Status:
WITHDRAWN
Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor
Lead Sponsor:
St. Jude Children's Research Hospital
Collaborating Sponsors:
Doris Duke Charitable Foundation
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
10-25 years
Phase:
PHASE2
Brief Summary
With recent advances in gene editing, gene therapy is becoming a viable curative treatment option for sickle cell disease. In order to do genetic manipulation, investigators need to collect hematopoie...
Detailed Description
Participants will be enrolled sequentially, and no two participants will undergo drug administration, mobilization or apheresis at the same time. A subsequent participant can only receive the study dr...
Eligibility Criteria
Inclusion
- Patients with severe SCD who are 10-25 years old and are willing to donate autologous HSCs for advancing future gene therapy for SCD. Parents/legal guardians of participants must be able and willing to consent for their participation in this study. Severe SCD, for the purpose of this study, will be defined as patients who are receiving chronic transfusion therapy due to SCD related complications. The need for undergoing chronic transfusion therapy must be determined by the primary hematologist. Some patients may continue to receive hydroxyurea in addition to and simultaneously with blood transfusion therapy. Such patients are eligible for inclusion on the study if they hold hydroxyurea for at least 4 weeks. The ability to hold hydroxyurea (or not) with ongoing chronic transfusion therapy will be made by the primary hematologist as well. All genotypes of SCD will be eligible.
- Adequate renal function: serum/plasma creatinine \< 1.5 mg/dL and creatinine clearance \> 50 mL/min (as calculated by the Crockcroft-Gault formula).
- Adequate liver function: direct bilirubin \< 2.5 times the upper limit of normal range, AST and ALT \< 5 times the upper limit of normal range.
- Blood counts: WBC \> 3,000/mm\^3, granulocytes \> 1,000/mm\^3, hemoglobin \> 7.0 g/dL, platelets \> 150,000/mm\^3.
- Female patients of childbearing age should have a negative serum pregnancy test within one week of beginning plerixafor administration, have had a hysterectomy, be post-menopausal.
- Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II.
- Participants should either have a central line in place or be able to undergo apheresis without the necessity of the insertion of a central venous catheter
- Participants of childbearing potential should agree to use of an effective form of contraception during treatment and for at least 1 week after the last dose of plerixafor.
- ECOG performance status/Karnofsky score/Lansky score \>80.
Exclusion
- Pregnancy. Female patients of childbearing age should have a negative serum pregnancy test within one week of beginning plerixafor administration, except those that have had a hysterectomy, or are post-menopausal.
- Active viral, bacterial, fungal, or parasitic infection.
- History of cancer, excluding squamous carcinoma of the skin and cervical carcinoma in situ.
- Active and painful splenomegaly or splenomegaly (size greater than upper limit of normal) or splenic sequestration determined by ultrasound.
- Previous history of splenomegaly or splenic sequestration, unless HbS level of \<30% is documented within 48-72 hours of each plerixafor dose
- Allergy to plerixafor.
- Patients receiving hydroxyurea will not be included in the study. However, they may be included if the primary hematologist determines that hydroxyurea can be safely discontinued for at least 4 weeks prior to the plerixafor administration and apheresis. Generally, patients receiving chronic transfusion therapy can safely discontinue hydroxyurea therapy as there is unlikely to be any added benefit, but this will be determined by the primary treating hematologist.
- Poor cardiac function, as defined by an ejection fraction \< 40%.
- History of clinically proven pulmonary hypertension.
- Emergency room admission or hospitalization in the past 14 days prior to first dose of study drug.
- Major surgery in the past 30 days prior to first dose of study drug.
Key Trial Info
Start Date :
April 1 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2023
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04817345
Start Date
April 1 2023
End Date
April 1 2023
Last Update
April 7 2023
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