NCT00040417

Brief Summary

The major goal of this study is to determine the risks and benefits of stem cell transplants in combination with a newer, less toxic conditioning chemotherapy treatment in patients with severe sickle cell disease (SCD) or sickle hemoglobin variants (hemoglobin SC or hemoglobin SB0/+), or homozygous b0/+ thalassemia or severe B0/+ thalassemia variants. Participation in this project will be for one year, with follow up evaluations done every 6 months thereafter for 10 years or until participants are 18 years old.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2000

Typical duration for phase_2

Geographic Reach
1 country

2 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2000

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

June 26, 2002

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 27, 2002

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 21, 2003

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 21, 2003

Completed
Last Updated

January 18, 2020

Status Verified

January 1, 2020

Enrollment Period

3.3 years

First QC Date

June 26, 2002

Last Update Submit

January 15, 2020

Conditions

Sickle Cell AnemiaHemoglobinopathyThalassemia

Interventions

FLUDARABINEDRUG
CAMPATH-IHDRUG
Total Body IrradiationPROCEDURE
FK506DRUG
G-SCF (Granulocyte-colony stimulating factor)DRUG

Eligibility Criteria

Age1 Day - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with a haploidentical related HLA donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ thalassemia and at least one of the following conditions:
  • 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.
  • Patients with a haploidentical related HLA donor and homozygous b0/+ thalassemia or severe variants of b0/+ thalassemia and require chronic transfusion therapy.
  • Women of childbearing potential must have a negative pregnancy test.
  • Between the ages of birth and 65 years.

You may not qualify if:

  • HLA identical or 5/6 HLA matched sibling donor
  • Biopsy proven chronic active hepatitis or portal fibrosis.
  • 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.
  • Unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate Stem Cell Transplant.
  • Patient or guardian(s) unable to understand the nature and risks inherent in the stem cell transplant process.
  • Pregnant or lactating females and those unwilling to use acceptable contraception.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

The Methodist Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Anemia, Sickle CellHemoglobinopathiesThalassemia

Interventions

fludarabineWhole-Body IrradiationTacrolimusGranulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

RadiotherapyTherapeuticsInvestigative TechniquesMacrolidesLactonesOrganic ChemicalsColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Officials

  • Malcolm K. Brenner, MD, FRCP

    Baylor College of Medicine

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

June 26, 2002

First Posted

June 27, 2002

Study Start

August 1, 2000

Primary Completion

November 21, 2003

Study Completion

November 21, 2003

Last Updated

January 18, 2020

Record last verified: 2020-01

Locations

US(2)