Trial Outcomes & Findings for Stem Cell Transplant for Hematologic Diseases (NCT NCT00058825)

NCT ID: NCT00058825

Last Updated: 2016-11-07

Results Overview

Percentage of patients with transplant related mortality

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 27 participants
• Primary outcome timeframe: 100 days
• Results posted on: 2016-11-07

Participant Flow

Participant milestones

Measure	Stem Cell Transplant Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.
Overall Study STARTED	27
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	22

Reasons for withdrawal

Measure	Stem Cell Transplant Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.
Overall Study Relapse	9
Overall Study Progressive Disease	5
Overall Study Graft Failure	5
Overall Study Death	3

Baseline Characteristics

Stem Cell Transplant for Hematologic Diseases

Baseline characteristics by cohort

Measure	Stem Cell Transplant n=27 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.
Age, Continuous	15 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days

Percentage of patients with transplant related mortality

Outcome measures

Measure	Stem Cell Transplant n=27 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs The CLINIMACS CD34 Reagent system was used for cell selection.
Transplant Related Mortality (TRM)	0 percentage of participants Interval 0.0 to 12.8	—

SECONDARY outcome

Timeframe: 30 days

Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days.

Outcome measures

Measure	Stem Cell Transplant n=27 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs The CLINIMACS CD34 Reagent system was used for cell selection.
Time in Days to ANC Engraftment	12 days Interval 6.0 to 21.0	—

SECONDARY outcome

Timeframe: 30 days

Population: Patients engrafted

Number of patients that engrafted who showed a chimerism (donor cells) of greater than 50% in the first 30 days

Outcome measures

Measure	Stem Cell Transplant n=22 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs The CLINIMACS CD34 Reagent system was used for cell selection.
Donor Chimerism Engraftment of Greater Than 50% Yes	20 participants	—
Donor Chimerism Engraftment of Greater Than 50% No	2 participants	—

SECONDARY outcome

Timeframe: 100 days

Number of patients with Acute Graft Versus Host Disease within 100 days post-transplant

Outcome measures

Measure	Stem Cell Transplant n=27 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs The CLINIMACS CD34 Reagent system was used for cell selection.
Acute Graft Versus Host Disease Grade IV	0 participants	—
Acute Graft Versus Host Disease Grade 0	26 participants	—
Acute Graft Versus Host Disease Grade I	0 participants	—
Acute Graft Versus Host Disease Grade II	0 participants	—
Acute Graft Versus Host Disease Grade III	1 participants	—

SECONDARY outcome

Timeframe: 1 year

Number of patients with Chronic Graft Versus Host Disease within 1 year post-transplant

Outcome measures

Measure	Stem Cell Transplant n=27 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs The CLINIMACS CD34 Reagent system was used for cell selection.
Chronic Graft Versus Host Disease Yes	1 participants	—
Chronic Graft Versus Host Disease No	26 participants	—

SECONDARY outcome

Timeframe: 2 years

Relapse-free survival (RFS) was calculated from the time of transplant to the date of relapse, death, or last follow-up, whichever occurred first. Survival data were analyzed by Kaplan-Meier method.

Outcome measures

Measure	Stem Cell Transplant n=27 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs The CLINIMACS CD34 Reagent system was used for cell selection.
2-year Relapse-free Survival	25.9 percentage of participants Interval 11.5 to 43.1	—

SECONDARY outcome

Timeframe: 2 years

Overall survival (OS) was calculated from the time of transplant to death from any cause or censored at last follow-up. Survival data were analyzed by Kaplan-Meier method.

Outcome measures

Measure	Stem Cell Transplant n=27 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs The CLINIMACS CD34 Reagent system was used for cell selection.
2-year Overall Survival	33.3 percentage of participants Interval 16.8 to 50.9	—

SECONDARY outcome

Timeframe: 30 days

Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days.

Outcome measures

Measure	Stem Cell Transplant n=19 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs n=8 Participants The CLINIMACS CD34 Reagent system was used for cell selection.
Number of Patients Who Engrafted With the Isolex/CLINIMACs System	15 participants	7 participants

SECONDARY outcome

Timeframe: 30 days

Engraftment was defined as the day of absolute neutrophil counts (ANC) exceeded 0.5 X 10^9/L on the first of 3 days.

Outcome measures

Measure	Stem Cell Transplant n=19 Participants Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.	CLINIMACs n=8 Participants The CLINIMACS CD34 Reagent system was used for cell selection.
Median Time to Engraftment With the Isolex/CLINIMACs System	12 days Interval 6.0 to 21.0	12 days Interval 9.0 to 17.0

Adverse Events

Stem Cell Transplant

Serious events: 4 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	Stem Cell Transplant n=27 participants at risk Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.
Blood and lymphatic system disorders Failure to engraft	3.7% 1/27 • Number of events 1 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Infections and infestations Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia	3.7% 1/27 • Number of events 1 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Infections and infestations Infection without neutropenia	7.4% 2/27 • Number of events 4 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Metabolism and nutrition disorders Hypokalemia	3.7% 1/27 • Number of events 6 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Respiratory, thoracic and mediastinal disorders Cough	3.7% 1/27 • Number of events 1 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.

Other adverse events

Measure	Stem Cell Transplant n=27 participants at risk Total body irradiation (TBI); Fludarabine and Campath 1H; FK506 or Cyclosporine; Stem Cell Transplant; G-CSF. Campath 1H: Day -5 to Day -2: Campath 1H dose schedule as per institutional SOP. Fludarabine: Day -5 to Day -2: Fludarabine 30 mg/m2. Stem Cell Transplant: Day 0: Donor stem cells infused. Total Body Irradiation (TBI): Day -6: Total body irradiation of 600 cGy as two doses without blocks at a rate of less than or equal to 10 cGy/minute. FK506 (Tacrolimus) or Cyclosporine: Day -2: FK506 or Cyclosporine as medically indicated to prevent GvHD.
Blood and lymphatic system disorders Failure to engraft	14.8% 4/27 • Number of events 4 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Blood and lymphatic system disorders Transfusion: Platelets	25.9% 7/27 • Number of events 16 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Gastrointestinal disorders Anorexia	25.9% 7/27 • Number of events 7 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Gastrointestinal disorders Nausea	11.1% 3/27 • Number of events 7 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Gastrointestinal disorders Vomiting	7.4% 2/27 • Number of events 5 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Hepatobiliary disorders GGT (Gamma-Glutamyl transpeptidase)	11.1% 3/27 • Number of events 5 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Hepatobiliary disorders SGPT (ALT) (serum glutamic pyruvic transaminase)	7.4% 2/27 • Number of events 4 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Infections and infestations Catheter-related infection	7.4% 2/27 • Number of events 3 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Infections and infestations Febrile Neutropenia	11.1% 3/27 • Number of events 3 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Infections and infestations Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia	29.6% 8/27 • Number of events 11 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Infections and infestations Infection without neutropenia	18.5% 5/27 • Number of events 12 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Metabolism and nutrition disorders Hyperglycemia	7.4% 2/27 • Number of events 3 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Metabolism and nutrition disorders Hypokalemia	11.1% 3/27 • Number of events 5 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
General disorders Abdominal Pain or Cramping	7.4% 2/27 • Number of events 2 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
General disorders Bone Pain	11.1% 3/27 • Number of events 3 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	7.4% 2/27 • Number of events 2 • Adverse event data were collected until 30 days after autologous transplants and until 100 days after allogeneic transplants.

Additional Information

George Carrum, MD

Baylor College of Medicine

Email: GCarrum@houstonmethodist.org

Results disclosure agreements

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