Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

• ClinicalTrials.gov Identifier: NCT00658385
• Recruitment Status: Completed
• First Posted: April 15, 2008
• Results First Posted: November 25, 2016
• Last Update Posted: November 25, 2016
• Sponsor: Memorial Sloan Kettering Cancer Center
• Information provided by (Responsible Party): Memorial Sloan Kettering Cancer Center

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Beta Thalassemia Major; Congenital Anemias
• Intervention: Genetic: GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
• Enrollment: 5

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	GCSF, Neupogen, Filgrastim, Central Venous Line Placement, SCT
Arm/Group Description	GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, Central venous line placement, Stem cell Collection (leukapheresis) GCSF, Central venous line placement, Stem cell Collection (leukapheresis): Daily injections under the skin of a GCSF. This is done for 5 to 6 days. On days 1, 3,5, and if need on day 6. To collect stem cells, we need good access to this blood. If the patient has good veins, we do this by placing an IV on each one of their arms. The peripheral blood stem cell collection is usually an outpatient procedure and takes about 3 to 4 hours. You will have blood work and a physical exam on days one, three, and five while you are getting GCSF. These will be done again 24 hours after your stem cells are collected.
Period Title: Overall Study
Started	5
Completed	5
Not Completed	0

Baseline Characteristics

Arm/Group Title		GCSF, Neupogen, Filgrastim, Central Venous Line Placement, SCT
Arm/Group Description		GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, Central venous line placement, Stem cell Collection (leukapheresis) GCSF, Central venous line placement, Stem cell Collection (leukapheresis): Daily injections under the skin of a GCSF. This is done for 5 to 6 days. On days 1, 3,5, and if need on day 6. To collect stem cells, we need good access to this blood. If the patient has good veins, we do this by placing an IV on each one of their arms. The peripheral blood stem cell collection is usually an outpatient procedure and takes about 3 to 4 hours. You will have blood work and a physical exam on days one, three, and five while you are getting GCSF. These will be done again 24 hours after your stem cells are collected.
Overall Number of Baseline Participants		5
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	5 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	5 100.0%
	>=65 years	0 0.0%
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	5 participants
	Female	4 80.0%
	Male	1 20.0%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With no Serious Adverse Events
Description	The entered value represents the number of participants with the absence of serious adverse events. G-CSF mobilization will be considered safe if there are no more than 1 of 5 patients with SAEs
Time Frame	Up to 14 Days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	GCSF, Neupogen, Filgrastim, Central Venous Line Placement, SCT
Arm/Group Description:	GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, Central venous line placement, Stem cell Collection (leukapheresis) GCSF, Central venous line placement, Stem cell Collection (leukapheresis): Daily injections under the skin of a GCSF. This is done for 5 to 6 days. On days 1, 3,5, and if need on day 6. To collect stem cells, we need good access to this blood. If the patient has good veins, we do this by placing an IV on each one of their arms. The peripheral blood stem cell collection is usually an outpatient procedure and takes about 3 to 4 hours. You will have blood work and a physical exam on days one, three, and five while you are getting GCSF. These will be done again 24 hours after your stem cells are collected.
Overall Number of Participants Analyzed	5
Measure Type: Number; Unit of Measure: participants
	5

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	GCSF, Neupogen, Filgrastim, Central Venous Line Placement, SCT
Arm/Group Description	GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, Central venous line placement, Stem cell Collection (leukapheresis) GCSF, Central venous line placement, Stem cell Collection (leukapheresis): Daily injections under the skin of a GCSF. This is done for 5 to 6 days. On days 1, 3,5, and if need on day 6. To collect stem cells, we need good access to this blood. If the patient has good veins, we do this by placing an IV on each one of their arms. The peripheral blood stem cell collection is usually an outpatient procedure and takes about 3 to 4 hours. You will have blood work and a physical exam on days one, three, and five while you are getting GCSF. These will be done again 24 hours after your stem cells are collected.
All-Cause Mortality
	GCSF, Neupogen, Filgrastim, Central Venous Line Placement, SCT
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	GCSF, Neupogen, Filgrastim, Central Venous Line Placement, SCT
	Affected / at Risk (%)	# Events
Total	0/5 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	GCSF, Neupogen, Filgrastim, Central Venous Line Placement, SCT
	Affected / at Risk (%)	# Events
Total	4/5 (80.00%)
Blood and lymphatic system disorders
Anemia † 1	4/5 (80.00%)	4
Investigations
Blood bilirubin increased † 1	2/5 (40.00%)	2
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, CTC-3.0

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Dr. Farid Boulad
• Organization: Memorial Sloan Kettering Cancer Center
• Phone: 212-639-6684
• EMail: bouladf@mskcc.org

• Responsible Party: Memorial Sloan Kettering Cancer Center
• ClinicalTrials.gov Identifier: NCT00658385
• Other Study ID Numbers: 08-030
• First Submitted: April 10, 2008
• First Posted: April 15, 2008
• Results First Submitted: April 7, 2015
• Results First Posted: November 25, 2016
• Last Update Posted: November 25, 2016