Hurler SyndromePhase 1NCT01917708
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
A Phase 1 study testing Abatacept in people with Hurler Syndrome and Fanconi Anemia, run by Emory University at 1 site in United States.
Trial summary
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological… Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia
- What is being tested?
- Abatacept
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2014to 19 Sep 2019
- Phase
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Must be between the ages of 0-21 years at the time of admission for transplant.
- Must have one of the following diseases:
- Glanzmann thrombasthenia
- Wiskott-Aldrich syndrome or other combined immune deficiency
- Chronic-granulomatous disease
- Severe congenital neutropenia (with resistance to granulocyte-colony stimulating factor (GCSF) or chronic requirement of GCSF doses ≥10 mcg/kg)
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- Leukocyte adhesion deficiency
- Shwachman-Diamond syndrome
- Diamond-Blackfan anemia ((transfusion dependent, including steroid failure or inability to wean steroids)
- Thalassemia major
- Fanconi anemia
- Hemophagocytic lymphohistiocytosis (inherited or acquired refractory to therapy or with recurrent episodes of hyperinflammation)
- Dyskeratosis-congenita
- Hurler Syndrome
- Chediak-Higashi syndrome
- Acquired (immune; non-inherited, non-congenital) severe aplastic anemia
- Sickle cell disease (SCD) (Hgb SS or S-Beta 0 thalassemia) will be eligible between ages 3 and 9.99 and with severe disease.
- Other inherited or congenital marrow failure syndromes complicated by severe aplastic anemia
- Other inherited or congenital red blood cell disorders requiring monthly chronic transfusion therapy.
- Congenital platelet disorders requiring frequent platelet transfusions (patient must have received at least 10 transfusions in the last 3 years).
- Other inherited or congenital granulocyte disorders resulting in at least three inpatient hospitalizations in the past three years for infection.
- Must have an unrelated adult donor (marrow or PBSC) who is at least a 7/8 match (A, B, C, DRB1; the mismatch can be at an allele or antigen level) or an unrelated cord blood unit that is matched at least seven of eight loci (A, B and C antigen level-DRB1 allele level) and provides a minimum pre-cryopreservation total nucleated cell (TNC) dose of 7.5 x 107 TNC/kg recipient weight. Mismatches at the DRB1 locus may be at an antigen or allele level.
Who cannot join
- Human leukocyte antigen (HLA) matched related donor.
- Severe combined immune deficiency.
- Bridging (portal to portal) fibrosis or cirrhosis of the liver.
- Pulmonary: diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin), forced expiratory volume (FEV1) or forced vital capacity (FVC) < 40% of predicted. In child unable to perform pulmonary function testing, a chronic need for supplemental oxygen will serve as the exclusionary criterion.
- Severe renal dysfunction defined as estimated glomerular filtration rate (GFR) of <60 ml/min/1.73m2.
- Severe cardiac dysfunction defined as shortening fraction < 25%.
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- Neurologic impairment other than hemiplegia, defined as full-scale intelligence quotient (IQ) less than or equal to 70, quadriplegia or paraplegia, inability to ambulate, or any impairment resulting in decline of Lansky performance score to < 70%.
- Clinical stroke within 6 months of anticipated transplant.
- Karnofsky or Lansky functional performance score < 50%
- HIV infection.
- Uncontrolled viral, bacterial, fungal or protozoal infection at the time of study enrollment.
- Patient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.
- Patient or patient's guardian(s) unable to understand the nature and risks inherent in the blood and marrow transplant process.
- History of non-compliance severe enough in the estimation of the treating team to preclude the patient from undergoing unrelated donor transplantation.
- Patient is pregnant or lactating
- Patients HLA antibody testing demonstrates an antibody directed against a disparate HLA molecule.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.
Read the detailed description
Allogeneic hematopoietic stem cell transplantation (HSCT) represents the only viable cure for children who suffer from a wide variety of rare, serious non-malignant diseases, such as Fanconi Anemia, Hurler syndrome, and hemophagocytic lymphohistiocytosis. A major obstacle to the success of HSCT is morbidity and mortality from graft versus host disease (GVHD), driven by donor T cells recognizing and reacting against disparate host antigens. This trial is being conducted as a step toward testing the long-term hypothesis that the costimulation blockade agent abatacept can be added to a standard post-transplant GVHD prophylaxis regimen, cyclosporine and mycophenolate mofetil, to improve disease-free survival after unrelated hematopoietic stem cell transplantation (HSCT) using reduced intensity conditioning for children with non-malignant diseases (NMD). This study will have the following Specific Aims:
Specific Aim #1: To conduct a multicenter pilot assessing the tolerability of abatacept (n=10). Patients will receive four doses (10 mg/kg IV on days -1, +5, +14 and +28), a schedule well tolerated by adolescents and adults with hematologic malignancies in a previous pilot. Abatacept will be combined with cyclosporine and mycophenolate mofetil.
Specific Aim #2: To examine the immunological effects of abatacept in this setting.
Three reduced intensity conditioning regimens that have been shown to be effective in achieving sustained engraftment in patients with non-malignant diseases are available for use, depending on the patient's disease:
- Patients with Fanconi anemia will receive fludarabine, low dose cyclophosphamide, and anti-thymocyte globulin.
- Patients with severe aplastic anemia will receive low dose total body irradiation, fludarabine, cyclophosphamide, and anti-thymocyte globulin.
- Patients with other NMD will receive either the low dose total body irradiation regimen or an alemtuzumab, fludarabine, thiotepa, and melphalan regimen.
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia
- Being tested
- Abatacept
- Keywords
- non malignant conditions, blood and marrow transplant
- Medical subject headings
- Mucopolysaccharidosis I, Fanconi Anemia, Thrombasthenia, Wiskott-Aldrich Syndrome, Granulomatous Disease, Chronic, Neutropenia, Severe Congenital, Autosomal Recessive 3, Leukocyte adhesion deficiency type 1, Shwachman-Diamond Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AbataceptDrug
Also called Orencia
All patients will receive 4 doses of abatacept in addition to standard GVHD prophylaxis with cyclosporine and mycophenolate mofetil.
| Study group | Role | What they receive |
|---|---|---|
| Abatacept | Experimental | 4 doses of abatacept 10 mg/kg/dose will be given on days -1, +5, +14, and +28. Receives: Drug: Abatacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tolerability of Abatacept
The primary endpoint for this trial will be tolerability, defined in terms of the success in administering all prescribed doses of abatacept.
Abatacept will be deemed to be poorly tolerated if any of the following conditions are met:
- More than one dose is withheld.
- Death from an infection that occurs within 30 days of receiving the last prescribed dose of abatacept, but that is not preceded by systemic immunosuppressive therapy for GVHD
- Post-transplant lymphoproliferative disorder (PTLD) that occurs within 100 days of receiving the last prescribed dose, but that is not preceded by systemic immunosuppressive therapy for GVHD.
If less than 4 patients (of at least 18 evaluable patients) tolerate abatacept poorly, abatacept will be deemed tolerable. If there are fewer than 18 evaluable patients, if 3 of the first 10 patients treated tolerate abatacept poorly, abatacept will be deemed tolerable.
Measured: 1 year post-transplant
Secondary outcomes (17)
Proportion of Participants Experiencing Regimen-related Toxicity (RRT)
Regimen-related toxicity is scored according to the Bearman scale. Major RRT, defined as grade 4 (causing death) in any organ system or grade 3 for pulmonary, cardiac, renal, oral mucosal, neurologic or hepatic, will be recorded.
Measured: Day 42 post-transplant
Days until Neutrophil Recovery
Neutrophil recovery is defined as the first of 3 consecutive days following the nadir that the absolute neutrophil count is at least 500/µl.
Measured: 1 year post-transplant
Days until Platelet Recovery
Platelet recovery is defined as the first day that the platelet count is at least 20 thousand/µl without a transfusion in the preceding 7 days.
Measured: 1 year post-transplant
Number of Participants with Non-engraftment
Non-engraftment is defined as lack of neutrophil recovery (defined as absolute neutrophil count (ANC )>0.5 *109/L for three consecutive days) by 28 days post-transplant or neutrophil recovery with lack of myeloid donor chimerism.
Measured: 1 year post-transplant
Number of Participants with Secondary Graft Failure
Secondary graft failure is defined by initial engraftment but subsequent development of an ANC <0.5*109/L for fourteen consecutive days.
Measured: 1 year post-transplant
Number of Participants with Graft Loss
Graft loss is defined by initial engraftment (assessed by neutrophil recovery and donor chimerism) with the subsequent loss of donor myeloid chimerism (regardless whether persistent neutropenia develops).
Measured: 1 year post-transplant
Number of Participants Experiencing Cytomegalovirus (CMV) Viremia
Cytomegalovirus (CMV) viremia is defined as positive blood antigen or polymerase chain reaction (PCR) test.
Measured: Up to Day 180
Number of Participants Experiencing CMV Invasive Disease
CMV invasive disease is defined in accordance with the Blood and Marrow Transplant Clinical Trials Network Manual of Procedures.
Measured: 1 year post-transplant
Number of Participants Experiencing Post-transplant Lymphoproliferative Disorder (PTLD)
Post-transplant lymphoproliferative disorder (PTLD) is defined in accordance with the Blood and Marrow Transplant Clinical Trials Network Manual of Procedures and the World Health Organization's Classification of Tumours of Haematopoietic and Lymphoid Tissues.
Measured: 1 year post-transplant
Number of Participants Experiencing Other Infections
Infections other than CMV viremia, CMV invasive disease, and PTLD is defined in accordance with the Blood and Marrow Transplant Clinical Trials Network Manual of Procedures.
Measured: 1 year post-transplant
Number of Participants Experiencing Immune Reconstitution
Immune reconstitution is assessed by the day 100 cluster of differentiation 4 (CD4+) T cell count and by the reaccumulation of natural killer (NK) cells, B cells, total T cells, and cluster of differentiation 8 (CD8+) T cells as assessed by multicolor flow cytometry.
Measured: 1 year post-transplant
Number of Participants Experiencing Acute Graft Versus Host Disease (GVHD)
Early onset (before day 100) and late onset (after day 100) acute GVHD is assessed according to the Blood and Marrow Transplant Clinical Trials Network Manual of Procedures using the NIH consensus criteria.
Measured: Up to 1 year post-transplant
Number of Participants Experiencing Chronic GVHD
Chronic GVHD, including overlap syndrome, is assessed according to the Blood and Marrow Transplant Clinical Trials Network Manual of Procedures using the NIH consensus criteria.
Measured: 2 years post-transplant
Immune Suppression-Free Survival Rate
Participant survival while off of immunosuppressive agents.
Measured: 1 year post-transplant
Immune Suppression-Free and Disease-Free Survival Rate
Participant disease-free survival while off of immunosuppressive agents.
Measured: 1 year post-transplant
Disease-free Survival Rate
Disease-free survival is defined as survival without recurrence of underlying disease.
Measured: 1 year post-transplant
Overall Survival Rate
Overall-survival is defined as survival with or without relapse of underlying disease
Measured: 1 year post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2013 (estimated)
- Study startJan 2014
- Primary completion19 Sep 2019
- Study completion19 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hurler SyndromeStudies 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
- Phase 1
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, John Horan, Emory University
- Investigators
- John T Horan, MD, Principal investigator, Children's Healthcare of Atlanta/Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01917708
- Sponsor's protocol ID
- IRB00069836
- Status last verified
- Dec 2019
- First submitted
- 24 Jul 2013
- Last updated
- 26 Dec 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases
- ClinicalTrials.gov ID
- NCT01917708
- Acronym / sponsor ID
- IRB00069836
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia
- Keywords
- non malignant conditions, blood and marrow transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Jan 2014
- Primary completion
- 19 Sep 2019 (actual)
- Study completion
- 19 Sep 2019 (actual)
- First posted
- 7 Aug 2013 (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.
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.
Detailed description
Allogeneic hematopoietic stem cell transplantation (HSCT) represents the only viable cure for children who suffer from a wide variety of rare, serious non-malignant diseases, such as Fanconi Anemia, Hurler syndrome, and hemophagocytic lymphohistiocytosis. A major obstacle to the success of HSCT is morbidity and mortality from graft versus host disease (GVHD), driven by donor T cells recognizing and reacting against disparate host antigens. This trial is being conducted as a step toward testing the long-term hypothesis that the costimulation blockade agent abatacept can be added to a standard post-transplant GVHD prophylaxis regimen, cyclosporine and mycophenolate mofetil, to improve disease-free survival after unrelated hematopoietic stem cell transplantation (HSCT) using reduced intensity conditioning for children with non-malignant diseases (NMD). This study will have the following Specific Aims:
Specific Aim #1: To conduct a multicenter pilot assessing the tolerability of abatacept (n=10). Patients will receive four doses (10 mg/kg IV on days -1, +5, +14 and +28), a schedule well tolerated by adolescents and adults with hematologic malignancies in a previous pilot. Abatacept will be combined with cyclosporine and mycophenolate mofetil.
Specific Aim #2: To examine the immunological effects of abatacept in this setting.
Three reduced intensity conditioning regimens that have been shown to be effective in achieving sustained engraftment in patients with non-malignant diseases are available for use, depending on the patient's disease:
- Patients with Fanconi anemia will receive fludarabine, low dose cyclophosphamide, and anti-thymocyte globulin.
- Patients with severe aplastic anemia will receive low dose total body irradiation, fludarabine, cyclophosphamide, and anti-thymocyte globulin.
- Patients with other NMD will receive either the low dose total body irradiation regimen or an alemtuzumab, fludarabine, thiotepa, and melphalan regimen.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Abatacept | Experimental | Drug: Abatacept4 doses of abatacept 10 mg/kg/dose will be given on days -1, +5, +14, and +28. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (22 listed)
- Must be between the ages of 0-21 years at the time of admission for transplant.
- Must have one of the following diseases:
- Must have an unrelated adult donor (marrow or PBSC) who is at least a 7/8 match (A, B, C, DRB1; the mismatch can be at an allele or antigen level) or an unrelated cord blood unit that is matched at least seven of eight loci (A, B and C antigen level-DRB1 allele level) and provides a minimum pre-cryopreservation total nucleated cell (TNC) dose of 7.5 x 107 TNC/kg recipient weight. Mismatches at the DRB1 locus may be at an antigen or allele level.
Key exclusion criteria (16 listed)
- Human leukocyte antigen (HLA) matched related donor.
- Severe combined immune deficiency.
- Bridging (portal to portal) fibrosis or cirrhosis of the liver.
- Pulmonary: diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin), forced expiratory volume (FEV1) or forced vital capacity (FVC) < 40% of predicted. In child unable to perform pulmonary function testing, a chronic need for supplemental oxygen will serve as the exclusionary criterion.
- Severe renal dysfunction defined as estimated glomerular filtration rate (GFR) of <60 ml/min/1.73m2.
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 |
|---|---|---|
| AbataceptAlso: OrenciaAll patients will receive 4 doses of abatacept in addition to standard GVHD prophylaxis with cyclosporine and mycophenolate mofetil. | Drug | Abatacept |
Primary outcome
Tolerability of Abatacept
Time frame: 1 year post-transplant
The primary endpoint for this trial will be tolerability, defined in terms of the success in administering all prescribed doses of abatacept. Abatacept will be deemed to be poorly tolerated if any of the following conditions are met: * More than one dose is withheld. * Death from an infection that occurs within 30 days of receiving the last prescribed dose of abatacept, but that is not preceded by systemic immunosuppressive therapy for GVHD * Post-transplant lymphoproliferative disorder (PTLD) that occurs within 100 days of receiving the last prescribed dose, but that is not preceded by systemic immunosuppressive therapy for GVHD. If less than 4 patients (of at least 18 evaluable patients) tolerate abatacept poorly, abatacept will be deemed tolerable. If there are fewer than 18 evaluable patients, if 3 of the first 10 patients treated tolerate abatacept poorly, abatacept will be deemed tolerable.
17 secondary outcomes
Proportion of Participants Experiencing Regimen-related Toxicity (RRT)
Time frame: Day 42 post-transplant
Days until Neutrophil Recovery
Time frame: 1 year post-transplant
Days until Platelet Recovery
Time frame: 1 year post-transplant
Number of Participants with Non-engraftment
Time frame: 1 year post-transplant
Number of Participants with Secondary Graft Failure
Time frame: 1 year post-transplant
Number of Participants with Graft Loss
Time frame: 1 year post-transplant
Number of Participants Experiencing Cytomegalovirus (CMV) Viremia
Time frame: Up to Day 180
Number of Participants Experiencing CMV Invasive Disease
Time frame: 1 year post-transplant
Number of Participants Experiencing Post-transplant Lymphoproliferative Disorder (PTLD)
Time frame: 1 year post-transplant
Number of Participants Experiencing Other Infections
Time frame: 1 year post-transplant
Number of Participants Experiencing Immune Reconstitution
Time frame: 1 year post-transplant
Number of Participants Experiencing Acute Graft Versus Host Disease (GVHD)
Time frame: Up to 1 year post-transplant
Number of Participants Experiencing Chronic GVHD
Time frame: 2 years post-transplant
Immune Suppression-Free Survival Rate
Time frame: 1 year post-transplant
Immune Suppression-Free and Disease-Free Survival Rate
Time frame: 1 year post-transplant
Disease-free Survival Rate
Time frame: 1 year post-transplant
Overall Survival Rate
Time frame: 1 year 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 26 Dec 2019.
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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.
John T Horan, MD principal investigator
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 Phase 1 study testing Abatacept in people with Hurler Syndrome and Fanconi Anemia, 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 22 inclusion and 16 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: finished, as of the sponsor's last update on 26 Dec 2019. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Tolerability of Abatacept (time frame: 1 year 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 26 Dec 2019.