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Growth Factor

Stem Cell Mobilization for T Cell Lymphocytopenia

Phase 2

Recruiting

Led By Irini Sereti, M.D.

Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria Checklist

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

Must have

HTLV-1 and HTLV-2 seronegative

Persons with documented history of ICL with genetic defects related to hematopoietic cells

Must not have

Need for anticoagulant medication other than aspirin, clopidogrel, or other antiplatelet agent

Uncontrolled hypertension

Timeline

Screening 3 weeks

Treatment Varies

Follow Up throughout the study

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a combination of drugs to see if they can help people with a rare syndrome called idiopathic CD4 lymphocytopenia. The syndrome is defined by low CD4 T cell counts, which can leave patients at risk for opportunistic infections. The drugs being tested are granulocyte colony stimulating factor (G-CSF) and plerixafor. The trial will involve healthy volunteers and patients with the syndrome.

Who is the study for?

This trial is for adults aged 18-65 with a rare condition called Idiopathic CD4 Lymphocytopenia (ICL), which involves very low levels of certain immune cells. Participants must have documented ICL, be HIV negative, and not currently have any serious illnesses or conditions that could interfere with the study. They should also agree to use effective birth control during the study.

What is being tested?

The trial tests Plerixafor and Filgrastim's ability to mobilize stem cells in patients with ICL compared to healthy volunteers. These drugs may help move important progenitor cells into the bloodstream for collection and research on T cell development within mice models.

What are the potential side effects?

Possible side effects include bone pain, headache, nausea, diarrhea, tiredness, and injection site reactions from Filgrastim; while Plerixafor can cause stomach upset, bloating or swelling at the injection site.

Eligibility Criteria

Inclusion Criteria

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

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I do not have HTLV-1 or HTLV-2.

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I have a genetic defect in my blood cells causing immune deficiency.

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I am healthy with a normal white blood cell count and hemoglobin level.

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I am between 18 and 65 years old.

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My weight is between 50 kg and 167 kg, and I am not over 175% of my ideal weight.

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I am willing to stay in the hospital for about a day.

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I can undergo procedures that require vein access.

Exclusion Criteria

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

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I need blood thinners other than aspirin or clopidogrel.

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My high blood pressure is not under control.

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I do not have an ongoing infection that is not under control.

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I am on medication for an autoimmune disease.

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I have a history of vasculitis.

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My spleen is enlarged due to my chronic liver condition.

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My kidney function is reduced with a creatinine clearance below 50 mL/min.

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I have symptoms of heart disease.

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I am currently breastfeeding.

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I am currently taking lithium.

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I have or had blood or lymph node cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ throughout the study

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~throughout the study

This trial's timeline: 3 weeks for screening, Varies for treatment, and throughout the study for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

To mobilize CD34+ HPCs in ICL patients and healthy volunteers for collection and transfer into immunocompromised mice to investigate thymic development, survival, and trafficking of these cells in murine lymphoid and non-lymphoid organs

Secondary study objectives

To assess peripheral CD4 T cell and CD34+ HPC numbers and functions in ICL subjects compared to controls following G CSF and plerixafor administration

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%

Tooth extraction

11%

Pharyngitis

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

2Treatment groups

Experimental Treatment

Group I: PlerixaforExperimental Treatment1 Intervention

ICL and healthy volunteers will be given 0.24 mg/kg as a single dose (maximum dose: 40 mg) 11 hours prior to apheresis

Group II: FilgrastimExperimental Treatment1 Intervention

ICL and healthy volunteers will be given 10 microgram/kg daily for 5 days administered according to a vial-based algorithm to reduce wastage and increase the G-CSF dose given to lighter-weight donors to improve CD34+ yields

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Filgrastim

2000

Completed Phase 3

~3690

Plerixafor

2011

Completed Phase 3

~710