Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor

NCT04817345 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: ASPIRES2
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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 hematopoietic stem cells from patients with sickle cell disease. In this study, investigators want to study the safety and feasibility of collecting peripheral blood stem cells from pediatric and young adult patients with sickle cell disease after administering plerixafor. Studying these peripheral blood stem cells will help in optimizing the yield of peripheral CD34+ cells from pediatric and young adult patients with sickle cell disease, which in turn will help to develop better gene therapies for these patients. Primary Objectives

• Determine the safety profile associated with administration of plerixafor in pediatric and young adult patients with sickle cell disease (SCD).
• To estimate the number of CD34+ cells/kg of body weight that can be collected with peripheral apheresis after administration of plerixafor in pediatric and young adult patients with SCD. Exploratory Objectives
• To describe the kinetics of CD34+ cell mobilization in peripheral blood after - + cells obtained from pediatric and young adult patients with SCD.
• To study the effect of hydroxyurea therapy on senescence in plerixafor-mobilized CD34+ cells obtained from pediatric and young adult patients with SCD.

Conditions

Sickle Cell Disease

Keywords

Plerixafor; Leukapheresis; Mobilization; Stem Cells; Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Number of participants with sufficient collection of hematopoietic stem cells (HSCs) without serious adverse events.

  Sufficient collection of HSCs from peripheral blood after plerixafor mobilization without serious adverse events (SAEs)

  2 days

Study Arms (1)

Plerixafor

EXPERIMENTAL

Participants will receive a subcutaneous dose of 0.24 mg/kg of plerixafor once daily (Q24hr) x 2 days.

Drug: Plerixafor

Interventions

Plerixafor DRUG

Subcutaneous

Also known as: Mozobil ™

Plerixafor

Eligibility Criteria

• Age: 10 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead
• Doris Duke Charitable Foundation: collaborator

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

plerixafor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Akshay Sharma, MBBS

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

• Mitch Weiss, MD, PhD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2021
• First Posted: March 26, 2021
• Study Start: April 1, 2023
• Primary Completion: April 1, 2023
• Study Completion: April 1, 2023
• Last Updated: April 7, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be made available at the time of article publication.
• Access Criteria: Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.