Trial Outcomes & Findings for G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome (NCT NCT00228813)

NCT ID: NCT00228813

Last Updated: 2017-09-11

Results Overview

The number of participants who received in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor who reached engraftment by Day 45.

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 8 participants
• Primary outcome timeframe: Day 45
• Results posted on: 2017-09-11

Participant Flow

Participants were recruited from Children's Healthcare of Atlanta between January 2005 and December 2013.

Participant milestones

Measure	Granulocyte Colony Stimulating Factor (G-CSF) Stimulation Participants with hematologic malignancies or bone marrow failure syndrome received in vivo T-cell depleted granulocyte colony stimulating factor (G-CSF) stimulated bone marrow from a partially mismatched related donor.
Overall Study STARTED	8
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	Granulocyte Colony Stimulating Factor (G-CSF) Stimulation Participants with hematologic malignancies or bone marrow failure syndrome received in vivo T-cell depleted granulocyte colony stimulating factor (G-CSF) stimulated bone marrow from a partially mismatched related donor.
Overall Study Death	4

Baseline Characteristics

G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome

Baseline characteristics by cohort

Measure	Granulocyte Colony Stimulating Factor (G-CSF) Stimulation n=8 Participants Participants with hematologic malignancies or bone marrow failure syndrome received in vivo T-cell depleted granulocyte colony stimulating factor (G-CSF) stimulated bone marrow from a partially mismatched related donor.
Age, Categorical <=18 years	7 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 45

The number of participants who received in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor who reached engraftment by Day 45.

Outcome measures

Measure	Granulocyte Colony Stimulating Factor (G-CSF) Stimulation n=8 Participants Participants received bone marrow from donors who underwent Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection.
Engraftment Rate	8 particpants

PRIMARY outcome

Timeframe: Day 100

Population: Participants who who developed acute GVHD was measured at Day 100 post bone marrow transplant.

The number of participants diagnosed with new acute graft-versus-host disease (GVHD).

Outcome measures

Measure	Granulocyte Colony Stimulating Factor (G-CSF) Stimulation n=8 Participants Participants received bone marrow from donors who underwent Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection.
Incidence of Acute Graft-versus-host Disease	6 participants

PRIMARY outcome

Timeframe: Duration of Study (Up to two years)

Population: Participants who survived beyond Day 100 post bone marrow transplant.

The number of participants diagnosed with chronic graft-versus-host disease (GVHD).

Outcome measures

Measure	Granulocyte Colony Stimulating Factor (G-CSF) Stimulation n=6 Participants Participants received bone marrow from donors who underwent Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection.
Incidence of Chronic Graft-versus-host Disease	2 participants

Adverse Events

Granulocyte Colony Stimulating Factor (G-CSF) Stimulation

Serious events: 4 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Granulocyte Colony Stimulating Factor (G-CSF) Stimulation n=8 participants at risk Participants with hematologic malignancies or bone marrow failure syndrome received in vivo T-cell depleted granulocyte colony stimulating factor (G-CSF) stimulated bone marrow from a partially mismatched related donor.
Blood and lymphatic system disorders Recurrent Disease	25.0% 2/8 • Number of events 2 • Adverse events were collected throughout the duration of the study and follow up period (up to two years).
General disorders Multi-organ system failure	25.0% 2/8 • Number of events 2 • Adverse events were collected throughout the duration of the study and follow up period (up to two years).

Other adverse events

Adverse event data not reported

Additional Information

Kuang-Yueh Chiang, MD

Emory University

Phone: 404-785-0858

Email: kchian2@emory.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place