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Stem Cell Transplantation for Sickle Cell Anemia

NCT01877837 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2022-11-07

Study results available
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Summary

This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of valine for glutamic acid at the sixth position in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to vaso-occlusion, which causes irreversible damage in almost all systems in the body, including the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys.

Conditions

Interventions

DRUG

Alemtuzumab

Adjusted Ideal Body Weight Formula: AIBW = IBW + \[(0.4) x (ABW - IBW)\] b) Medications i.) Alemtuzumab I. Hb S% must be \< or = 45% within 7 days prior to initiation of Alemtuzumab II. Iron chelation and hydroxyurea must be discontinued \>48 hours before initiating therapy III. Alemtuzumab will be diluted in 100mL of 0.9% NS and infused at a rate as below

DRUG

Fludarabine

I. Fludarabine should be diluted in 100 ml 0.9%NS and given over 30 minutes. II. A daily dose of an antiemetic should be given 30 minutes prior to administration of the Fludarabine

DRUG

Melphalan

I. Melphalan should be diluted in 0.9%NS to a concentration of 0.1 -0.45 mg/mL and given over 45 minutes. \*Entire dose must be infused within 60 minutes of reconstitution in Pharmacy. II. A daily dose of an antiemetic should be given 30 minutes prior to administration of the Melphalan III. Patients should be encouraged to suck on a popsicle or something similar during the Melphalan infusion.

PROCEDURE

Stem Cells

Infusion of Hematopoietic Stem Cells

Sponsors & Collaborators

  • Hackensack Meridian Health

    lead OTHER

Principal Investigators

  • Jennifer Krajewski, MD · Hackensack Meridian Health

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-06-30
Primary Completion
2021-09-30
Completion
2021-09-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01877837 on ClinicalTrials.gov