Sickle Cell AnemiaPhase 2NCT00040469
Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies
A Phase 2 study testing Campath -1H, Dilantin and Busulfan in people with Sickle Cell Anemia and Hemoglobinopathy, run by Baylor College of Medicine at 2 sites in United States.
Trial summary
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years. Read more about this study
- Who is it for?
- Ages 1 day to 64 years
- Conditions the study is for
- Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
- What is being tested?
- Campath -1H, Dilantin and Busulfan
- How many people
- 15 participants
- Where
- 2 sitesUnited States
- Study dates
- Aug 2000to 21 Nov 2003
- 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 1 day to 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with homozygous B0/+ thalassemia or severe variants of B0/+ thalassemia with an HLA genotypically identical donor.
- Patients with an HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb 0/+ and at least one of the following:
- Previous central nervous system vaso-occlusive episode with or without residual neurologic findings; Frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy; Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.
- Severe anemia which prevents acceptable quality of life and necessitates chronic transfusion therapy.
- The patient must have an HLA genotype identical donor.
- Between the ages of birth and 65 years.
- Women of childbearing potential must have a negative pregnancy test.
Who cannot join
- Biopsy proven chronic active hepatitis or fibrosis with portal bridging.
- SCD chronic lung disease >/= stage 3.
- Severe renal dysfunction defined as creatinine clearance <40 ml/min/1.73 M2
- Severe cardiac dysfunction defined as shortening fraction <25%.
- HIV infection.
- Severe but unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplant (BMT).
- Inadequate intellectual capacity to understand the nature and risk inherent in the BMT process and give informed consent (in the case of minors, this criteria must be fulfilled by the legal guardian).
- Pregnant, lactating or unwilling to use appropriate birth control.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Texas Children's Hospital
Houston, Texas, 77030
The Methodist Hospital
Houston, Texas, 77030
About this study
Official title: Allogeneic Bone Marrow Transplant From HLA Identical Related Donors for Patients With High Risk Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, Hemoglobin SB0/+ Thalassemia, or Homozygous B0/+ Thalassemia or Severe Variants of B0/+ Thalassemia
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.
Read the detailed description
To do the bone marrow transplant, we must first kill the cells in the bone marrow that make the abnormal red blood cells that are found in patients with severe thalassemia or sickle cell disease.
We will do this by using three drugs: busulfan, cyclophosphamide, and CAMPATH-1H. CAMPATH-IH is an investigational drug. CAMPATH-1H is used to prevent participants from rejecting or refusing to let the donor blood cells grow in the body. After the drug treatment, participants will be given bone marrow from a brother or sister who has healthy bone marrow that matches.
- Conditions
- Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
- Being tested
- Campath -1H, Dilantin, Busulfan, Cyclophosphamide
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobinopathies, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Campath -1HDrug
DilantinDrug
BusulfanDrug
CyclophosphamideDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2002 (estimated)
- Study startAug 2000
- Primary completion21 Nov 2003
- Study completion21 Nov 2003
Why it stopped: accrual was slow and sporadic so the study was closed
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, Robert Krance, Baylor College of Medicine
- Investigators
- Malcolm K. Brenner, MD, Study chair, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00040469
- Sponsor's protocol ID
- H6847
- Other IDs
- Scallo2
- Status last verified
- Jan 2020
- First submitted
- 26 Jun 2002
- Last updated
- 18 Jan 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Bone Marrow Transplant From HLA Identical Related Donors for Patients With High Risk Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, Hemoglobin SB0/+ Thalassemia, or Homozygous B0/+ Thalassemia or Severe Variants of B0/+ Thalassemia
- ClinicalTrials.gov ID
- NCT00040469
- Acronym / sponsor ID
- H6847
- Conditions
- Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: accrual was slow and sporadic so the study was closed
- Enrolment
- 15 participants
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Aug 2000
- Primary completion
- 21 Nov 2003 (actual)
- Study completion
- 21 Nov 2003 (actual)
- First posted
- 28 Jun 2002 (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 major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.
Detailed description
To do the bone marrow transplant, we must first kill the cells in the bone marrow that make the abnormal red blood cells that are found in patients with severe thalassemia or sickle cell disease.
We will do this by using three drugs: busulfan, cyclophosphamide, and CAMPATH-1H. CAMPATH-IH is an investigational drug. CAMPATH-1H is used to prevent participants from rejecting or refusing to let the donor blood cells grow in the body. After the drug treatment, participants will be given bone marrow from a brother or sister who has healthy bone marrow that matches.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 1 day to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Patients with homozygous B0/+ thalassemia or severe variants of B0/+ thalassemia with an HLA genotypically identical donor.
- Patients with an HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb 0/+ and at least one of the following:
- Previous central nervous system vaso-occlusive episode with or without residual neurologic findings; Frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy; Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.
- Severe anemia which prevents acceptable quality of life and necessitates chronic transfusion therapy.
- The patient must have an HLA genotype identical donor.
Key exclusion criteria (8 listed)
- Biopsy proven chronic active hepatitis or fibrosis with portal bridging.
- SCD chronic lung disease >/= stage 3.
- Severe renal dysfunction defined as creatinine clearance <40 ml/min/1.73 M2
- Severe cardiac dysfunction defined as shortening fraction <25%.
- HIV infection.
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 |
|---|---|---|
| Campath -1H | Drug | – |
| Dilantin | Drug | – |
| Busulfan | Drug | – |
| Cyclophosphamide | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early (reason given: accrual was slow and sporadic so the study was closed). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2020.
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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.
Malcolm K. Brenner, MD study chair
Baylor College of Medicine
2 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 Campath -1H, Dilantin and Busulfan in people with Sickle Cell Anemia and Hemoglobinopathy, run by Baylor College of Medicine at 2 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 7 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 18 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
The study was stopped early (reason given: accrual was slow and sporadic so the study was closed). Results are not available in the source record.
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.
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 18 Jan 2020.