Access Program for Use of AMD3100 to Increase Peripheral Blood Stem Cells for Transplantation in Patients Who Have Failed Standard Therapy Stem Cell Mobilization

Autologous Stem Cell Transplant Clinical Trial

— CUP  

Official title:

Compassionate Use Protocol for the Use of AMD3100 to Mobilize Peripheral Blood Stem Cells for Collection and Transplantation

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT00291811
• Other study ID #: AMD3100-CUP001
• Status: Approved for marketing
• Phase: N/A
• First received: February 13, 2006
• Last updated: February 10, 2014
• Start date: October 2003

Study information

• Verified date: February 2014
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health Canada
• Study type: Expanded Access

Clinical Trial Summary

The purpose of this program is to provide access to AMD3100 for patients who would benefit from an autologous stem cell transplant but who have either previously failed to collect enough cells for transplant following standard therapy or are not considered by their physician to have a reasonable chance of collecting enough cells for transplant.

Description:

The purpose of this program is to provide access to AMD3100 for patients who would benefit from an autologous stem cell transplant but who have either previously failed to collect enough cells for transplant following standard therapy or are not considered by their physician to have a reasonable chance of collecting enough cells for transplant.

Compassionate use is a way to provide experimental treatment to a patient who is not eligible to receive that therapy in a clinical trial, but who has a serious or life-threatening illness for which other treatments are not available.

Peripheral blood stem cells are obtained by apheresis following a stem cell mobilization regimen. The standard of care regimen for stem cell mobilization includes a growth factor, G-CSF. AMD3100 is given in addition to G-CSF prior to each apheresis session. If enough peripheral blood stem cells for transplant are collected, the patient is treated with high dose chemotherapy in preparation for transplant and is transplanted with cells obtained from the AMD3100 and G-CSF regimen. Patients are followed for safety and transplant outcomes for up to 12 months after transplant.

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 78 Years

Eligibility

Inclusion Criteria:

• Patient is eligible for autologous transplant

• Patient has failed previous conventional therapies for stem cell collection, or is not considered by the physician to have a reasonable chance of collecting enough cells for transplant

• Patient has Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

• Patient has recovered from all acute toxic effects of prior chemotherapy

• Patient has white blood cell count greater than 2.5 x 10^9 cells/L

• Patient has absolute neutrophil count (ANC) greater than 1.5 x 10^9 cells/L

• Patient has platelet count greater than 85 x 10^9 cells/L

• Patient has serum creatinine less than or equal to 1.5 mg/dL

• Patient has creatinine clearance greater than 60 mL/min

• Patient has liver function tests less than 2x upper limit of normal

• Patient has left ventricle ejection fraction greater than 45%

• Patient has forced expiratory volume in 1 second (FEV1) greater than 60% of predicted or diffusing capacity of lung carbon monoxide (DLCO) and greater than or equal to 45% of predicted

• Patient has no active infection of hepatitis B or C

• Patient is negative for HIV

• Patient has signed informed consent

• Women of child bearing potential agree to use an approved form of contraception

Exclusion Criteria:

• Patients with leukemia (Note: Patients with multiple myeloma must be evaluated for plasma cells or blasts within 24 hours prior to the first dose of AMD3100)

• Patient has an existing condition which, in the view of the Investigator, renders the patient at high risk from treatment complications

• Patient has a residual acute medical condition resulting from prior chemotherapy

• Patient has brain metastases or carcinomatous meningitis

• Patient has an acute infection

• Patient has a fever (temperature greater than 38 degrees C/100.4 degrees F)

• Patient has hypercalcaemia greater than 1 mg/dL above the upper limit of normal

• Female patient has positive pregnancy test

• Female patient is lactating

• Patient is of child-bearing potential and is unwilling to use adequate birth control

• Patients with actual body weight exceeding 175% of their ideal body weight

• Patients who previously received experimental therapy within 4 weeks of enrolling in this protocol or who are currently enrolled in another experimental protocol

• Patients who experience a deterioration in health between the time of enrollment and transplant (such that they no longer meet entry criteria) may be removed from study at the discretion of the treating physician, principal Investigator, or Sponsor

Study Design

N/A

Related Conditions & MeSH terms

• Autologous Stem Cell Transplant

Intervention

Drug:
• AMD3100 + G-CSF
Subcutaneous injection of 240 mcg/kg on the evening prior to each apheresis session

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company