Metadata Catalog
Natural History Data Resource

Working...

Study Detail

Study ID 1019
Name Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Unrelated Donor Reduced Intensity Bone Marrow Transplant for Children With Severe Sickle Cell Disease (0601)
Contact Email meapen@mcw.edu
Acronym BMT CTN - UD HCT for cSCD-0601
Period 2008 - 2016
Design Clinical Trial
Study PI Mary Horowitz
Institution Medical College of Wisconsin
URL https://biolincc.nhlbi.nih.gov/studies/bmt_0601/
CTGovID https://clinicaltrials.gov/ct2/show/NCT00745420
Funding Agency NHLBI
Grant Number 1U01HL069294-01,5U24CA076518-02
Overview The primary objective is to determine event-free survival (EFS) at 1 year after unrelated donor (URD) hematopoietic stem cell transplantation (HCT) using bone marrow (BM) in patients with sickle cell disease (SCD).
Focus Areas 1. Hematopoietic Stem Cell Transplantation
2. Bone Marrow Transplant with GVHD Prophylaxis Regimen
Outcomes 1. Percentage of Participants With Event-Free Survival (EFS) [ Time Frame: 2 years ]
2. Percentage of Participants With Overall Survival (OS) [ Time Frame: 2 years ]
3. Neutrophil and Platelet Recovery [ Time Frame: Up to 100 days ]
4. Graft Rejection [ Time Frame: 1 year ]
5. Percentage of Participants With Acute Graft-vs-Host-Disease (GVHD) [ Time Frame: 100 days ]
6. Percentage of Participants With Chronic GVHD [ Time Frame: 1 year post-transplant ]
7. Number of Participants With Chronic GVHD by Severity [ Time Frame: 1 year post-transplant ]
8. Percentage of Participants With Posterior Reversible Encephalopathy Syndrome (PRES) [ Time Frame: 1 year ]
9. Change From Baseline to Day 100 in Participant Reported Health-Related Quality of Life (HRQL) [ Time Frame: 100 days post-transplant ]
10. Change From Baseline to Day 100 in Parent Proxy Reported Health-Related Quality of Life (HRQL) [ Time Frame: 100 days post-transplant ]
11. Change From Baseline to Day 180 in Participant Reported Health-Related Quality of Life (HRQL) [ Time Frame: 180 days post-transplant ]
12. Change From Baseline to Day 180 in Parent Proxy Reported Health-Related Quality of Life (HRQL) [ Time Frame: 180 days post-transplant ]
13. Change From Baseline to 1 Year in Participant Reported Health-Related Quality of Life (HRQL) [ Time Frame: 1 year post-transplant ]
14. Change From Baseline to 1 Year in Parent Proxy Reported Health-Related Quality of Life (HRQL) [ Time Frame: 1 year post-transplant ]
Data Location BioLINCC, BioData Catalyst
Biospecimen Available N/A
Data Consent Data sharing via BioLINCC
Biospecimen Consent NA
Contact Consent NA
Genomic Data No
Age Range 4-19
No. Participants 30
Inclusion Criteria 1. SCD (genotype hemoglobin SS disease [Hb SS], genotype hemoglobin SC disease [HbSC],sickle ß°[Sß°] thalassemia, or sickle ß^+[Sß^+]thalassemia) with one or more of the following:1. Patients must have symptomatic SCD (genotype Hb SS, Hb SC, Sß° thalassemia or Sß+ thalassemia), AND have 1 or more of the following clinical complications:(i) Clinically significant neurologic event (stroke) or any neurologic deficit lasting more than 24 hours that is accompanied by an infarct on cerebral MRI; OR (ii) patients who have a Transcranial Doppler (TCD) velocity that exceeds 200 cm/sec by the non-imaging technique (or TCD measurement greater than 185 cm/sec by the imaging technique) measured at a minimum of 2 separate occasions one month or more apart; OR,2. Minimum of two episodes of acute chest syndrome in the 2 years before study entry, defined as new pulmonary alveolar consolidation involving at least one complete lung segment (associated with acute symptoms including fever, chest pain, tachypnea, wheezing, rales, or cough that is not attributed to asthma or bronchiolitis) despite adequate supportive care measures3. History of 3 or more severe pain events per year in the 2 years before study entry
2. Lansky/Karnofsky performance score greater than or equal to 40
3. Patients must have an unrelated adult bone marrow donor who is Human Leukocyte Antigen (HLA)-matched at 8 of 8 HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing.
4. Patients with adequate physical function: a)Cardiac: Left ventricular ejection fraction (LVEF) greater than 40%, or LV shortening fraction greater than 26%; b) Pulmonary: Pulse oxymetry with a baseline O2 saturation of greater than or equal to 85% is required for all patients, Carbon Monoxide Diffusing Capacity (DLCO) greater than 40% (corrected for hemoglobin) for patients in whom pulmonary function testing can be performed; c) Renal: Serum creatinine less than or equal to 1.5 x upper limit of normal for age and glomerular filtration rate (GFR) greater than 100 mL/min/1.73 m. For patients older than or equal to 16 years of age, GFR should be greater than 70 mL/min/1.73 m^2; d) Hepatic: Serum conjugated (direct) bilirubin less than 2x upper limit of normal for age as per local laboratory; alanine transaminase (ALT) and aspartate transaminase (AST) less than 5 times upper limit of normal as per local laboratory.
5. If the patient has been receiving chronic transfusion therapy for more than or equal to 1 year AND has clinical evidence of iron overload (serum ferritin level of greater than 1000 ng/ml), a liver biopsy shall be obtained within 90 days of starting conditioning therapy (alemtuzumab). Histologic exam of the liver must document absence of bridging fibrosis or cirrhosis of the liver. In other cases, a liver biopsy is optional.
6. Hemoglobin S (Hb S) level less than or equal to 45%, seven days prior to initiation of alemtuzumab
Exclusion Criteria 1. Evidence of uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms) within 1 month prior to starting the conditioning regimen. Patients with fever or suspected minor infection should await resolution of symptoms before starting the conditioning regimen
2. Pregnant or breastfeeding
3. Patients with 8/8 HLA-matched family donors able to donate
4. Seropositivity for HIV
5. Prior allogeneic marrow or stem cell transplant
6. Iron chelation must be discontinued more than or equal to 48 hours before initiating the conditioning regimen
7. Hydroxyurea (if receiving this therapy) must be discontinued more than or equal to 48 hours before initiating the conditioning regimen
Study Documentation
  Select All
   1. 0601 Sickle Cell Protocol v11
   2. BMT_0601_2021a
Forms
  Select All
   1. BMT CTN 0601 CRFs Segment A
   2. BMT CTN 0601 CRFs Segment B

Data ElementVariables
Achenbach Child Behavior Checklist (CBCL)
Bayley Scale of Infant and Toddler Development
Behavior Rating Inventory of Executive Function (BRIEF) NCBRBRPC
NCBRBRRS
NCBRBRTS
NCBRECPC
NCBRECRS
NCBRECTS
NCBREMPC
NCBREMRS
NCBREMTS
NCBRFLPC
NCBRFLRS
NCBRFLTS
NCBRGCPC
NCBRGCRS
NCBRGCTS
NCBRICPC
NCBRICRS
NCBRICTS
NCBRINPC
NCBRINRS
NCBRINTS
NCBRISPC
NCBRISRS
NCBRISTS
NCBRITPC
NCBRITRS
NCBRITTS
NCBRMCPC
NCBRMCRS
NCBRMCTS
NCBRMNPC
NCBRMNRS
NCBRMNTS
NCBRNGPC
NCBRNGRS
NCBRNGTS
NCBROMPC
NCBROMRS
NCBROMTS
NCBRPOPC
NCBRPORS
NCBRPOTS
NCBRSHPC
NCBRSHRS
NCBRSHTS
NCBRWMPC
NCBRWMRS
NCBRWMTS
California Verbal Learning Test NCCVDCPC
NCCVDCSS
NCCVDCTS
NCCVLDPC
NCCVLDSS
NCCVLDTS
NCCVSCPC
NCCVSCSS
NCCVSCTS
NCCVSDPC
NCCVSDSS
NCCVSDTS
NCCVTTPC
NCCVTTSS
NCCVTTTS
Children's Memory Scale NCCMACRS
NCCMACSS
NCCMDDRS
NCCMDDSS
NCCMDLRS
NCCMDLSS
NCCMDRRS
NCCMDRSS
NCCMDTRS
NCCMDTSS
NCCMFDRS
NCCMFDSS
NCCMFIRS
NCCMFISS
NCCMGMRS
NCCMGMSS
NCCMLNRS
NCCMLNSS
NCCMNTRS
NCCMNTSS
NCCMSDRS
NCCMSDSS
NCCMSIRS
NCCMSISS
NCCMSQRS
NCCMSQSS
NCCMSRRS
NCCMSRSS
NCCMVBDR
NCCMVBDS
NCCMVBIR
NCCMVBIS
NCCMVSDR
NCCMVSDS
NCCMVSIR
NCCMVSIS
NCCMWDRS
NCCMWDSS
NCCMWLRS
NCCMWLSS
NCCMWRRS
NCCMWRSS
NCCMWTRS
NCCMWTSS
Connor's Continuous Performance Test II NCCPCMPC
NCCPCMTS
NCCPCMVA
NCCPHRPC
NCCPHRTS
NCCPHRVA
NCCPHSPC
NCCPHSTS
NCCPHSVA
NCCPOMPC
NCCPOMTS
NCCPOMVA
Delis-Kaplan Executive Function System (D-KEFS)
NeuroQOL Cognitive Function
Peabody Picture Vocabulary Test (PPVT)
Data ElementVariables
Adaptive Behavior Assessment System-II NCABCMRS
NCABCMSS
NCABCPTS
NCABCURS
NCABCUSS
NCABFARS
NCABFASS
NCABFPRS
NCABFPSS
NCABGACS
NCABHLRS
NCABHLSS
NCABHSRS
NCABHSSS
NCABLSRS
NCABLSSS
NCABMRRS
NCABMRSS
NCABPRCS
NCABSCRS
NCABSCSS
NCABSDRS
NCABSDSS
NCABSLRS
NCABSLSS
NCABSOCS
NCABWKRS
NCABWKSS
Adverse Childhood Experience Questionnaire (ACE-Q)
Beery Developmental Test of Visual-Motor Integration NCVMIPC
NCVMIRS
NCVMISS
Benton Tactile Form Perception Test
Mini Mental Status Examination (MMSE)
Oral Verbal Fluency Test
PROMIS Cognitive Function
Purdue Pegboard (SPA version)
Test of Everyday Attention (TEA)
Vineland Adaptive Behavior Summary
Wechsler Intelligence Scales NCWABDRS
NCWABDTS
NCWAFIQS
NCWAMRRS
NCWAMRTS
NCWAPIQS
NCWASMRS
NCWASMTS
NCWAVIQS
NCWAVORS
NCWAVOTS
NCWPBDRS
NCWPBDSC
NCWPCDRS
NCWPCDSC
NCWPFIQS
NCWPINRS
NCWPINSC
NCWPMRRS
NCWPMRSC
NCWPOARS
NCWPOASC
NCWPPCRS
NCWPPCSC
NCWPPIQS
NCWPPNRS
NCWPPNSC
NCWPRVRS
NCWPRVSC
NCWPVIQS
NCWPVORS
NCWPVOSC
NCWPWRRS
NCWPWRSC
Wechsler Memory Scale
Wisconsin Card Sorting Test
Woodcock-Johnson Revised, Tests of Achievement
Woodcock-Johnson, Revised, Tests of Cognitive Ability
Data ElementVariables
ASCQ-Me SCD Medical History Checklist
ASCQ-Me Sexual Health
Child Health Questionnaire (CHQ) ABILITPA
ACTIVIYF
ALONGYF
ARGUEBEH
ATHLETSE
ATLIMIYF
ATTNBEH
ATWORRYF
BEHSWDO
BEHSWKIN
BEHSWTM
BENDPA
BODPAIN
BOTHERWB
CANCLYF
CAREPA
CATCHCH
CHEERWB
COMPARCH
CRYINGWB
DISAGRYF
FAMILYSE
FRIENDSE
GAACTYNG
GAAGEBEH
GAALONE
GAALONGO
GAARGUED
GACLUMSY
GAGOTMAD
GAHARDOT
GALIECHT
GAMEAN
GANOTDO
GAOTHRLK
GAPAYATN
GARANAWA
GASPEECH
GASTOLEH
GASTOLEO
GBHEALTH
GENHLTGH
GWAFRAID
GWBOTHER
GWCHEER
GWCRY
GWENJOY
GWFUN
GWHAPPY
GWHEADAC
GWJITTER
GWLIKEYO
GWLONELY
GWNERVOU
GWSAD
GWSLEEP
GWUNHAPP
GWWORRY
INTRPTYF
KIDSBEH
KINDBEEA
KINDHLEA
LESSHLCH
LIEBEH
LIFECH
LIFESE
LONELYWB
LOOKSSE
LOTENEPA
NERVOUWB
NVRILLCH
OFBDPAIN
PABEND
PAEATDRS
PAGETARO
PAHOUSE
PAINBOD
PAINOFBD
PAINOUT
PALOTENE
PASOMENE
PAWALKBL
PAWALKSE
PERFBEEA
PHLIMIYF
PHWORRYF
PHYSWDO
PHYSWKIN
PHYSWTM
PICKGOYF
SADSWKDO
SADSWKIN
SADSWKTM
SCHOOLSE
SEALONG
SEBODY
SECANDO
SEFAMILY
SEFEEL
SEFNDSHP
SEFRIEND
SEHEALTH
SELIFE
SEOTFEEL
SESCHLWK
SESPORTS
SETALK
SEYOURSE
SOMENEPA
STOLEBEH
TANTRBEH
TENSNYF
TIMBEEA
TIMHLEA
WALKPA
WBLIMIYF
WBWORRYF
WORRYCH
YFALONG
YFCANCEL
YFDISAGR
YFINTACT
YFLIMACT
YFPICKGO
YFTENSIO
YHALSICK
YHHLTHEX
YHHLTHNW
YHLESOLD
YHLESSHL
YHNESICK
YHNEWORR
YHNOSICK
YHONEYR
YHSOSICK
YHVEROLD
YHWORRY
Ladder of Life
Pediatric Quality of Life Inventory Child Report (ages 13-18)
Pediatric Quality of Life Inventory Child Report (ages 8-12)
Pediatric Quality of Life Inventory Parent Report for Child (ages 13-18)
Pediatric Quality of Life Inventory Parent Report for Child (ages 8-12) CAREPA
PROMIS Global Health
PROMIS Global Physical Health
PROMIS Self-Efficacy for Managing Medications and Treatment
SF-36 Health Survey
Back to Study List