Trial Outcomes & Findings for Autologous Stem Cell Rescue With CD133+ Selected Cells in High-Risk Neuroblastoma (NCT NCT00539500)

Last Updated: 2019-07-26

Results Overview

Engraftment Failure Rate is number of participants with engraftment failure out of total participants. Engraftment failure is defined as failure to achieve an absolute neutrophil count (ANC) \>500/ul by day 42, and has no evidence of donor chimerism on bone marrow examination.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

3 participants

Primary outcome timeframe

Participant evaluation at Day 42, total study up to 3 Years

Results posted on

2019-07-26

Participant Flow

The study was activated in October of 2007 and, due to slow accrual, was closed in December 2010. All recruitment done at UT MD Anderson Cancer Center.

Participant milestones

Participant milestones
Measure	Transplantation CD133+ Cells Stem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan Carboplatin: Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1. Etoposide: 300 mg/m\^2 by vein over 24 hours for 4 days Melphalan: 70 mg/m\^2 Intravenous Bolus for 3 Days Stem Cell Infusion: Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0. ClinicMACS: Device used to process the blood and separate the CD 133+ cells needed for transplantation
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Transplantation CD133+ Cells Stem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan Carboplatin: Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1. Etoposide: 300 mg/m\^2 by vein over 24 hours for 4 days Melphalan: 70 mg/m\^2 Intravenous Bolus for 3 Days Stem Cell Infusion: Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0. ClinicMACS: Device used to process the blood and separate the CD 133+ cells needed for transplantation
Overall Study Disease Progression	1
Overall Study Lost to Follow-up	2

Baseline Characteristics

Autologous Stem Cell Rescue With CD133+ Selected Cells in High-Risk Neuroblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Transplantation CD133+ Cells n=3 Participants Stem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan Carboplatin: Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1. Etoposide: 300 mg/m\^2 by vein over 24 hours for 4 days Melphalan: 70 mg/m\^2 Intravenous Bolus for 3 Days Stem Cell Infusion: Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0. ClinicMACS: Device used to process the blood and separate the CD 133+ cells needed for transplantation
Age, Continuous	4 years n=5 Participants
Age, Categorical <=18 years	3 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Participant evaluation at Day 42, total study up to 3 Years

Population: Study analysis not possible due to small number of participants. Of the 3 participants enrolled, 0 had engraftment failure.

Outcome measures

Outcome measures
Measure	Transplantation CD133+ Cells n=3 Participants Stem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan Carboplatin: Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1. Etoposide: 300 mg/m\^2 by vein over 24 hours for 4 days Melphalan: 70 mg/m\^2 Intravenous Bolus for 3 Days Stem Cell Infusion: Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0. ClinicMACS: Device used to process the blood and separate the CD 133+ cells needed for transplantation
Engraftment Failure Rate	0 Participant

SECONDARY outcome

Timeframe: 3 Years

Toxicity associated with CliniMACS device associated with transplantation of CD133+ cells in high-risk neuroblastoma. Adverse events assessed and graded according to NCI's Common Terminology Criteria for Adverse Events (CTCAE) v3.0.

Outcome measures

Outcome measures
Measure	Transplantation CD133+ Cells n=3 Participants Stem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan Carboplatin: Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1. Etoposide: 300 mg/m\^2 by vein over 24 hours for 4 days Melphalan: 70 mg/m\^2 Intravenous Bolus for 3 Days Stem Cell Infusion: Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0. ClinicMACS: Device used to process the blood and separate the CD 133+ cells needed for transplantation
Number of Participants With Device-related Toxicity Associated With Transplantation of CD133+ Cells	3 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Participant evaluation at Day 42

Population: Three subjects engrafted after receiving the investigational product, no other analysis available. Of the 3 participants enrolled, 0 had engraftment failure.

Engraftment failure is defined as if by day +42 participant does not have an absolute neutrophil count (ANC) \>500/ul, and has no evidence of donor chimerism on bone marrow examination.

Outcome measures

Outcome measures
Measure	Transplantation CD133+ Cells n=3 Participants Stem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan Carboplatin: Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1. Etoposide: 300 mg/m\^2 by vein over 24 hours for 4 days Melphalan: 70 mg/m\^2 Intravenous Bolus for 3 Days Stem Cell Infusion: Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0. ClinicMACS: Device used to process the blood and separate the CD 133+ cells needed for transplantation
Number of Treated Participants With Engraftment Failure at Day 42	0 participants

Adverse Events

Transplantation CD133+ Cells

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Transplantation CD133+ Cells n=3 participants at risk Stem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan Carboplatin: Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1. Etoposide: 300 mg/m\^2 by vein over 24 hours for 4 days Melphalan: 70 mg/m\^2 Intravenous Bolus for 3 Days Stem Cell Infusion: Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0. ClinicMACS: Device used to process the blood and separate the CD 133+ cells needed for transplantation
Metabolism and nutrition disorders Anorexia	66.7% 2/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Nausea	100.0% 3/3 • Number of events 3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
General disorders Pain (Abdomen NOS)	66.7% 2/3 • Number of events 2 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Ascites	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Distention/Bloating	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Mucocitis (Clinical Exam)	100.0% 3/3 • Number of events 3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
General disorders Pain (Other)	100.0% 3/3 • Number of events 3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Vomiting	100.0% 3/3 • Number of events 3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Infections and infestations Febrile Neutropenia	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Diarrhea	100.0% 3/3 • Number of events 3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Skin and subcutaneous tissue disorders Erythema multiforme	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders ALT/AST Disorder	66.7% 2/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders BICARBONATE, SERUM-LOW	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Esophagitis	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Hemorrhage, GI (stoma)	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders Hyperglycemia	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders Hypoalbuminemia	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders Hypocalcemia	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders Hypoglycemia	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders Hypophosphatemia	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Metabolism and nutrition disorders Hypotension	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Nervous system disorders Dizziness	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Nervous system disorders Neuropathy: Sensory	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Nervous system disorders Headache	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.
Gastrointestinal disorders Bloating	33.3% 1/3 • Adverse events collected from Day -8 to Stem Cell Infusion (Day 0) through approximately Day 60 post infusion. Overall study period October 2007 to March 2011.

Additional Information

Laura L. Worth, MD / Professor, Pediatrics

University of Texas MD Anderson Cancer Center

Phone: 713-792-6620

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place