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Hematopoietic Stem Cell Mobilizer

Plerixafor for Sickle Cell Disease

Phase 1

Waitlist Available

Led By Farid Boulad, MD

Research Sponsored by Memorial Sloan Kettering Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Acceptable organ and marrow function: WBC ≥ 3,000/μL, ANC ≥ 1,500/μL, platelets ≥150,000/μL, Hemoglobin ≥ 6 gm/dL, Calculated creatinine clearance ≥ 60ml/min using the Cockcroft-gault equation

Patients must have confirmed and measurable Sickle Cell Disease, defined by SS or Sβ thalassemia confirmed by hemoglobin fractionation

Must not have

Patients with poor cardiac function as defined by an ejection fraction < 40% are excluded due to potential poor tolerance of the fluid shifts with leukapheresis

Patients with a creatinine clearance of < 60 ml/min

Timeline

Screening 3 weeks

Treatment Varies

Follow Up ≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.

Awards & highlights

No Placebo-Only Group

Summary

This trial will test the safety and efficacy of the drug Plerixafor in patients with sickle cell disease.

Who is the study for?

This trial is for adults aged 18-65 with confirmed Sickle Cell Disease (SS or Sβ thalassemia). Participants must have a certain level of physical fitness, adequate organ and marrow function, and not be pregnant or breastfeeding. They should not have any major health issues that could affect study participation and must agree to use contraception.

What is being tested?

The trial is testing the safety and effectiveness of Plerixafor in patients with sickle cell disease. Plerixafor is already FDA-approved for cancer patients to increase blood stem cell counts before collection but hasn't been approved yet for those with sickle cell disease.

What are the potential side effects?

While specific side effects for sickle cell patients are being studied, known side effects from other uses include diarrhea, nausea, tiredness, headache, joint pain, dizziness, and injection site reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My blood and kidney functions are within the required ranges.

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I have confirmed Sickle Cell Disease, either SS or Sβ thalassemia.

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I am mostly independent and can carry out daily activities.

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I am between 18 and 65 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My heart's pumping ability is normal.

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My kidney function is reduced with a creatinine clearance below 60 ml/min.

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I am not pregnant or breastfeeding.

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I have an active hepatitis B, C, or HIV infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ ≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.

This trial's timeline: 3 weeks for screening, Varies for treatment, and ≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

efficacy

safety

Side effects data

From 2021 Phase 2 & 3 trial • 20 Patients • NCT02231879

37%

Bone pain

32%

Upper Respiratory Tract Infection

26%

Weight gain

16%

Blood alkaline phosphatase increased

16%

Upper respiratory infection

16%

Injection Site Reaction

16%

Hyperuricemia

16%

Hypocalcemia

16%

Headache

16%

Rash

11%

Abscess

11%

Pharyngitis

11%

Tooth extraction

11%

Herpes simplex

11%

Urinary Tract Infection

11%

Tinea corporis

11%

Fracture

11%

Migraine

11%

Acute bronchitis

11%

Elective surgery

11%

Acute sinusitis

11%

Alanine aminotransferase increased

11%

Creatinine increased

11%

Knee pain

11%

Arthralgia

11%

Papular rash

11%

Pruritic rash

Arthritis

Cellulitis

Skin Infection

Iron Deficiency Anemia

Nausea

Tinea capitis

Aspartate aminotransferase increased

Anemia

Tinnitus

Diarrhea

Infectious Diarrhea

Otitis media

Bone mineral content decreased

Hyperglycemia

Hypernatremia

Hyperkalemia

Joint pain

Low back pain

Ovarian cyst

100%

80%

60%

40%

20%

Study treatment Arm

Plerixafor

G-CSF

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: PlerixaforExperimental Treatment1 Intervention

Patients will receive a single dose of subcutaneous plerixafor with peripheral blood studies at approximately 0-2 hours before, approximately 6-12 hours after, and approximately 20-48 hours after plerixafor administration, with leukapheresis in the last 3 patients on the protocol. Collected HPCs will be transferred to the MSKCC CTCEF to determine if the HPCs are amenable to transduction with a lentiviral vector encoding the normal ß- globin gene.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Plerixafor

2011

Completed Phase 3

~710

0 / 8Eligibility criteria met