Plerixafor for Anemia, Sickle Cell

1
Effectiveness
2
Safety
St. Jude Children's Research Hospital, Memphis, TN
Anemia, Sickle Cell+1 More
Plerixafor - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Anemia, Sickle Cell

Study Summary

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

See full description

Eligible Conditions

  • Anemia, Sickle Cell
  • Sickle Cell Disease (SCD)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether Plerixafor will improve 1 primary outcome in patients with Anemia, Sickle Cell. Measurement will happen over the course of 2 days.

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

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Compared to trials

Side Effects for

Filgrastim (Neupogen) and Plerixafor
Bone pain
43%
Lymphocyte count decreased
41%
Platelet count decreased
39%
Anemia
35%
Alkaline phosphatase increased
24%
Lymphocyte count increased
20%
Leukocytosis
20%
Nausea
16%
Diarrhea
12%
Arthralgia
12%
Chills
10%
Dizziness
10%
Headache
8%
Edema - limbs
8%
Bleeding at catheter site
8%
Hypoalbuminemia
8%
Back pain
8%
Paresthesia
8%
Activated partial thromboplastin time prolonged
8%
Vomiting
8%
Dyspnea
8%
INR increased
8%
Constipation
6%
Fatigue
6%
Hyperuricemia
6%
Abdominal pain
6%
White blood cell decreased
6%
Anxiety
6%
Hypercalcemia
4%
Hypokalemia
4%
Hypotension
4%
Non-cardiac chest pain
4%
Injection site reaction
4%
Hypocalcemia
4%
Pain in extremity
4%
Hypertension
4%
Fever
4%
Hypophosphatemia
4%
Peripheral sensory neuropathy
4%
Hypomagnesemia
2%
Weight loss
2%
Creatinine increased
2%
Aspartate aminotransferase increased
2%
Rhinovirus positive culture
2%
Cellulitis
2%
Febrile neutropenia
2%
Scleral disorder
2%
Neutrophil count decreased
2%
Hyperglycemia
2%
Hand cramping
2%
Confusion
2%
Proteinuria
2%
Hypohidrosis
2%
Rash maculo-papular
2%
Drainage at catheter
2%
Pain
2%
Alanine aminotransferase increased
2%
Central line site cellulitis
2%
Oral dysesthesia
2%
Blood bilirubin increased
2%
Anorexia
2%
Joint range of motion decreased
2%
Neck pain
2%
Numbness of face
2%
Productive cough
2%
Pruritus
2%
Hyponatremia
2%
Purpura
0%
Upper respiratory infection
0%
Hyperhidrosis
0%
Pain at biopsy site
0%
Acute kidney injury
0%
Palpitations
0%
Bloating
0%
Gastroesophageal reflux disease
0%
Pain at catheter site
0%
Discharge at catheter
0%
Bruising
0%
Hyperkalemia
0%
Hypernatremia
0%
Hypoglycemia
0%
Generalized muscle weakness
0%
Neuralgia
0%
Movements involuntary
0%
Tremor
0%
Depression
0%
Urinary retention
0%
Cough
0%
Skin ulceration
0%
Erythema at catheter site
0%
Fall
0%
Atrial fibrillation
0%
Dyspepsia
0%
Dysphagia
0%
Fever blister
0%
Panic attack
0%
Hypersomnia
0%
Sore throat
0%
This histogram enumerates side effects from a completed 2016 Phase 2 trial (NCT02098109) in the Filgrastim (Neupogen) and Plerixafor ARM group. Side effects include: Bone pain with 43%, Lymphocyte count decreased with 41%, Platelet count decreased with 39%, Anemia with 35%, Alkaline phosphatase increased with 24%.

Trial Design

2 Treatment Groups

Control
Plerixafor

This trial requires 15 total participants across 2 different treatment groups

This trial involves 2 different treatments. Plerixafor is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Plerixafor
Drug
Participants will receive a subcutaneous dose of 0.24 mg/kg of plerixafor once daily (Q24hr) x 2 days.
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Plerixafor
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 2 days
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 2 days for reporting.

Closest Location

St. Jude Children's Research Hospital - Memphis, TN

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 9 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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.
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.

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who should consider clinical trials for anemia, sickle cell?

Add answer

There were several clinical considerations that led us to feel there were enough anemic pediatric SSC patients that might benefit from such an evaluation. These were: 1) Anemic SSC patients present in a range of ages, including newborn babies. 2) A large number of SSC patients have anemia from other causes. 3) Many SSC patients present with other chronic medical conditions, and their enrollment criteria might be more appropriate for these studies or the treatment of which they are enrolled.

Unverified Answer

What are common treatments for anemia, sickle cell?

Add answer

There are many types of treatment used for the management of SCD, including transfusions and medications, and the effect of aspirin on the process, can be variable. The most successful treatment is to give patients the optimal levels of oxygen that would support their red blood cell production and a regular check-up to monitor the patients for health problems that might be related to sickle cell anemia. A common treatment for all patients is giving daily folic Acid supplements to support red blood cell production and can prevent anemia and its related health complications. It is critical to consider the medical history of the patients when managing with anemia therapy and to make use of the available information to develop an effective plan to achieve desired treatment results.

Unverified Answer

What causes anemia, sickle cell?

Add answer

Although genetic factors are involved in the development of anemia, they only account for a minority of anemia cases. Rather, the most likely causes of anemia, sickle cell disease or iron deficiency, affect hundreds of millions of people, and are potentially preventable with proper nutrition at a population level.

Unverified Answer

Can anemia, sickle cell be cured?

Add answer

If an anemia is present, it is advisable to take folinic acid (10 micrograms/d) to increase red cell production because it reduces the sickling of red blood cells. (This can cause symptoms of anemia in those already malnourished or malnourished, for example, due to the effects of vitamin deficiency). With folinic acid, there is an increased iron absorption in stool, so it is advisable to give this to those with anemia for this reason. Vitamin A supplementation is also required. Patients treated for anemia are often iron deficient, because erythrocytes (red blood cells) have a shorter life span in those who are iron deficient.

Unverified Answer

What are the signs of anemia, sickle cell?

Add answer

Most common signs and symptoms of anemia are fatigue, shortness of breath, dizziness, paleness, tiredness and headache. However, signs and symptoms of anemia are not specific to anemia, sickle cell. Because of this, if you cannot pinpoint the cause of your symptoms, you should see a medical professional for further work-up.

Unverified Answer

What is anemia, sickle cell?

Add answer

There are several types of anemia. The common manifestation in children that are mostly caused by a reduction in hemoglobin levels. Children with sickle cell anemia are much more prone toward abnormal development such as strokes. The use of oral sirolimus as a first line therapy to treat children with sickle cell anemia has been approved by a number of major national and international bodies like the ECMO International Clinical Practice Guidelines.

Unverified Answer

How many people get anemia, sickle cell a year in the United States?

Add answer

About 5.2 million US adults are diagnosed with hemoglobinopathies. This makes up 13.4% of US adults. Anemia or sickle cell disease affects over 12 million Americans.

Unverified Answer

Does plerixafor improve quality of life for those with anemia, sickle cell?

Add answer

In a small group of patients with hemolysis and/or anemia, treatment with PTK improves HRQOL, and this effect is sustained at 12 weeks following cessation of PTK treatment.

Unverified Answer

What does plerixafor usually treat?

Add answer

Plerixafor is a synthetic type II CXCR4 ligand, which can affect the adhesion of various hematological cell types. It possesses very different biological activities including anti-tumor, anti-inflammatory, myelosuppressive, neurotropic and immunosuppressive activities. This compound often functions as anti-adhesion and anti-tumor activity for these hematological cells, and plays similar roles with related chemokines for immune cells including T and B cells. We believe the use of plerixafor and its related chemokines for cancer treatment could be a more effective and safe therapy for multiple myeloma and leukemia.

Unverified Answer

How serious can anemia, sickle cell be?

Add answer

Anemia has been and remains an important cause of mortality among adults in the US. The seriousness of anemia remains unknown and warrants further study. Anemia, and the resultant increased risk for morbidity and mortality, warrants increased efforts to treat it.

Unverified Answer

What is the primary cause of anemia, sickle cell?

Add answer

Approximately 60% of HbF individuals have no identifiable cause, but when patients have anemia from other identifiable causes, the primary cause is usually from an unrecognized cause, such as Vitamin B12 deficiency.

Unverified Answer

What is the latest research for anemia, sickle cell?

Add answer

There is evidence that regular NSAIDs may increase the risk of blood clots, so one may want to consult their physician before taking them. In a review article in Annals of Internal Medicine, Dr. Siegel describes the risks that ibuprofen may have. Although it appears that this risk is not a concern for NSAIDs, it is prudent to consult the drug information (DI) for each anti-injury drug before taking them. If one wishes to use an NSAID to reduce pain, then it is important to understand the risk of blood clots and consult with one's physician. The risk of bleeding of a bloodletting drug is also extremely low.

Unverified Answer
See if you qualify for this trial
Get access to this novel treatment for Anemia, Sickle Cell by sharing your contact details with the study coordinator.