Leukemia, Myeloid, ChronicPhase 2NCT00393380
Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor
A Phase 2 study testing Parathyroid Hormone (teriparatide) in people with Leukemia, Myeloid, Chronic and Anemia, Aplastic, run by The Emmes Company, LLC at 4 sites in United States.
Trial summary
The purpose of this study is to determine whether the addition of parathyroid hormone after a sequential cord blood transplant will improve engraftment, which is the ability of the transplanted stem cells to grow and to successfully begin producing new blood cells. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Leukemia, Myeloid, Chronic, Anemia, Aplastic, Myelofibrosis
- What is being tested?
- Parathyroid Hormone (teriparatide)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 4 sitesUnited States
- Study dates
- Sep 2006to Mar 2012
- Phase
- Phase 2
- 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 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- One of the following diagnoses:
- Chronic myelogenous leukemia (CML) accelerated phase or second stable phase; individuals in the first chronic phase are eligible if they have resistance to imatinib
- Myelodysplasia
- Aplastic anemia that is not responding to immunosuppressive therapy
- Myelofibrosis, either primary or secondary to polycythemia vera
- Relapsed lymphoma or Hodgkin's disease
Show all 15Show fewer
- Stage III/IV chronic lymphocytic leukemia (CLL), relapsed after or refractory to at least one fludarabine containing regimen
- Acute myelogenous leukemia (AML) or acute lymphoblastic leukemia (ALL) in complete remission (CR) 2 or greater, or CR 1 with high risk features
- No prior autologous stem cell transplant
- Eastern Cooperative Oncology Group (ECOG) performance status of less than 2
- Lack of 6/6 or 5/6 matched related donor OR lack of 10/10 matched unrelated donor OR no available donor in the appropriate time frame to perform a potentially curative stem cell transplant
- Diffusing capacity of the lung for carbon monoxide (DLCO) greater than 50% of predicted value
- Left ventricular ejection fraction (LVEF) greater than 50% of predicted value
- Calcium levels less than 10.5 mg/dl
- Phosphate levels greater than 1.6 mg/dl
Who cannot join
- Heart disease, as determined by symptomatic congestive heart failure, radionuclide ventriculogram (RVG), or echocardiogram-determined LVEF of less than 50%, active angina pectoris, or uncontrolled high blood pressure
- Pulmonary disease, as determined by severe chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of less than 50% of predicted value
- Kidney disease, as determined by serum creatinine levels greater than 2.0 mg/dl
- Liver disease, as determined by serum bilirubin levels greater than 2.0 mg/dl (except in the case of Gilbert's syndrome or hemolytic anemia in which the bilirubin can be elevated greater than 2.0mg/dl), SGOT or SGPT greater than 3 times the upper limit of normal
- Neurologic disease, as determined by symptomatic leukoencephalopathy, active central nervous system (CNS) cancer, or other neuropsychiatric abnormalities that may prevent transplantation (previous CNS cancer and presently in CR is acceptable)
- HIV antibodies
- Uncontrolled infection
- Pregnant or breastfeeding
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02115
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
University of Florida
Gainesville, Florida, 32610
About this study
Official title: A Phase II Study of Parathyroid Hormone Following Myeloablative Sequential Unrelated Cord Blood Transplantation
The purpose of this study is to determine whether the addition of parathyroid hormone after a sequential cord blood transplant will improve engraftment, which is the ability of the transplanted stem cells to grow and to successfully begin producing new blood cells.
Read the detailed description
In this phase II, single stage study, participants will include 40 adults who are candidates for a hematopoietic stem cell transplant. All participants will undergo a sequential cord blood transplant using a well-known myeloablative regimen of fludarabine, cyclophosphamide, and total body irradiation, which is appropriate for those individuals who are likely to benefit from an ablative regimen. Tacrolimus will be combined with mycophenolate mofetil (MMF) for the graft-versus-host disease (GVHD) prophylaxis regimen. Parathyroid hormone (PTH) will be added to this regimen in an attempt to improve engraftment. PTH is an approved drug with minimal side effects in individuals with osteoporosis; the dose of PTH has been determined from a phase I study in individuals with hematologic cancer.
- Conditions
- Leukemia, Myeloid, Chronic, Anemia, Aplastic, Myelofibrosis, Lymphoma, Hodgkin Disease, Leukemia, Lymphocytic, Chronic
- Being tested
- Parathyroid Hormone (teriparatide)
- Keywords
- Myelogenous Leukemia, Chronic, Myelodysplasia, Parathyroid Hormone, Umbilical Cord Blood Stem Cell Transplantation
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Anemia, Aplastic, Primary Myelofibrosis, Lymphoma, Hodgkin Disease, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Parathyroid Hormone (teriparatide)Drug
Also called Parathyroid hormone, PTH, teriparatide
Day +1: PTH 40 mcg, Day +2: PTH 60 mcg, Day +3: PTH 80 mcg, Day +4 to Day +29 or until ANC>2000/microL: PTH 100 mcg
| Study group | Role | What they receive |
|---|---|---|
| Parathyroid Hormone (teriparatide) | Experimental | Parathyroid hormone after double umbilical cord blood transplant. Receives: Drug: Parathyroid Hormone (teriparatide) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500)
Median time to neutrophil engraftment (defined as an absolute neutrophil count [ANC] greater than 500)
Measured: Statistic is calculated at Day 42 but ANC counts are measured daily up through discharge.
Secondary outcomes (7)
Cumulative Incidence of Acute GVHD Grades II-IV at Day 100
Cumulative Incidence of Acute GVHD Grades II-IV at day 100
Measured: Measured at Day 100
Cumulative Incidence of Chronic GVHD
Cumulative Incidence of Chronic GVHD
Measured: Measured at 2 years
Platelet Engraftment (Greater Than 20,000)
Platelet engraftment (greater than 20,000)
Measured: Measured at Day 180
100-day Transplant-related Mortality
100-day transplant-related mortality
Measured: Measured at Day 100
Cumulative Incidence of Relapse
Cumulative Incidence of Relapse
Measured: Measured at 2 years
Overall Survival
Overall Survival
Measured: Measured at 2 years
Disease-free Survival
Disease-free survival
Measured: Measured at 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2006 (estimated)
- Study startSep 2006
- Primary completionNov 2009
- Study completionMar 2012
Why it stopped: Study stopped because of toxicity concerns.
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Leukemia, Myeloid, ChronicStudies 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 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Emmes Company, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Karen K. Ballen, MD, Principal investigator, Massachusetts General HospitalJoseph Antin, MD, Principal investigator, Dana-Farber Cancer InstituteDavid Avigan, MD, Principal investigator, Beth Israel Deaconess Medical CenterElizabeth J Shpall, MD, Principal investigator, MD Anderson Cancer Research CenterColleen Delaney, MD, Principal investigator, Fred Hutchinson Cancer CenterRam Kamble, MD, Principal investigator, Baylor College of MedicineKatarzyna Jamieson, M.D., Principal investigator, University of FloridaPhilip McCarthy, M.D., Principal investigator, Roswell Park Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00393380
- Sponsor's protocol ID
- 435
- Other IDs
- U54HL081030-02
- Status last verified
- Apr 2013
- First submitted
- 25 Oct 2006
- Last updated
- 29 Apr 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Ballen K, Mendizabal AM, Cutler C, Politikos I, Jamieson K, Shpall EJ, Dey BR, Attar E, McAfee S, Delaney C, McCarthy P, Ball ED, Kamble R, Avigan D, Maziarz RT, Ho VT, Koreth J, Alyea E, Soiffer R, Wingard JR, Boussiotis V, Spitzer TR, Antin JH. Phase II trial of parathyroid hormone after double umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2012 Dec;18(12):1851-8. doi: 10.1016/j.bbmt.2012.06.016. Epub 2012 Jul 2.
PubMed 22766223
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Parathyroid Hormone Following Myeloablative Sequential Unrelated Cord Blood Transplantation
- ClinicalTrials.gov ID
- NCT00393380
- Acronym / sponsor ID
- 435
- Conditions
- Leukemia, Myeloid, Chronic, Anemia, Aplastic, Myelofibrosis, Lymphoma, Hodgkin Disease, Leukemia, Lymphocytic, Chronic
- Keywords
- Myelogenous Leukemia, Chronic, Myelodysplasia, Parathyroid Hormone, Umbilical Cord Blood Stem Cell Transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study stopped because of toxicity concerns.
- Enrolment
- 13 participants (actual)
- Lead sponsor
- The Emmes Company, LLCIndustry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Sep 2006
- Primary completion
- Nov 2009 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 27 Oct 2006 (estimated)
- Results
- Posted 7 Dec 2012
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 study is to determine whether the addition of parathyroid hormone after a sequential cord blood transplant will improve engraftment, which is the ability of the transplanted stem cells to grow and to successfully begin producing new blood cells.
Detailed description
In this phase II, single stage study, participants will include 40 adults who are candidates for a hematopoietic stem cell transplant. All participants will undergo a sequential cord blood transplant using a well-known myeloablative regimen of fludarabine, cyclophosphamide, and total body irradiation, which is appropriate for those individuals who are likely to benefit from an ablative regimen. Tacrolimus will be combined with mycophenolate mofetil (MMF) for the graft-versus-host disease (GVHD) prophylaxis regimen. Parathyroid hormone (PTH) will be added to this regimen in an attempt to improve engraftment. PTH is an approved drug with minimal side effects in individuals with osteoporosis; the dose of PTH has been determined from a phase I study in individuals with hematologic cancer.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Parathyroid Hormone (teriparatide) | Experimental | Drug: Parathyroid Hormone (teriparatide)Parathyroid hormone after double umbilical cord blood transplant. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (15 listed)
- One of the following diagnoses:
- No prior autologous stem cell transplant
- Eastern Cooperative Oncology Group (ECOG) performance status of less than 2
- Lack of 6/6 or 5/6 matched related donor OR lack of 10/10 matched unrelated donor OR no available donor in the appropriate time frame to perform a potentially curative stem cell transplant
- Diffusing capacity of the lung for carbon monoxide (DLCO) greater than 50% of predicted value
Key exclusion criteria (8 listed)
- Heart disease, as determined by symptomatic congestive heart failure, radionuclide ventriculogram (RVG), or echocardiogram-determined LVEF of less than 50%, active angina pectoris, or uncontrolled high blood pressure
- Pulmonary disease, as determined by severe chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of less than 50% of predicted value
- Kidney disease, as determined by serum creatinine levels greater than 2.0 mg/dl
- Liver disease, as determined by serum bilirubin levels greater than 2.0 mg/dl (except in the case of Gilbert's syndrome or hemolytic anemia in which the bilirubin can be elevated greater than 2.0mg/dl), SGOT or SGPT greater than 3 times the upper limit of normal
- Neurologic disease, as determined by symptomatic leukoencephalopathy, active central nervous system (CNS) cancer, or other neuropsychiatric abnormalities that may prevent transplantation (previous CNS cancer and presently in CR is acceptable)
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 |
|---|---|---|
| Parathyroid Hormone (teriparatide)Also: Parathyroid hormone, PTH, teriparatideDay +1: PTH 40 mcg, Day +2: PTH 60 mcg, Day +3: PTH 80 mcg, Day +4 to Day +29 or until ANC>2000/microL: PTH 100 mcg | Drug | Parathyroid Hormone (teriparatide) |
Primary outcome
Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500)
Time frame: Statistic is calculated at Day 42 but ANC counts are measured daily up through discharge.
Median time to neutrophil engraftment (defined as an absolute neutrophil count [ANC] greater than 500)
7 secondary outcomes
Cumulative Incidence of Acute GVHD Grades II-IV at Day 100
Time frame: Measured at Day 100
Cumulative Incidence of Chronic GVHD
Time frame: Measured at 2 years
Platelet Engraftment (Greater Than 20,000)
Time frame: Measured at Day 180
100-day Transplant-related Mortality
Time frame: Measured at Day 100
Cumulative Incidence of Relapse
Time frame: Measured at 2 years
Overall Survival
Time frame: Measured at 2 years
Disease-free Survival
Time frame: Measured at 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 22766223Ballen K, Mendizabal AM, Cutler C, Politikos I, Jamieson K, Shpall EJ, Dey BR, Attar E, McAfee S, Delaney C, McCarthy P, Ball ED, Kamble R, Avigan D, Maziarz RT, Ho VT, Koreth J, Alyea E, Soiffer R, Wingard JR, Boussiotis V, Spitzer TR, Antin JH. Phase II trial of parathyroid hormone after double umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2012 Dec;18(12):1851-8. doi: 10.1016/j.bbmt.2012.06.016. Epub 2012 Jul 2.
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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.
Karen K. Ballen, MD principal investigator
Massachusetts General Hospital
Joseph Antin, MD principal investigator
Dana-Farber Cancer Institute
David Avigan, MD principal investigator
Beth Israel Deaconess Medical Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Parathyroid Hormone (teriparatide) in people with Leukemia, Myeloid, Chronic and Anemia, Aplastic, run by The Emmes Company, LLC at 4 sites 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 15 inclusion and 8 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: stopped early, as of the sponsor's last update on 29 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Dec 2012). 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?
Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500) (time frame: Statistic is calculated at Day 42 but ANC counts are measured daily up through discharge.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00393380, 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 29 Apr 2013.