Aplastic AnemiaPhase 3NCT00004474
Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia
A Phase 3 study testing Anti-thymocyte globulin, Cyclophosphamide and Bone marrow transplantation in people with Aplastic Anemia, run by Roswell Park Cancer Institute at 4 sites in United States.
Trial summary
OBJECTIVES: I. Compare outcome, including graft failure, graft versus host disease, and survival of HLA-identical sibling bone marrow transplants for aplastic anemia using cyclophosphamide with or without antithymocyte globulin as a conditioning regimen. Read more about this study
- Who is it for?
- Ages Up to 59 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Anti-thymocyte globulin, Cyclophosphamide and Bone marrow transplantation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 224 participants
- Where
- 4 sitesUnited States
- Study dates
- Sep 1998to Aug 2007
- Phase
- Phase 3
- 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 59 years
- All sexes
- No healthy volunteers
- Child, Adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Severe aplastic anemia with the following criteria:
Hypocellular bone marrow with cellularity less than 20%
At least 2 of the following hematologic abnormalities:
- Neutrophil count no greater than 500/mm3
- Platelet count no greater than 20,000/mm3
- Reticulocyte count no greater than 50,000/mm3
HLA-identical sibling donor available
No clonal cytogenetic abnormalities, paroxysmal nocturnal hemoglobinuria, or myelodysplastic syndrome within 3 months of diagnosis of aplastic anemia
No congenital or constitutional aplastic anemia or Fanconi anemia
--Patient Characteristics--
Hepatic: Bilirubin less than 3 times upper limit of normal (ULN)
Renal: Creatinine less than 2 times ULN
Cardiovascular: Normal cardiac function
Other:
- No uncontrolled infection
- No severe concurrent disease
- HIV negative
- Fertile patients must use effective contraception
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Roswell Park Cancer Institute
Buffalo, New York, 14263-0001
University of Texas - MD Anderson Cancer Center
Houston, Texas, 77030-4009
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Midwest Children's Cancer Center
Milwaukee, Wisconsin, 53226
About this study
OBJECTIVES:
- Compare outcome, including graft failure, graft versus host disease, and survival of HLA-identical sibling bone marrow transplants for aplastic anemia using cyclophosphamide with or without antithymocyte globulin as a conditioning regimen.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, multicenter study. Patients are randomized to receive cyclophosphamide IV over 60 minutes on days -5 to -2 with or without antithymocyte globulin IV over 4 hours.
All patients then receive bone marrow over 60-120 minutes on day 0, 36 hours after the last dose of cyclophosphamide.
Patients are followed at day 100, at 6 months, and at 1 year posttransplant.
- Conditions
- Aplastic Anemia
- Being tested
- Anti-thymocyte globulin, Cyclophosphamide, Bone marrow transplantation
- Keywords
- Hematologic Disorders, Rare Disease
- Medical subject headings
- Anemia, Aplastic, Hematologic Diseases, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-thymocyte globulinDrug
Given via IV over 4 hours
CyclophosphamideDrug
Given via IV over 60 minutes on Days -5 to -2
Bone marrow transplantationProcedure or surgery
Given on Day 0
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Participants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2 with antithymocyte globulin IV over 4 hours; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0. Receives: Drug: Anti-thymocyte globulin; Drug: Cyclophosphamide; Procedure: Bone marrow transplantation |
| 2 | Experimental | Participants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0. Receives: Drug: Cyclophosphamide; Procedure: Bone marrow transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Graft failure, graft versus host disease, and survival
Measured: Measured at Day 100, Month 6, and Year 1 post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startSep 1998
- Primary completionAug 2007
- Study completionAug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Enrolment
- 224 participants
Sponsor and investigators
- Lead sponsor
- Roswell Park Cancer Institute
- Sponsor type
- University, hospital or other
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004474
- Sponsor's protocol ID
- 199/14004
- Other IDs
- RPCI-RP-9804, NCI-G98-1491, IBMTR-1
- Status last verified
- Oct 2008
- First submitted
- 18 Oct 1999
- Last updated
- 12 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia
- ClinicalTrials.gov ID
- NCT00004474
- Acronym / sponsor ID
- 199/14004
- Conditions
- Aplastic Anemia
- Keywords
- Hematologic Disorders, Rare Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 224 participants
- Lead sponsor
- Roswell Park Cancer InstituteUniversity, hospital or other
- Study start
- Sep 1998
- Primary completion
- Aug 2007 (actual)
- Study completion
- Aug 2007 (actual)
- First posted
- 19 Oct 1999 (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.
OBJECTIVES:
- Compare outcome, including graft failure, graft versus host disease, and survival of HLA-identical sibling bone marrow transplants for aplastic anemia using cyclophosphamide with or without antithymocyte globulin as a conditioning regimen.
Detailed description
PROTOCOL OUTLINE: This is a randomized, multicenter study. Patients are randomized to receive cyclophosphamide IV over 60 minutes on days -5 to -2 with or without antithymocyte globulin IV over 4 hours.
All patients then receive bone marrow over 60-120 minutes on day 0, 36 hours after the last dose of cyclophosphamide.
Patients are followed at day 100, at 6 months, and at 1 year posttransplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Anti-thymocyte globulin, Drug: Cyclophosphamide, Procedure: Bone marrow transplantationParticipants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2 with antithymocyte globulin IV over 4 hours; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0. |
| 2 | Experimental | Drug: Cyclophosphamide, Procedure: Bone marrow transplantationParticipants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0. |
Who may qualify
- Ages
- Up to 59 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 globulinGiven via IV over 4 hours | Drug | 1 |
| CyclophosphamideGiven via IV over 60 minutes on Days -5 to -2 | Drug | 1, 2 |
| Bone marrow transplantationGiven on Day 0 | Procedure or surgery | 1, 2 |
Primary outcome
Graft failure, graft versus host disease, and survival
Time frame: Measured at Day 100, Month 6, and Year 1 post-transplant
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 12 Mar 2018.
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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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Anti-thymocyte globulin, Cyclophosphamide and Bone marrow transplantation in people with Aplastic Anemia, run by Roswell Park Cancer Institute 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?
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 12 Mar 2018. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Graft failure, graft versus host disease, and survival (time frame: Measured at Day 100, Month 6, and Year 1 post-transplant). 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 12 Mar 2018.