LeukemiaPhase 2NCT00003336
Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions
A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Leukemia and Myelodysplastic Syndromes, run by Case Comprehensive Cancer Center at 1 site in United States.
Trial summary
RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells. PURPOSE: This phase II trial is studying how well umbilical cord blood transplantation works in treating patients with severe aplastic anemia, malignant thymoma, or… Read more about this study
- Who is it for?
- Ages Up to 55 years
- Conditions the study is for
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- What is being tested?
- anti-thymocyte globulin, busulfan and cyclophosphamide
- Type of treatment
- Surgery or procedure
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 1998to Feb 2006
- Phase
- Phase 2
- 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
DISEASE CHARACTERISTICS:
- Histologically confirmed diagnosis of severe aplastic anemia based on bone marrow cellularity of less than 20%
- Must meet at least two of the following criteria:
- Granulocyte count less than 500/mm^3
- Platelet count less than 20,000/mm^3
- Reticulocyte count less than 50,000/mm^3
- Following etiologies eligible:
- Fanconi's anemia
- Hypoplastic leukemia
- Monosomy 7
- Drug exposure (chloramphenicol, NSAIDS)
- Viral exposure (EBV, hepatitis, parvovirus, HIV)
- Nutritional deficiencies
- Thymoma
- Paroxysmal nocturnal hemoglobinuria
- Amegakaryocytic thrombocytopenia OR
- Histologically confirmed myelodysplastic syndrome (MDS) that is refractory to medical management or with cytogenic abnormalities predictive of transformation into acute leukemia, including 5q-, 7q-, monosomy 7, or trisomy 8
- The following etiologies only are eligible:
- Refractory anemia
- Refractory anemia with ringed sideroblasts
- De novo primary MDS
- Therapy-related secondary MDS OR
- Confirmed diagnosis of inherited hematopoietic disorder that is refractory to medical management
- Following etiologies eligible:
- Severe combined immunodeficiency
- Familial erythrophagocytic lymphohistiocytosis
- Wiskott-Aldrich syndrome
- Kostmann's syndrome (infantile histiocytosis)
- Chronic granulomatous disease
- Leukocytic adhesion deficiency
- Chediak-Higashi syndrome
- Paroxysmal nocturnal hemoglobinuria
- Fanconi's anemia
- Dyskeratosis congenita
- Diamond-Blackfan anemia
- Amegakaryocytic thrombocytopenia
- Osteopetrosis
- Gaucher's disease
- Lesch-Nyhan syndrome
- Mucopolysaccharidoses
- Lipodoses
- Autologous or haploidentical related peripheral blood stem cells available as backup
- Serologically matched umbilical cord blood unit available in the New York Blood Center's Placental Blood Project, or other acceptable umbilical cord blood registry
PATIENT CHARACTERISTICS:
Age:
- 55 and under
Performance status:
- Zubrod 0-1
- Karnofsky 80-100%
Life expectancy:
- At least 3 months
Hematopoietic:
- See Disease Characteristics
Hepatic:
- ALT/AST no greater than 4 times normal
- Bilirubin no greater than 2.0 mg/dL
Renal:
- Creatinine no greater than 2.0 mg/dL
- Creatinine clearance at least 50 mL/min
Cardiovascular:
- Normal cardiac function by echocardiogram or radionuclide scan
- Shortening fraction or ejection fraction at least 80% normal for age
- Non-Fanconi patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide
Pulmonary:
- FVC and FEV_1 at least 60% of predicted for age
- DLCO at least 60% of predicted in adult patients
Other:
- No active concurrent malignancy
- No active infection
- Not pregnant or nursing
- HIV negative
- Must have an available serologic matched Umbilical Cord Blood Unit (UCBU) in the New York Blood Center's Placental Blood Project, or other acceptable Umbilical Cord Blood (UCB) registry
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Not specified
Chemotherapy:
- No concurrent cytotoxic chemotherapy
Endocrine therapy:
- No concurrent immunosuppressive medications
Radiotherapy:
- No concurrent radiotherapy
Surgery:
- Not specified
Trial locations and contacts
1 site in 1 country.
United States1 site
Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center
Cleveland, Ohio, 44106-7284
About this study
Official title: A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders
RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: This phase II trial is studying how well umbilical cord blood transplantation works in treating patients with severe aplastic anemia, malignant thymoma, or myelodysplasia.
Read the detailed description
OBJECTIVES:
- Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders, refractory to medical management, who are undergoing high-dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation.
- Evaluate the rate and quality of immunologic reconstitution in this patient population.
OUTLINE: Patients are stratified according to weight (under 45 kg vs over 45 kg).
Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). The regimen varies according to the underlying cause of the anemia, but could include busulfan, cyclophosphamide or melphalan, anti-thymocyte globulin or methylprednisolone, and/or radiation therapy. One day after the conditioning regimen is completed, patients receive the UCBT.
Patients are followed weekly for 3 months, at 6 months, then every 6 months for 2.5 years, then annually thereafter.
PROJECTED ACCRUAL: A total of 4-90 patients will be accrued for this study within 5 years.
- Being tested
- anti-thymocyte globulin, busulfan, cyclophosphamide, melphalan, methylprednisolone
- Keywords
- refractory anemia, refractory anemia with ringed sideroblasts, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, atypical chronic myeloid leukemia, myelodysplastic/myeloproliferative disease, unclassifiable, childhood myelodysplastic syndromes
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Anemia, Refractory, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
The regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT).
busulfanDrug
The regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT).
cyclophosphamideDrug
The regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT).
melphalanDrug
The regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT).
The regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT).
bone marrow ablation with stem cell supportProcedure or surgery
umbilical cord blood transplantationProcedure or surgery
One day after the conditioning regimen is completed, patients receive the UCBT.
radiation therapyRadiation
The regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT).
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival by disease assessment
Measured: at 100 days and at 6, 9, 12, 18, and 24 months
Secondary outcomes (1)
Umbilical cord blood donor engraftment by chimerism and complete blood count (CBC) at time of myeloid recovery.
Measured: 100 days and at 6, 9, 12, 18, and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startJan 1998
- Primary completionDec 2005
- Study completionFeb 2006
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 LeukemiaStudies of the same condition, open and under way.
- More trials run by Case Comprehensive 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Case Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Mary J. Laughlin, MD, Principal investigator, Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003336
- Sponsor's protocol ID
- CWRU5Y97
- Other IDs
- P30CA043703, CASE-CWRU-5Y97, NCI-G98-1431, CASE-5Y97
- Status last verified
- Jun 2010
- First submitted
- 1 Nov 1999
- Last updated
- 11 Jun 2010
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Laughlin MJ, Barker J, Bambach B, Koc ON, Rizzieri DA, Wagner JE, Gerson SL, Lazarus HM, Cairo M, Stevens CE, Rubinstein P, Kurtzberg J. Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N Engl J Med. 2001 Jun 14;344(24):1815-22. doi: 10.1056/NEJM200106143442402.
PubMed 11407342
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders
- ClinicalTrials.gov ID
- NCT00003336
- Acronym / sponsor ID
- CWRU5Y97
- Keywords
- refractory anemia, refractory anemia with ringed sideroblasts, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, atypical chronic myeloid leukemia, myelodysplastic/myeloproliferative disease, unclassifiable, childhood myelodysplastic syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Case Comprehensive Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jan 1998
- Primary completion
- Dec 2005 (actual)
- Study completion
- Feb 2006 (actual)
- First posted
- 27 Jan 2003 (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.
RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: This phase II trial is studying how well umbilical cord blood transplantation works in treating patients with severe aplastic anemia, malignant thymoma, or myelodysplasia.
Detailed description
OBJECTIVES:
- Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders, refractory to medical management, who are undergoing high-dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation.
- Evaluate the rate and quality of immunologic reconstitution in this patient population.
OUTLINE: Patients are stratified according to weight (under 45 kg vs over 45 kg).
Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). The regimen varies according to the underlying cause of the anemia, but could include busulfan, cyclophosphamide or melphalan, anti-thymocyte globulin or methylprednisolone, and/or radiation therapy. One day after the conditioning regimen is completed, patients receive the UCBT.
Patients are followed weekly for 3 months, at 6 months, then every 6 months for 2.5 years, then annually thereafter.
PROJECTED ACCRUAL: A total of 4-90 patients will be accrued for this study within 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
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 |
|---|---|---|
| anti-thymocyte globulinThe regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). | Biological | – |
| busulfanThe regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). | Drug | – |
| cyclophosphamideThe regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). | Drug | – |
| melphalanThe regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). | Drug | – |
| methylprednisoloneThe regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). | Drug | – |
| bone marrow ablation with stem cell support | Procedure or surgery | – |
| umbilical cord blood transplantationOne day after the conditioning regimen is completed, patients receive the UCBT. | Procedure or surgery | – |
| radiation therapyThe regimen varies according to the underlying cause of the anemia.Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). | Radiation | – |
Primary outcome
Event-free survival by disease assessment
Time frame: at 100 days and at 6, 9, 12, 18, and 24 months
1 secondary outcome
Umbilical cord blood donor engraftment by chimerism and complete blood count (CBC) at time of myeloid recovery.
Time frame: 100 days and at 6, 9, 12, 18, and 24 months
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 11 Jun 2010.
- Background: PubMed 11407342Laughlin MJ, Barker J, Bambach B, Koc ON, Rizzieri DA, Wagner JE, Gerson SL, Lazarus HM, Cairo M, Stevens CE, Rubinstein P, Kurtzberg J. Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N Engl J Med. 2001 Jun 14;344(24):1815-22. doi: 10.1056/NEJM200106143442402.
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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.
Mary J. Laughlin, MD principal investigator
Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Leukemia and Myelodysplastic Syndromes, run by Case Comprehensive 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?
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 11 Jun 2010. 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 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?
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?
Event-free survival by disease assessment (time frame: at 100 days and at 6, 9, 12, 18, and 24 months). 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 NCT00003336, 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 Jun 2010.