Sickle Cell DiseaseEarly Phase 1NCT00257543
SALT: Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease
A Early Phase 1 study testing Alternative donor bone marrow and cord blood in people with Sickle Cell Disease, run by Emory University at 1 site in United States.
Trial summary
We hope to gain valuable information about the safety, success of engraftment, and rates of complications using alternate donor transplantation for children with severe SCD. Crucial information will be also collected about late effects from alternate donor BMT sickle cell, providing valuable information to clinicians… Read more about this study
- Who is it for?
- Ages Up to 16 yearsHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Alternative donor bone marrow and cord blood
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2006to Aug 2012
- Phase
- Early 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 16 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Hemoglobin SS, hemoglobin SC, or hemoglobin S0 thalassemia
- Donor available: Partially (5/6) HLA-matched relative (PMRD), matched (6/6) unrelated marrow donor or umbilical cord (5/6 or 6/6) of appropriate size (see 6.3.2) , using high-resolution HLA typing. Donor must not be homozygous for HgbS and must meet standard donor eligibility criteria of the Blood and Marrow Transplant Program.
- Severe SCD, defined by one of the following (modified Walters criteria):
- oPrevious (6 months prior) central nervous system event lasting longer than 24 hours, plus objective imaging evidence of CNS vasculopathy, with or without residual neurologic findings oFrequent (3 per year for 2 years) painful vaso-occlusive episodes (defined as episode lasting 4 hours and requiring hospitalization or outpatient treatment with parenteral narcotics) oRecurrent (3 in lifetime) acute chest syndrome events which have necessitated exchange transfusion or chronic transfusion therapy. Must have failed a good-faith trial of hydroxyurea (failure defined as a reduction of less than 50% in the incidence of vaso-occlusive events over a period of at least 18 months) or have demonstrated an inability to take the drug due to side effects.
- oAny combination of 3 acute chest syndrome episodes and vaso-occlusive pain episodes (defined as above) yearly for 3 years. Must have failed a good-faith trial of hydroxyurea (failure defined as a reduction of less than 50% in the incidence of vaso-occlusive events over a period of at least 18 months) or have demonstrated an inability to take the drug due to side effects.
- oStage I or II sickle lung disease oRed-cell alloimmunization (2 antibodies) on chronic transfusion therapy
Who cannot join
- oSuitable HLA-identical relative donor is available oBiopsy proven chronic active hepatitis, portal fibrosis, or cirrhosis, or serologic evidence of active hepatitis.
- oSCD chronic lung disease stage III (see Appendix) oSevere renal dysfunction defined as <50% of predicted normal GFR for age. oSevere cardiac dysfunction defined as shortening fraction < 25%. oSevere residual neurologic impairment other than hemiplegia alone, defined as full-scale IQ 70, quadriplegia or paraplegia, inability to ambulate, inability to communicate without assistive device, or any impairment resulting in decline of Lansky performance score to <70%.
- oCNS event occurring within 6 months prior to transplant oKarnofsky or Lansky functional performance score < 70% (see Appendix) oConfirmed HIV seropositivity. oPatient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.
- oPatient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process.
- oHistory of lack of compliance with medical care that would jeopardize transplant course.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Altanta
Atlanta, Georgia, 30322
About this study
Official title: SALT - Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease
We hope to gain valuable information about the safety, success of engraftment, and rates of complications using alternate donor transplantation for children with severe SCD. Crucial information will be also collected about late effects from alternate donor BMT sickle cell, providing valuable information to clinicians and families making decisions among interventions for children with severe sickle cell disease. If successful, alternate donor transplantation in this setting could pave the way to offering curative treatment to many more patients with severe SCD.
Read the detailed description
Unfortunately, less than 1/4th of patients with severe SCD will have a matched sibling donor that can serve as a BMT donor. This research protocol proposes to study the safety and usefulness of "alternate donor transplant" (using donors other than matched siblings). We will offer this treatment to children with severe sickle cell disease that do not have a matched sibling BMT donor. Alternative donors can be family members who are slightly less than completely matched, unrelated volunteer donors who are completely matched, and donated banked umbilical cord blood that is completely or nearly completely matched.
Alternative donor transplant has been performed commonly in patients with cancer, and also provides curative therapy for several non-malignant diseases (severe immunodeficiency, marrow failure and metabolic storage diseases). Alternate donor transplants carry higher risks of complications, including graft-versus-host disease, infection, and graft failure. Therefore, we will be selective about which patients are invited to participate, limiting eligibility to those patients that have had a severe SCD related problem (rather than those who are doing well and are likely to have few SCD related problems), but excluding patients who have such severe organ damage that they are more likely to die during transplant, and limiting eligibility to a young age group. A multi-step review algorithm that includes internal, local and external expert review has been constructed to provide a thorough, safe and ethical accrual process. We will treat patients using drugs and methods commonly used in alternate donor transplant for other diseases such as leukemia, and incorporate lessons learned from our previous experience in BMT for sickle cell by modifying supportive care measures. Special attention will be given to evaluation of post-BMT effects in this population, as well as potential reasons for adverse effects (such as graft failure).
We think that Atlanta is a particularly good place to study this kind of transplant for several reasons. One reason is experience: our program has transplanted more children with SCD than any other single institution in North America, with excellent outcomes. Additionally, SCD patients in our area often have been treated on a special red cell transfusion program that limits the number of people donating the blood; we think this is likely to reduce the chance of graft failure.
- Conditions
- Sickle Cell Disease
- Being tested
- Alternative donor bone marrow and cord blood
- Keywords
- sickle cell disease, bone marrow transplant
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alternative donor bone marrow and cord bloodProcedure or surgery
bone marrow transplant - alternative donors for bone marrow and cord blood transplants
| Study group | Role | What they receive |
|---|---|---|
| single arm study | Experimental | this is a single arm study Receives: Procedure: Alternative donor bone marrow and cord blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the frequency of donor engraftment after alternate donor transplantation in children with severe sickle cell disease.
Measured: 1 year after completion of study accrual
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2005 (estimated)
- Study startJan 2006
- Primary completionAug 2012
- Study completionAug 2012
Why it stopped: lack of enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ann E. Haight, Emory University
- Investigators
- Ann Haight, M.D., Study chair, Children's Healthcare of Atlanta/Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00257543
- Sponsor's protocol ID
- 876-2003
- Status last verified
- Sep 2013
- First submitted
- 22 Nov 2005
- Last updated
- 17 Sep 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SALT - Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00257543
- Acronym / sponsor ID
- 876-2003
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease, bone marrow transplant
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: lack of enrollment
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Jan 2006
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 23 Nov 2005 (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.
We hope to gain valuable information about the safety, success of engraftment, and rates of complications using alternate donor transplantation for children with severe SCD. Crucial information will be also collected about late effects from alternate donor BMT sickle cell, providing valuable information to clinicians and families making decisions among interventions for children with severe sickle cell disease. If successful, alternate donor transplantation in this setting could pave the way to offering curative treatment to many more patients with severe SCD.
Detailed description
Unfortunately, less than 1/4th of patients with severe SCD will have a matched sibling donor that can serve as a BMT donor. This research protocol proposes to study the safety and usefulness of "alternate donor transplant" (using donors other than matched siblings). We will offer this treatment to children with severe sickle cell disease that do not have a matched sibling BMT donor. Alternative donors can be family members who are slightly less than completely matched, unrelated volunteer donors who are completely matched, and donated banked umbilical cord blood that is completely or nearly completely matched.
Alternative donor transplant has been performed commonly in patients with cancer, and also provides curative therapy for several non-malignant diseases (severe immunodeficiency, marrow failure and metabolic storage diseases). Alternate donor transplants carry higher risks of complications, including graft-versus-host disease, infection, and graft failure. Therefore, we will be selective about which patients are invited to participate, limiting eligibility to those patients that have had a severe SCD related problem (rather than those who are doing well and are likely to have few SCD related problems), but excluding patients who have such severe organ damage that they are more likely to die during transplant, and limiting eligibility to a young age group. A multi-step review algorithm that includes internal, local and external expert review has been constructed to provide a thorough, safe and ethical accrual process. We will treat patients using drugs and methods commonly used in alternate donor transplant for other diseases such as leukemia, and incorporate lessons learned from our previous experience in BMT for sickle cell by modifying supportive care measures. Special attention will be given to evaluation of post-BMT effects in this population, as well as potential reasons for adverse effects (such as graft failure).
We think that Atlanta is a particularly good place to study this kind of transplant for several reasons. One reason is experience: our program has transplanted more children with SCD than any other single institution in North America, with excellent outcomes. Additionally, SCD patients in our area often have been treated on a special red cell transfusion program that limits the number of people donating the blood; we think this is likely to reduce the chance of graft failure.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single arm study | Experimental | Procedure: Alternative donor bone marrow and cord bloodthis is a single arm study |
Who may qualify
- Ages
- Up to 16 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Hemoglobin SS, hemoglobin SC, or hemoglobin S0 thalassemia
- Donor available: Partially (5/6) HLA-matched relative (PMRD), matched (6/6) unrelated marrow donor or umbilical cord (5/6 or 6/6) of appropriate size (see 6.3.2) , using high-resolution HLA typing. Donor must not be homozygous for HgbS and must meet standard donor eligibility criteria of the Blood and Marrow Transplant Program.
- Severe SCD, defined by one of the following (modified Walters criteria):
- oPrevious (6 months prior) central nervous system event lasting longer than 24 hours, plus objective imaging evidence of CNS vasculopathy, with or without residual neurologic findings oFrequent (3 per year for 2 years) painful vaso-occlusive episodes (defined as episode lasting 4 hours and requiring hospitalization or outpatient treatment with parenteral narcotics) oRecurrent (3 in lifetime) acute chest syndrome events which have necessitated exchange transfusion or chronic transfusion therapy. Must have failed a good-faith trial of hydroxyurea (failure defined as a reduction of less than 50% in the incidence of vaso-occlusive events over a period of at least 18 months) or have demonstrated an inability to take the drug due to side effects.
- oAny combination of 3 acute chest syndrome episodes and vaso-occlusive pain episodes (defined as above) yearly for 3 years. Must have failed a good-faith trial of hydroxyurea (failure defined as a reduction of less than 50% in the incidence of vaso-occlusive events over a period of at least 18 months) or have demonstrated an inability to take the drug due to side effects.
Key exclusion criteria (5 listed)
- oSuitable HLA-identical relative donor is available oBiopsy proven chronic active hepatitis, portal fibrosis, or cirrhosis, or serologic evidence of active hepatitis.
- oSCD chronic lung disease stage III (see Appendix) oSevere renal dysfunction defined as <50% of predicted normal GFR for age. oSevere cardiac dysfunction defined as shortening fraction < 25%. oSevere residual neurologic impairment other than hemiplegia alone, defined as full-scale IQ 70, quadriplegia or paraplegia, inability to ambulate, inability to communicate without assistive device, or any impairment resulting in decline of Lansky performance score to <70%.
- oCNS event occurring within 6 months prior to transplant oKarnofsky or Lansky functional performance score < 70% (see Appendix) oConfirmed HIV seropositivity. oPatient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.
- oPatient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process.
- oHistory of lack of compliance with medical care that would jeopardize transplant course.
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 |
|---|---|---|
| Alternative donor bone marrow and cord bloodbone marrow transplant - alternative donors for bone marrow and cord blood transplants | Procedure or surgery | single arm study |
Primary outcome
To determine the frequency of donor engraftment after alternate donor transplantation in children with severe sickle cell disease.
Time frame: 1 year after completion of study accrual
Results and safety
The study was stopped early (reason given: lack of enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2013.
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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.
Ann Haight, M.D. study chair
Children's Healthcare of Atlanta/Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Alternative donor bone marrow and cord blood in people with Sickle Cell Disease, run by Emory University 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 6 inclusion and 5 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 17 Sep 2013. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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: lack of enrollment). 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.
What does the primary outcome measure?
To determine the frequency of donor engraftment after alternate donor transplantation in children with severe sickle cell disease. (time frame: 1 year after completion of study accrual). 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 17 Sep 2013.