MDSPhase 1NCT00692926
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
A Phase 1 study testing ALDHbr Umbilical Cord Blood Cells in people with MDS and Anemia, Aplastic, run by Joanne Kurtzberg, MD at 1 site in United States.
Trial summary
The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that have been sorted (collected from a special machine called a cell sorter) and then either… Read more about this study
- Who is it for?
- Ages Up to 55 years
- Conditions the study is for
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism
- What is being tested?
- ALDHbr Umbilical Cord Blood Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2005to Jul 2012
- Phase
- Phase 1
- 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 Up to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- -Hematologic Malignancy: High risk ALL in first complete remission ALL or ANLL in second or subsequent remission ANLL in relapse MDS CML in any chronic phase or accelerated phase Severe aplastic anemia refractory to medical therapy The subject is negative for CNS disease at time of enrollment.
- Inborn errors of metabolism Hurler Syndrome (MPS I) Hurler-Scheie Syndrome Hunter Syndrome (MPS II) Sanfilippo Syndrome (MPS III) Morquio Syndrome (MPS IV) Maroteaux-Lamy Syndrome (MPS VI) Krabbe Disease (Globoid Leukodystrophy) Metachromatic Leukodystrophy (MLD) Adrenoleukodystrophy(ALD and AMN) Sandhoff Disease Tay Sachs Disease The subject does NOT have uncontrolled seizures, apnea, evidence of aspiration pneumonia or evidence of brain stem involvement on MRI scans
- Congenital marrow failure Amegakaryocytic thrombocytopenia TAR Kostmann's Syndrome Schwachman-Diamond Syndrome Blackfan-Diamond Anemia
- Congenital immunodeficiency syndromes requiring myeloablative therapy Wiscott Aldrich Syndrome LAD CGD FEL/HLH CVID/CID
- SUBJECT'S DONOR Subject does NOT have a 6/6 or 5/6 antigen matched related bone marrow donor. Suitably matched cord blood unit with adequate cell dose is available. Unit must be in a dual compartment bag.
- PERFORMANCE STATUS and ORGAN FUNCTION
Show all 18Show fewer
- <55 years of age at time of enrollment.
- Lansky score between 60% and 100%, or a Karnofsky score between 50% and 100%
- Adequate function of other organ systems
- Creatinine < 2.0 mg/dl and creatinine clearance > 50 cc/min/m2
- Hepatic transaminases (ALT/AST) < 4 x normal, bilirubin < 2.0 mg/dl
- Normal cardiac function by echocardiogram or radionuclide scan
- Pulmonary function tests demonstrating FVC, CVC, and FEV1 of >60% of predicted for age. For adult patients DLCO > 60% of predicted. If patient cannot perform PFTs, clearance by the pediatric or adult pulmonologist will be required
- No uncontrolled infections at the time of cytoreduction
- NOT pregnant or lactating (must have a current negative pregnancy test)
- HIV negative
- Subject is not concurrently involved in any other clinical trial that affects engraftment or immune reconstitution (e.g. other hematopoietic growth factors).
- Subject does not have any co-morbid condition, which in the view of the Principal Investigators, renders the patient at too high a risk from treatment complications and regimen related morbidity/mortality.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center
Durham, North Carolina, 27705
About this study
Official title: A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells
The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that have been sorted (collected from a special machine called a cell sorter) and then either infused a few hours after the standard transplant or for some patients grown in a special system in the laboratory prior to the transplant, designed to increase the number of stem cells transplanted. This system is currently in the early phases of testing.
Read the detailed description
The main purpose of this study is to test whether transplantation of umbilical cord blood cells can be safely supplemented with transfusion of a portion of these cells that have been grown in a special system (designed to increase the number of cells transplanted) in the laboratory prior to the transplant. This system is currently in the early phases of testing in a FDA-IND-sponsored clinical trial. If the patient consents to participate in this study, approximately 1/5th (20%) of the cord blood unit selected for the transplant will be treated per protocol. The first 3 patients will receive ALDHbr sorted cells but not primed in culture. This is to test the safety of the ALDHbr cells. The treated cells will be given to the patient on the day of transplant approximately 4 hours after the standard or conventional transplant which will be given from the 80% fraction of the cord blood unit. A total of 26 evaluable patients are to be enrolled as outlined below (protocol has been amended to allow this enrollment):
- 10 evaluable patients who received ALDHbr freshly sorted cells (20% portion)
- 10 evaluable patients who received ALDHbr sorted and cytokine primed cells (20% portion)
- 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted (sort of the UCB unit will be done on Day -1 due to the time it takes to actually perform the sort)
- 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted and cytokine primed (sort and priming will be done on day -5 as described later in the protocol)
- Conditions
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
- Being tested
- ALDHbr Umbilical Cord Blood Cells
- Keywords
- ALDHbr, Umbilical Cord Blood, Transplantation, Hematological malignancy, MDS, severe aplastic anemia, inborn error of metabolism, congenital marrow failure or congenital immunodeficiency syndrome, Hurler's Disease, Tay Sach's, PMD, Hunter's Syndrome, Krabbe, MLD, ALD, ALL, AML
- Medical subject headings
- Anemia, Aplastic, Metabolism, Inborn Errors, Primary Immunodeficiency Diseases, Hematologic Neoplasms, Mucopolysaccharidosis I, Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALDHbr Umbilical Cord Blood CellsBiological
ALDHbr sorted Umbilical Cord Blood Cells
| Study group | Role | What they receive |
|---|---|---|
| 20% primed UCB | Other | 20% of UCB is ALDHbr sorted and primed and give on transplant day after conventional graft Receives: Biological: ALDHbr Umbilical Cord Blood Cells |
| 20% un-primed | Other | 20% of UCB is ALDHbr freshly sorted and give on transplant day 4-8 hrs after conventional graft Receives: Biological: ALDHbr Umbilical Cord Blood Cells |
| Double- 1 unit primed | Other | patient receives 1 conventional UCB unit and 1 unit that has been ALDHbr sorted and primed Receives: Biological: ALDHbr Umbilical Cord Blood Cells |
| Double- 1 unit unprimed | Other | Patient receives 1 UCB unit and a second UCB unit that has been freshly ALDHbr sorted Receives: Biological: ALDHbr Umbilical Cord Blood Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the safety of infusing ex vivo cytokine-primed ALDHbr cells isolated from banked unrelated umbilical cord blood (CB) into patients also receiving a standard unmanipulated unrelated CB transplant delivering >2.5x10e7 nucleated cells per kg.
Measured: Prospective
Secondary outcomes (2)
To describe the biological effect of ex vivo cytokine-primed ALDHbr cells in these patients on neutrophil and platelet engraftment and immune reconstitution.
Measured: prospective
To describe the clinical outcomes in these patients: - incidence of infections -Non relapse mortality -incidence of acute and chronic graft-vs-host disease -overall survival at 180 days
Measured: prospective
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2008 (estimated)
- Study startOct 2005
- Primary completionJul 2012
- Study completionJul 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MDSStudies 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Joanne Kurtzberg, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Aldagen
- Responsible party
- Sponsor-investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Joanne Kurtzberg, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00692926
- Sponsor's protocol ID
- Pro00008970
- Other IDs
- 7177
- Status last verified
- Jun 2017
- First submitted
- 3 Jun 2008
- Last updated
- 29 Jun 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells
- ClinicalTrials.gov ID
- NCT00692926
- Acronym / sponsor ID
- Pro00008970
- Conditions
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
- Keywords
- ALDHbr, Umbilical Cord Blood, Transplantation, Hematological malignancy, MDS, severe aplastic anemia, inborn error of metabolism, congenital marrow failure or congenital immunodeficiency syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Joanne Kurtzberg, MDUniversity, hospital or other
- Collaborators
- Aldagen
- Study start
- Oct 2005
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 6 Jun 2008 (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 main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that have been sorted (collected from a special machine called a cell sorter) and then either infused a few hours after the standard transplant or for some patients grown in a special system in the laboratory prior to the transplant, designed to increase the number of stem cells transplanted. This system is currently in the early phases of testing.
Detailed description
The main purpose of this study is to test whether transplantation of umbilical cord blood cells can be safely supplemented with transfusion of a portion of these cells that have been grown in a special system (designed to increase the number of cells transplanted) in the laboratory prior to the transplant. This system is currently in the early phases of testing in a FDA-IND-sponsored clinical trial. If the patient consents to participate in this study, approximately 1/5th (20%) of the cord blood unit selected for the transplant will be treated per protocol. The first 3 patients will receive ALDHbr sorted cells but not primed in culture. This is to test the safety of the ALDHbr cells. The treated cells will be given to the patient on the day of transplant approximately 4 hours after the standard or conventional transplant which will be given from the 80% fraction of the cord blood unit. A total of 26 evaluable patients are to be enrolled as outlined below (protocol has been amended to allow this enrollment):
- 10 evaluable patients who received ALDHbr freshly sorted cells (20% portion)
- 10 evaluable patients who received ALDHbr sorted and cytokine primed cells (20% portion)
- 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted (sort of the UCB unit will be done on Day -1 due to the time it takes to actually perform the sort)
- 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted and cytokine primed (sort and priming will be done on day -5 as described later in the protocol)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 20% primed UCB | Other | Biological: ALDHbr Umbilical Cord Blood Cells20% of UCB is ALDHbr sorted and primed and give on transplant day after conventional graft |
| 20% un-primed | Other | Biological: ALDHbr Umbilical Cord Blood Cells20% of UCB is ALDHbr freshly sorted and give on transplant day 4-8 hrs after conventional graft |
| Double- 1 unit primed | Other | Biological: ALDHbr Umbilical Cord Blood Cellspatient receives 1 conventional UCB unit and 1 unit that has been ALDHbr sorted and primed |
| Double- 1 unit unprimed | Other | Biological: ALDHbr Umbilical Cord Blood CellsPatient receives 1 UCB unit and a second UCB unit that has been freshly ALDHbr sorted |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- -Hematologic Malignancy: High risk ALL in first complete remission ALL or ANLL in second or subsequent remission ANLL in relapse MDS CML in any chronic phase or accelerated phase Severe aplastic anemia refractory to medical therapy The subject is negative for CNS disease at time of enrollment.
- Inborn errors of metabolism Hurler Syndrome (MPS I) Hurler-Scheie Syndrome Hunter Syndrome (MPS II) Sanfilippo Syndrome (MPS III) Morquio Syndrome (MPS IV) Maroteaux-Lamy Syndrome (MPS VI) Krabbe Disease (Globoid Leukodystrophy) Metachromatic Leukodystrophy (MLD) Adrenoleukodystrophy(ALD and AMN) Sandhoff Disease Tay Sachs Disease The subject does NOT have uncontrolled seizures, apnea, evidence of aspiration pneumonia or evidence of brain stem involvement on MRI scans
- Congenital marrow failure Amegakaryocytic thrombocytopenia TAR Kostmann's Syndrome Schwachman-Diamond Syndrome Blackfan-Diamond Anemia
- Congenital immunodeficiency syndromes requiring myeloablative therapy Wiscott Aldrich Syndrome LAD CGD FEL/HLH CVID/CID
- SUBJECT'S DONOR Subject does NOT have a 6/6 or 5/6 antigen matched related bone marrow donor. Suitably matched cord blood unit with adequate cell dose is available. Unit must be in a dual compartment bag.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| ALDHbr Umbilical Cord Blood CellsALDHbr sorted Umbilical Cord Blood Cells | Biological | 20% primed UCB, 20% un-primed, Double- 1 unit primed, Double- 1 unit unprimed |
Primary outcome
To assess the safety of infusing ex vivo cytokine-primed ALDHbr cells isolated from banked unrelated umbilical cord blood (CB) into patients also receiving a standard unmanipulated unrelated CB transplant delivering >2.5x10e7 nucleated cells per kg.
Time frame: Prospective
2 secondary outcomes
To describe the biological effect of ex vivo cytokine-primed ALDHbr cells in these patients on neutrophil and platelet engraftment and immune reconstitution.
Time frame: prospective
To describe the clinical outcomes in these patients: - incidence of infections -Non relapse mortality -incidence of acute and chronic graft-vs-host disease -overall survival at 180 days
Time frame: prospective
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2017.
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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.
Joanne Kurtzberg, MD principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALDHbr Umbilical Cord Blood Cells in people with MDS and Anemia, Aplastic, run by Joanne Kurtzberg, MD 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 18 inclusion and 0 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 29 Jun 2017. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To assess the safety of infusing ex vivo cytokine-primed ALDHbr cells isolated from banked unrelated umbilical cord blood (CB) into patients also receiving a standard unmanipulated unrelated CB transplant delivering >2.5x10e7 nucleated cells per kg. (time frame: Prospective). 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 29 Jun 2017.