Beta Thalassemia MajorNCT00658385
Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
A study testing GCSF, Central venous line placement, Stem cell Collection (leukapheresis) in people with Beta Thalassemia Major and Congenital Anemias, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial summary
Betathalassemia major is a disease of the blood and bone marrow. You were born with it and it has made you unable to make normal hemoglobin and red cells. You have been receiving red blood cell transfusions all your life. These transfusions do not cure your disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta Thalassemia Major, Congenital Anemias
- What is being tested?
- GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2008to Feb 2011
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must be 18 years or older
- Subjects may be of either gender or of any ethnic background
- Subjects must have a confirmed diagnosis of ßthalassemia major and have been enrolled in a hypertransfusion program with a confirmed annual transfusion of ≥ or = to 100 mL/kg/yr AND ≥ or = to 8 Transfusions of blood per year over a minimum of two years.
- Patients must be off hydroxyurea (HU) or erythropoietin (EPO) treatment for at least three months prior to entry onto the study
- Subjects must have a performance score of Karnofsky > or = to 70 of the time of entry into the study.
- Subjects must have a normal EKG and a normal chest xray
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- Each patient must be willing to participate as a research subject and must sign an informed consent form.
- Subjects must be splenectomized or have no palpable spleen
- Negative pregnancy test, if female
Who cannot join
- Active infections including Hepatitis B and C, HTLV 1 and 2, West Nile Virus, and HIV 1
- Female patient pregnant or breast feeding
- Patients with uncontrolled seizure disorders
- Allergy to GCSF or bacterial E. coli products
- History of sickle cell disease or sickle trait
- History of thrombosis or known thrombophilia
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Pilot Trial to Assess the Feasibility and Safety of GCSF Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
Betathalassemia major is a disease of the blood and bone marrow. You were born with it and it has made you unable to make normal hemoglobin and red cells. You have been receiving red blood cell transfusions all your life. These transfusions do not cure your disease. The problem with transfusions is that they contain a lot of iron. With time iron builds up in your body and will eventually hurt some of your organs . Because of this buildup of iron , you are taking medicine that helps your body get rid of the extra iron.
Today, the only other treatment is bone marrow or stem cell transplant. It can only be done when a matched donor is available. This is most often a brother, sister, or parent. Bone marrow transplant may cure betathalassemia major. If you have a transplant and it is successful, you will no longer have the disease. Without a matched sibling or parent, the standard treatment is to keep having transfusions.
Read more
In the near future, we will be testing a new treatment for making normal hemoglobin and normal red blood cells. We have recreated the healthy hemoglobin gene in a test tube. We are able to use it and put it back into cells. This is called gene therapy. We have been able to put this gene into the stem cells of mice with thalassemia. These mice were cured. We now plan to take that gene and put it into stem cells from people who have betathalassemia major. We will then inject those stem cells back into that person's blood.
In general, we can obtain more stem cells from the blood of a person than from the bone marrow . In order to do so, we must give that person a blood growth factor. The growth factor stimulates the bone marrow to make more stem cells. That growth factor is called granulocyte colony stimulating factor (GCSF), or Filgrastim.
The purpose of this trial is to find out if the drug GCSF has any side effects on you, and if you will make more stem cells in response to it. This trial is not a gene therapy trial. This trial will not help your thalassemia.
- Conditions
- Beta Thalassemia Major, Congenital Anemias
- Being tested
- GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
- Keywords
- GCSF
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GCSF, Central venous line placement, Stem cell Collection (leukapheresis)Genetic
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.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, Central venous line placement, Stem cell Collection (leukapheresis) Receives: Genetic: GCSF, Central venous line placement, Stem cell Collection (leukapheresis) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With no Serious Adverse Events
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
Measured: Up to 14 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2008 (estimated)
- Study startApr 2008
- Primary completionFeb 2011
- Study completionFeb 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Beta Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Farid Boulad, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00658385
- Sponsor's protocol ID
- 08-030
- Status last verified
- Oct 2016
- First submitted
- 10 Apr 2008
- Last updated
- 25 Nov 2016
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Trial to Assess the Feasibility and Safety of GCSF Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
- ClinicalTrials.gov ID
- NCT00658385
- Acronym / sponsor ID
- 08-030
- Conditions
- Beta Thalassemia Major, Congenital Anemias
- Keywords
- GCSF
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- Apr 2008
- Primary completion
- Feb 2011 (actual)
- Study completion
- Feb 2011 (actual)
- First posted
- 15 Apr 2008 (estimated)
- Results
- Posted 25 Nov 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Betathalassemia major is a disease of the blood and bone marrow. You were born with it and it has made you unable to make normal hemoglobin and red cells. You have been receiving red blood cell transfusions all your life. These transfusions do not cure your disease. The problem with transfusions is that they contain a lot of iron. With time iron builds up in your body and will eventually hurt some of your organs . Because of this buildup of iron , you are taking medicine that helps your body get rid of the extra iron.
Today, the only other treatment is bone marrow or stem cell transplant. It can only be done when a matched donor is available. This is most often a brother, sister, or parent. Bone marrow transplant may cure betathalassemia major. If you have a transplant and it is successful, you will no longer have the disease. Without a matched sibling or parent, the standard treatment is to keep having transfusions.
In the near future, we will be testing a new treatment for making normal hemoglobin and normal red blood cells. We have recreated the healthy hemoglobin gene in a test tube. We are able to use it and put it back into cells. This is called gene therapy. We have been able to put this gene into the stem cells of mice with thalassemia. These mice were cured. We now plan to take that gene and put it into stem cells from people who have betathalassemia major. We will then inject those stem cells back into that person's blood.
In general, we can obtain more stem cells from the blood of a person than from the bone marrow . In order to do so, we must give that person a blood growth factor. The growth factor stimulates the bone marrow to make more stem cells. That growth factor is called granulocyte colony stimulating factor (GCSF), or Filgrastim.
The purpose of this trial is to find out if the drug GCSF has any side effects on you, and if you will make more stem cells in response to it. This trial is not a gene therapy trial. This trial will not help your thalassemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Genetic: GCSF, Central venous line placement, Stem cell Collection (leukapheresis)GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, Central venous line placement, Stem cell Collection (leukapheresis) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subjects must be 18 years or older
- Subjects may be of either gender or of any ethnic background
- Subjects must have a confirmed diagnosis of ßthalassemia major and have been enrolled in a hypertransfusion program with a confirmed annual transfusion of ≥ or = to 100 mL/kg/yr AND ≥ or = to 8 Transfusions of blood per year over a minimum of two years.
- Patients must be off hydroxyurea (HU) or erythropoietin (EPO) treatment for at least three months prior to entry onto the study
- Subjects must have a performance score of Karnofsky > or = to 70 of the time of entry into the study.
Key exclusion criteria (6 listed)
- Active infections including Hepatitis B and C, HTLV 1 and 2, West Nile Virus, and HIV 1
- Female patient pregnant or breast feeding
- Patients with uncontrolled seizure disorders
- Allergy to GCSF or bacterial E. coli products
- History of sickle cell disease or sickle trait
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 |
|---|---|---|
| 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. | Genetic | 1 |
Primary outcome
Number of Participants With no Serious Adverse Events
Time frame: Up to 14 Days
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Nov 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
Related trials for Beta Thalassemia Major
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
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.
Farid Boulad, MD principal investigator
Memorial Sloan Kettering Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing GCSF, Central venous line placement, Stem cell Collection (leukapheresis) in people with Beta Thalassemia Major and Congenital Anemias, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
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 9 inclusion and 6 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: finished, as of the sponsor's last update on 25 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Yes. Results are posted in the registry (first posted 25 Nov 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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.
What does the primary outcome measure?
Number of Participants With no Serious Adverse Events (time frame: Up to 14 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Nov 2016.