Autologous Stem Cell TransplantCUPNCT00291811
Access Program for Use of AMD3100 to Increase Peripheral Blood Stem Cells for Transplantation in Patients Who Have Failed Standard Therapy Stem Cell Mobilization
A study testing AMD3100 + G-CSF in people with Autologous Stem Cell Transplant, run by Genzyme, a Sanofi Company.
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… Read more about this study
- Who is it for?
- Ages 18 to 78 years
- Conditions the study is for
- Autologous Stem Cell Transplant
- What is being tested?
- AMD3100 + G-CSF
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Oct 2003to not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 78 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 16Show fewer
- 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
Who cannot join
- 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)
Show all 13Show fewer
- 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
About this study
Official title: Compassionate Use Protocol for the Use of AMD3100 to Mobilize Peripheral Blood Stem Cells for Collection and Transplantation
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.
Read the detailed 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.
- Conditions
- Autologous Stem Cell Transplant
- Being tested
- AMD3100 + G-CSF
- Keywords
- AMD3100, G-CSF
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMD3100 + G-CSFDrug
Also called Plerixafor(injection)
Subcutaneous injection of 240 mcg/kg on the evening prior to each apheresis session
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2006 (estimated)
- Study startOct 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autologous Stem Cell TransplantStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00291811
- Sponsor's protocol ID
- AMD3100-CUP001
- Acronym
- CUP
- Status last verified
- Feb 2014
- First submitted
- 13 Feb 2006
- Last updated
- 11 Feb 2014
- Results posted
- No
Publications
Deol A, Abrams J, Masood A, Al-Kadhimi Z, Abidi MH, Ayash L, Lum LG, Ratanatharathorn V, Uberti JP. Long-term follow up of patients proceeding to transplant using plerixafor mobilized stem cells and incidence of secondary myelodysplastic syndrome/AML. Bone Marrow Transplant. 2013 Aug;48(8):1112-6. doi: 10.1038/bmt.2013.10. Epub 2013 Mar 11.
PubMed 23474805
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use Protocol for the Use of AMD3100 to Mobilize Peripheral Blood Stem Cells for Collection and Transplantation
- ClinicalTrials.gov ID
- NCT00291811
- Acronym / sponsor ID
- CUP / AMD3100-CUP001
- Conditions
- Autologous Stem Cell Transplant
- Keywords
- AMD3100, G-CSF
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Oct 2003
- First posted
- 15 Feb 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
Who may qualify
- Ages
- 18 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AMD3100 + G-CSFAlso: Plerixafor(injection)Subcutaneous injection of 240 mcg/kg on the evening prior to each apheresis session | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2014.
- Derived from this study: PubMed 23474805Deol A, Abrams J, Masood A, Al-Kadhimi Z, Abidi MH, Ayash L, Lum LG, Ratanatharathorn V, Uberti JP. Long-term follow up of patients proceeding to transplant using plerixafor mobilized stem cells and incidence of secondary myelodysplastic syndrome/AML. Bone Marrow Transplant. 2013 Aug;48(8):1112-6. doi: 10.1038/bmt.2013.10. Epub 2013 Mar 11.
Related trials for Autologous Stem Cell Transplant
RecruitingPhase 2
MT2022-60: Ph 2 Study of Pembro+ BEAM With ASCT for Relapsed Hodgkin Lymphoma
Autologous Stem Cell Transplant, Classic Hodgkin Lymphoma
RecruitingObservational study
ATLG Levels in Autologous HSCT for Multiple Sclerosis
Multiple Sclerosis, Autologous Hematopoietic Stem Cell Transplant
Active, not recruitingPhase 2
Isatuximab, Lenalidomide, Bortezomib, and Dexamethasone in NDMM
Multiple Myeloma, Newly Diagnosed Multiple Myeloma (NDMM)
Recruiting
Nutrition OUtReach In Systems of Healthcare
ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION, Autologous Haemopoietic Stem Cell Transplant
Active, not recruitingPhase 2
Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases
Autoimmune Disease, Neurologic Autoimmune Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
Questions about this trial
What is this clinical trial studying?
A study testing AMD3100 + G-CSF in people with Autologous Stem Cell Transplant, run by Genzyme, a Sanofi Company.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 11 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00291811, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2014; AzTrials last refreshed its copy on 2 Nov 2026.