Treosulfan-Based Conditioning Regimen Before A Blood Or Bone Marrow Transplant For The Treatment Of Bone Marrow Failure Diseases (BMT CTN 1904)

Study Overview

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.

OUTLINE: CONDITIONING REGIMEN: Patients receive treosulfan intravenously (IV) over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and lapine T-lymphocyte immune globulin (rATG) IV over 4-6 hours on days -4 to -2. TRANSPLANTATION: Patients undergo bone marrow or peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV continuously beginning on day -2 and a taper beginning on day 180. Patients may also receive tacrolimus orally (PO). Patients also receive methotrexate IV on days 1, 3, 6, and 11. Patients undergo echocardiography (ECHO) or multigated acquisition scan (MUGA) as well as possible chest radiography (x-ray) or computed tomography (CT) at baseline and undergo bone marrow biopsy and aspiration at baseline and follow up. Patients also undergo blood sample collection throughout the trial. After completion of study treatment, patients are followed up at 1 year from transplant.

Bone Marrow Failure Syndrome
Congenital Amegakaryocytic Thrombocytopenia
Diamond-Blackfan Anemia
Hereditary Sideroblastic Anemia
Paroxysmal Nocturnal Hemoglobinuria
Shwachman-Diamond Syndrome
Hematologic Neoplasm With Germline GATA2 Mutation
Hematologic Neoplasm With Germline SAMD9 Mutation
Hematologic Neoplasm With Germline SAMD9L Mutation

DRUG: Treosulfan
DRUG: Fludarabine Phosphate
DRUG: Tacrolimus
DRUG: Methotrexate
BIOLOGICAL: Lapine T-Lymphocyte Immune Globulin
PROCEDURE: Peripheral Blood Stem Cell Transplantation
PROCEDURE: Allogeneic Bone Marrow Transplantation
OTHER: Quality-of-Life Assessment
PROCEDURE: Echocardiography
PROCEDURE: Multigated Acquisition Scan
PROCEDURE: Bone Marrow Biopsy
PROCEDURE: Bone Marrow Aspiration
PROCEDURE: Biospecimen Collection
PROCEDURE: X-Ray Imaging
PROCEDURE: Computed Tomography

BMTCTN1904
2U10HL069294-11 (U.S. NIH Grant/Contract)
RG1121820 (OTHER Identifier) (OTHER: Fred Hutch/University of Washington Cancer Consortium)
10840 (OTHER Identifier) (OTHER: Fred Hutch/University of Washington Cancer Consortium)
NCI-2021-13862 (REGISTRY Identifier) (REGISTRY: CTRP (Clinical Trial Reporting Program))

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates	Results Reporting Dates	Study Record Updates
First Submitted 2021-06-21	Results First Submitted N/A	Last Update Submitted that met QC Criteria 2025-09-03
First Submitted that Met QC Criteria 2021-07-07	Results First Submitted that Met QC Criteria N/A	Last Update Posted (ESTIMATED) 2025-09-05
First Posted (ESTIMATED) 2021-07-16	Results First Posted N/A	Last Verified 2025-09

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

Primary Purpose:
Treatment

Allocation:
Na

Interventional Model:
Single Group

Masking:
None

Arms and Interventions

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Treatment (conditioning regimen; transplant; GVHD prophylaxis) CONDITIONING REGIMEN: Patients receive treosulfan IV over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and rATG IV over 4-6 hours on days -4 to -2. TRANSPLANTATION: Patients undergo bone marrow or peripheral bl	DRUG: Treosulfan Given IV DRUG: Fludarabine Phosphate Given IV DRUG: Tacrolimus Given IV and PO DRUG: Methotrexate Given IV BIOLOGICAL: Lapine T-Lymphocyte Immune Globulin Given IV PROCEDURE: Peripheral Blood Stem Cell Transplantation Undergo PBSC PROCEDURE: Allogeneic Bone Marrow Transplantation Undergo bone marrow transplant OTHER: Quality-of-Life Assessment Ancillary studies PROCEDURE: Echocardiography Undergo ECHO PROCEDURE: Multigated Acquisition Scan Undergo MUGA PROCEDURE: Bone Marrow Biopsy Undergo bone marrow biopsy and aspiration PROCEDURE: Bone Marrow Aspiration Undergo bone marrow biopsy and aspiration PROCEDURE: Biospecimen Collection Undergo blood sample collection PROCEDURE: X-Ray Imaging Undergo chest x-ray PROCEDURE: Computed Tomography Undergo chest CT

Participant Group/Arm

Intervention/Treatment

EXPERIMENTAL: Treatment (conditioning regimen; transplant; GVHD prophylaxis)

CONDITIONING REGIMEN: Patients receive treosulfan IV over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and rATG IV over 4-6 hours on days -4 to -2. TRANSPLANTATION: Patients undergo bone marrow or peripheral bl

DRUG: Treosulfan

Given IV

DRUG: Fludarabine Phosphate

Given IV

DRUG: Tacrolimus

Given IV and PO

DRUG: Methotrexate

Given IV

BIOLOGICAL: Lapine T-Lymphocyte Immune Globulin

Given IV

PROCEDURE: Peripheral Blood Stem Cell Transplantation

Undergo PBSC

PROCEDURE: Allogeneic Bone Marrow Transplantation

Undergo bone marrow transplant

OTHER: Quality-of-Life Assessment

Ancillary studies

PROCEDURE: Echocardiography

Undergo ECHO

PROCEDURE: Multigated Acquisition Scan

Undergo MUGA

PROCEDURE: Bone Marrow Biopsy

Undergo bone marrow biopsy and aspiration

PROCEDURE: Bone Marrow Aspiration

Undergo bone marrow biopsy and aspiration

PROCEDURE: Biospecimen Collection

Undergo blood sample collection

PROCEDURE: X-Ray Imaging

Undergo chest x-ray

PROCEDURE: Computed Tomography

Undergo chest CT

Primary Outcome Measures	Measure Description	Time Frame
Graft-Versus Host-Disease (GVHD)-Free Event-Free Survival (EFS)	The primary endpoint is the incidence of 1-year GVHD free, EFS (GEFS). An event is defined as death due to any cause, primary or secondary graft failure/rejection, or 2nd HCT whichever occurs first. Grade III-IV acute GVHD and chronic GVHD (using National Institutes of Health [NIH] consensus criteria) requiring systemic immune suppression will be considered in this estimate.	1 year post-HCT

Secondary Outcome Measures	Measure Description	Time Frame
Overall Survival	Overall survival at day 100 after HCT	Day 100 post-HCT
Overall Survival	Overall survival at 6 months after HCT	6 months post-HCT
Overall Survival	Overall survival at 1 year after HCT	1 year post-HCT
Event-Free Survival	Event-free survival will be estimated at 12 months after HCT. An event is defined as death due to any cause, primary or secondary graft failure/rejection, or 2nd HCT, whichever occurs first	1 year post-HCT
Hematologic Recovery: Neutrophil recovery	Hematologic recovery will be assessed according to neutrophil recovery after transplant. Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) ≥ to 500/mm3 for 3 consecutive measurements on 3 different days. The first of the three days will be designated the day of neutrophil recovery. The competing event is death without neutrophil recovery.	Assessed up to 1 year post-HCT
Hematologic Recovery: Platelet recovery	Hematologic recovery will be assessed according to platelet counts recovery after transplant. Platelet recovery is defined as the first day of a minimum of 3 days that the patient has a sustained platelet count ≥ 20,000/mm3 with no platelet transfusions in the preceding 7 days. The first day of sustained platelet count above these thresholds will be designated the day of platelet engraftment.	Day 100 post-HCT
Donor Chimerism (CD3 and Myeloid)	Proportions of patients with full (>95%), mixed (5-95%), or rejection (<5%) myeloid	Day 28 post-HCT
Donor Chimerism (CD3 and Myeloid)	Proportions of patients with full (>95%), mixed (5-95%), or rejection (<5%) myeloid	Day 100 post-HCT
Donor Chimerism (CD3 and Myeloid)	Proportions of patients with full (>95%), mixed (5-95%), or rejection (<5%) myeloid	1 year post-HCT
Primary graft failure/rejection	Defined as never achieving ANC ≥ 500/μL or never achieving ≥ 5% donor myeloid chimerism assessed by peripheral blood chimerism assays by day +42 post-HCT. Second infusion of hematopoietic cells is also considered indicative of primary graft failure by day +42 post-HCT	Day 42 post-HCT
Secondary graft failure/rejection post-HCT	Defined as < 5% donor myeloid chimerism in peripheral blood beyond day +42 post-HCT in patients with prior documentation of hematopoietic recovery with ≥ 5% donor cells by day +42 post-HCT. Second infusion of hematopoietic cells is also considered indicative of secondary graft failure.	Assessed up to 1 year post-HCT
Grade II-IV and grade III-IV GVHD at day 100	The cumulative incidences of acute grade II-IV and III-IV GVHD at day 100 after HCT will be determined.	Day 100 post-HCT
Grade II-IV and grade III-IV GVHD at day 180	The cumulative incidences of acute grade II-IV and III-IV GVHD at day 100 after HCT will be determined.	Day 180 post-HCT
Chronic GVHD	The cumulative incidence of chronic GVHD (using NIH consensus criteria) requiring systemic immune suppression at 1 year after HCT will be determined. Data will be collected directly from providers and chart review as defined by the NIH Consensus Conference Criteria	1 year post-HCT
Incidence of grade 3-5 toxicities	Grade 3-5 toxicities by day 30 after HCT	Day 30 post-HCT
Incidence of grade 3-5 toxicities	Grade 3-5 toxicities by day 100 after HCT	Day 100 post-HCT
Incidence of grade 2-3 systemic infections	All microbiologically documented infections or significant infections requiring antibiotic/antifungal therapy occurring up to 6 months after HCT	6 months post-HCT
Incidence of Epstein Barr virus (EBV) reactivation requiring therapy	The incidence of EBV reactivation requiring therapy in the first 180 days after HCT, and of EBV-associated lymphoproliferative disorder in the first 180 days after HCT will be evaluated.	Day 180 post-HCT
Incidence of EBV-associated lymphoproliferative disorder		Day 180 post-HCT
Incidence of cytomegalovirus (CMV) reactivation requiring therapy by day 180 post-HCT		Up to day 180 post-HCT

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person’s general health condition or prior treatments.

Ages Eligible for Study:
ALL

Sexes Eligible for Study:
1 Year

Accepts Healthy Volunteers:

Patient must be >= 1.0 year of age and less than 50.0 years of age at the time of enrollment (i.e. patient must have celebrated their 1st birthday when enrolled and must NOT have celebrated their 50th birthday when enrolled; 49.99 years)
Underlying BMFD treatable by allogenic HCT
Shwachman-Diamond syndrome

Criteria for Diagnosis:

A pathogenic mutation(s) for Shwachman-Diamond syndrome
For those patients tested but lacking a genetic mutation they must meet both **
criteria below:

Exocrine pancreatic dysfunction as defined by at least one of the following:

Pancreatic isoamylase below normal (age >= 3 years old), OR
Fecal elastase < 200, AND
Bone marrow failure as evidence by at least one of the following:

Intermittent or persistent neutropenia (absolute neutrophil count < 1,500/uL), OR
Hypo-productive anemia with a hemoglobin concentration below the age-related adjusted norms, OR
Unexplained macrocytosis, OR
Platelet count < 150,000/uL without alternative etiology, OR
Hypocellular bone marrow
Indications for HCT:

Severe neutropenia (absolute neutrophil count [ANC] < 500/uL), OR
Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 eligibility review committee (ERC). In addition, patients with severe or recurrent infections will be reviewed by the ERC if they do not meet the indications for transplant listed above
Diamond Blackfan Anemia

Criteria for Diagnosis:

A pathogenic mutation for Diamond Blackfan anemia
For those patients tested but lacking a genetic mutation the patient must meet the first **
criteria and at least one of the subsequent **
criteria listed below:

History of deficiency of erythroid precursors in an otherwise cellular bone marrow AND,
Reticulocytopenia, OR
Elevated adenosine deaminase activity, OR
Elevated hemoglobin F, OR
Macrocytosis, OR
Congenital anomalies
Indications for HCT:

Red blood cell (RBC) transfusion dependent anemia despite an adequate trial of steroids; OR
Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
Congenital Sideroblastic anemia

Criteria for Diagnosis:

A pathogenic mutation(s) for sideroblastic anemia
For those patients tested but lacking a genetic mutation:

Presence of ringed sideroblasts in the bone marrow excluding acquired causes of ringed sideroblasts such as lead poisoning & zinc toxicity
Indications for HCT:

Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia OR
Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
GATA2 mutation with associated marrow failure

Criteria for Diagnosis:

A pathogenic mutation(s) for GATA2
Indications for HCT:

Severe neutropenia (ANC < 500/uL), OR
Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC. In addition, patients with severe or recurrent infections will be reviewed by the ERC if they do not meet indications for transplant listed above
SAMD9 or SAMD9L disorders

Criteria for Diagnosis:

A pathogenic mutation(s) for SAMD9 or SAMD9L
Indications for HCT:

Severe neutropenia (ANC < 500/uL), OR
Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
Congenital amegakaryocytic thrombocytopenia

Criteria for Diagnosis:

A pathogenic mutation(s) for congenital amegakaryocytic thrombocytopenia.
For those patients tested but lacking a genetic mutation the patient must meet criteria below:

Thrombocytopenia early in life, AND
History of bone marrow demonstrating megakaryocyte hypoplasia
Indications for HCT:

Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
Neutropenia defined as an ANC < 500/uL, OR
Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
Paroxysmal nocturnal hemoglobinuria

Criteria for Diagnosis:

Paroxysmal nocturnal hemoglobinuria (PNH) clone size in granulocytes >= 10%, AND
Complement mediated intravascular hemolysis with an elevated LDH (above institutional upper limits of normal)
Indications for HCT:

PNH with thrombosis despite adequate medical management, OR
PNH with intravascular hemolysis requiring transfusion support despite adequate medical management, OR
Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC. In addition, patients with PNH and cytopenias may be considered for the protocol eligibility following review by protocol 1904 ERC
An undefined BMFD: a patient with a BMFD for whom a genetic mutation responsible for their bone marrow failure phenotype has not been identified (excluding PNH) will be eligible for this clinical trial following approval by Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 1904 ERC

A BMFD with a known genetic mutation but not listed above will be eligible for this clinical trial following approval by BMT CTN 1904 ERC
Patient and/or legal guardian must sign informed consent prior to initiation of conditioning for BMT CTN 1904
Note: The following patients MUST be reviewed by the BMT CTN 1904 ERC in order to determine if they are eligible for this trial:

All patients with Shwachman-Diamond syndrome, Diamond Blackfan anemia, congenital sideroblastic anemia, and congenital amegakaryocytic thrombocytopenia who have had genetic testing and a genetic mutation responsible for their disease was not identified
All patients with an undefined BMFD: a patient with a BMFD for whom a genetic mutation responsible for their bone marrow failure phenotype has not been identified, excluding PNH
All patients with a BMFD and a known genetic mutation that is not listed above
All patients with GATA2 mutation and associated marrow failure
All patients with SAMD9 or SAMD9L disorders
There may be circumstances where a treating physician will consider a transplant for a patient with a BMFD who does not meet all the criteria listed under "indications for HCT". In these situations, treating physicians may submit their patient to the BMT CTN 1904 ERC for review in order to determine if the patient is eligible for this clinical trial based on additional clinical or laboratory information
Many patients with BMFD can have bone marrow evaluations that raise concern for possible myelodysplastic syndrome (MDS) including but not limited to dysplastic bone marrow evaluations or cytogenetic abnormalities. However, in patients BMFD these findings are not necessarily diagnostic or consistent with MDS. Therefore, given the complexities of diagnosing MDS in patients with BMFD, all patients with bone marrow evaluations concerning for possible MDS should be submitted to the ERC for review to confirm or exclude MDS. This is particularly important as we do not want to exclude potentially eligible patients due to an incorrect diagnosis of MDS
HLA-MATCHED RELATED DONOR: HLA-matched sibling: Must be a minimum HLA-6/6 matched to the recipient at HLA-A, -B (serologic typing) and DRB1 (high-resolution typing)
HLA-MATCHED RELATED DONOR: HLA-matched related (phenotypic match): Fully matched for HLA-A, -B, -C, -DRB1, and DQB1 by high-resolution typing.
HLA-MATCHED RELATED DONOR: If a genetic mutation is known for the patient, the HLA-matched related donor [either HLA-matched sibling or HLA-matched related (phenotypic match)] must be screened for the same genetic mutation if clinically appropriate and should be confirmed to not have the same genetic disease (this does not include patients with PNH). Consult the protocol team with questions
HLA-MATCHED RELATED DONOR: If a patient has an undefined BMFD (a patient with a BMFD for whom a genetic mutation responsible for their bone marrow failure phenotype has not been identified), the HLA-matched related donor [either HLA-matched sibling or HLA-matched related (phenotypic match)] must have an evaluation as directed by the treating physician to confirm that the donor does not have the same underlying disease. This will include a complete blood count (CBC) with differential and potentially a bone marrow evaluation or other studies as directed by the treating physician
UNRELATED DONOR: Fully matched for HLA-A, -B, -C, -DRB1, and DQB1 by high-resolution typing OR
UNRELATED DONOR: Mismatched for a single HLA-class 1 allele (HLA-A, -B, or -C) by high-resolution typing; OR
UNRELATED DONOR: Mismatched for a single HLA DQB1 allele or antigen by high-resolution typing

Note: donor patient (DP) matching per institutional practice
DONOR SELECTION RECCOMENDATIONS: in the case where there are multiple donor options, donors should be selected based on the following priority numbered below:

Unaffected fully HLA-matched sibling
Unaffected fully phenotypically HLA-matched related donor
Fully HLA-matched unrelated donor
Unrelated donor with single allele or antigen level mismatch at DQB1
Unrelated donor with single allele level mismatch at class 1 (HLA-A, -B, or -C)

Patients with idiopathic aplastic anemia, Fanconi anemia, dyskeratosis congenita, and congenital neutropenia
Patients with MDS as defined by the World Health Organization (WHO) or leukemia
Prior allogeneic HCT
Patient's weight =< 10.0 kg (actual body weight and adjusted body weight) at time of study enrollment
Lansky (patients < 16 years of age) or Karnofsky (patients >= 16 years of age) performance < 70%
Left ventricular ejection fraction < 50% by echocardiogram or multi-gated acquisition (MUGA) scan

For patients unable to obtain a left ventricular ejection fraction, left ventricular shortening fraction of < 26%
Diffusing capacity of the lungs for carbon monoxide (DLCO) (corrected/adjusted for hemoglobin) < 50%, forced expiratory volume (FEV)1 < 50% predicted, and forced vital capacity (FVC) < 50% predicted
For patients unable to perform pulmonary function tests (PFTs) due to age or developmental delay: oxygen (O2) saturation < 92% on room air
On supplemental oxygen
Estimated creatinine clearance < 60 mL/minute/1.73m^2 (estimated per institutional practice)
Dialysis dependent
Conjugated bilirubin > 2 x upper limit of normal for age (ULN, unless attributable to Gilbert's syndrome)
Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 4 x ULN for age, or
Fulminant liver failure or cirrhosis
Iron overload - This exclusion criterion only applies to patients who are considered at risk for hepatic or cardiac iron overload. Therefore, not all patients enrolled on this protocol will undergo formal hepatic or cardiac iron assessment

For patients with a history of significant transfusions defined as >= 8 packed red blood cell transfusions per year for >= 1 year or have received >= 20 packed red blood cell transfusions (lifetime cumulative) will require formal hepatic and cardiac iron measurement. In addition, patients with a prior history of hepatic or cardiac iron overload will also require formal assessment for iron overload. Patients are excluded if:
Hepatic iron content >= 8 mg Fe/g dry weight by liver magnetic resonance imaging (MRI) using a validated methodology (such as T2
MRI or ferriscan) or liver biopsy per institutional practice
Cardiac iron content < 25 msec by cardiac T2
MRI
Uncontrolled bacterial infection within 1 week of study enrollment. Uncontrolled is defined as currently taking medication with no clinical improvement or progression on adequate medical treatment
Uncontrolled viral or fungal infection within 30 days of study enrollment. Uncontrolled is defined as currently taking medication with no clinical improvement or progression on adequate medical treatment
Positive for human immunodeficiency virus (HIV)
Presence of clinically significant anti-donor human leukocyte antigen (HLA)-antibodies per institutional practice
Prior solid organ transplant
Patients with prior malignancies except resected non-melanoma skin cancer or treated cervical carcinoma in situ
Females who are pregnant or breast-feeding
Females and males of childbearing potential who are unwilling to practice an effective method of contraception or agree to abstinence from the time of signing informed consent through 12 months post-transplant or off tacrolimus whichever is later
Known hypersensitivity to treosulfan or fludarabine
Known life-threatening reaction (i.e. anaphylaxis) to Thymoglobulin that would prohibit use for the patient as this study requires use of the Thymoglobulin preparation of anti-thymocyte globulin (ATG)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
National Heart, Lung, and Blood Institute (NHLBI)

Investigators

STUDY_CHAIR: Lauri Burroughs, MD, Fred Hutch/University of Washington Cancer Consortium

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