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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up ≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
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.
Select...
I am mostly independent and can carry out daily activities.
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I am between 18 and 65 years old.
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.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
efficacy
safety
Side effects data
From 2021 Phase 2 & 3 trial • 20 Patients • NCT0223187937%
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%
Tooth extraction
11%
Herpes simplex
11%
Urinary Tract Infection
11%
Abscess
11%
Pharyngitis
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
5%
Cellulitis
5%
Iron Deficiency Anemia
5%
Arthritis
5%
Nausea
5%
Skin Infection
5%
Tinea capitis
5%
Aspartate aminotransferase increased
5%
Anemia
5%
Tinnitus
5%
Diarrhea
5%
Infectious Diarrhea
5%
Otitis media
5%
Bone mineral content decreased
5%
Hyperglycemia
5%
Hypernatremia
5%
Hyperkalemia
5%
Joint pain
5%
Low back pain
5%
Ovarian cyst
100%
80%
60%
40%
20%
0%
Study treatment Arm
Plerixafor
G-CSF
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
~720
Find a Location
Who is running the clinical trial?
New York Blood CenterOTHER
23 Previous Clinical Trials
26,669 Total Patients Enrolled
Memorial Sloan Kettering Cancer CenterLead Sponsor
1,933 Previous Clinical Trials
585,597 Total Patients Enrolled
Weill Medical College of Cornell UniversityOTHER
1,054 Previous Clinical Trials
1,316,503 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My blood and kidney functions are within the required ranges.My heart's pumping ability is normal.I have confirmed Sickle Cell Disease, either SS or Sβ thalassemia.I agree to use birth control during the study.My kidney function is reduced with a creatinine clearance below 60 ml/min.I am not pregnant or breastfeeding.I do not have any uncontrolled illnesses or recent major surgeries.I am mostly independent and can carry out daily activities.I have an active hepatitis B, C, or HIV infection.I am between 18 and 65 years old.
Research Study Groups:
This trial has the following groups:- Group 1: Plerixafor
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the intake capacity for this clinical investigation?
"Indeed, the data on clinicaltrials.gov verify that this research study is actively recruiting participants at present. The trial was debuted on September 1st 2014 and updated most recently in November 15th 2022 with a goal of including 25 patients from one site."
Answered by AI
Has Plerixafor obtained certification from the Food and Drug Administration?
"Our in-house review team at Power scored Plerixafor's safety as a 1 due to the limited amount of data concerning its efficacy and security, characteristic of Phase 1 trials."
Answered by AI
Is a person under the age of 50 qualified to participate in this medical experimentation?
"The prerequisites for entering this trial require that participants are aged 18 or above and not exceeding 65 years old."
Answered by AI
Are there any eligibility requirements for participation in this medical trial?
"This medical trial is targeting 25 individuals aged between 18 and 65 suffering from anemia or sickle cell. Eligibility requirements include a ECOG performance score of 2 or lower, as well as Karnofsky score above 70%."
Answered by AI
Has Plerixafor been previously studied in other clinical trials?
"Currently, 18 on-going studies are observing Plerixafor with none in the final stage of testing. 54 different medical trial sites across America; most notably Seattle, Washington houses a few of them."
Answered by AI
Are there any recruitment opportunities for this experiment currently open?
"Affirmative, based on the information available at clinicaltrials.gov, this medical investigation is currently recruiting participants. The trial was initially posted in September 2014 and most recently updated on November 15th 2022. A total of 25 individuals will be accepted across one test site."
Answered by AI
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