysis: Optional correlative studies
General disorders Back pain	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	42.9% 3/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Bones (Including Sternum and Ribs) pain	0.00% 0/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Dizziness, vertigo, or lightheadedness	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	28.6% 2/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Fatigue	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	85.7% 6/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Fever in the absence of infections	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Headache	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Hip pain	33.3% 2/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	28.6% 2/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders IV Site pain	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Injection site reaction	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	28.6% 2/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Insomnia (Inability to sleep)	0.00% 0/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Joints (Excluding hip) pain	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Limbs (Arm, legs, hands feet) pain	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Loss of Appetite (Anorexia)	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	57.1% 4/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Muscles pain	0.00% 0/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Nausea	0.00% 0/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	42.9% 3/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Neck pain	0.00% 0/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Other pain	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	0.00% 0/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Rashes on skin	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	14.3% 1/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Throat pain	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	57.1% 4/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.
General disorders Vomiting	16.7% 1/6 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.	28.6% 2/7 • First year after Bone Marrow Harvest Modified Toxicity Criteria and Pain Assessment was used with higher grades corresponding to more severe AEs.