RecruitingPhase 2NCT03055247

Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD

A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD


Sponsor

IRCCS San Raffaele

Enrollment

3 participants

Start Date

Nov 6, 2015

Study Type

INTERVENTIONAL

Conditions

Summary

This is a phase II exploratory study conducted to evaluate the safety and efficacy of the combination of Ibuprofen, G-CSF and Plerixafor as stem cell mobilization regimen in patients affected by X-CGD.


Eligibility

Sex: MALEMin Age: 18 YearsMax Age: 45 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a combination of three drugs — ibuprofen, G-CSF, and plerixafor — to mobilize stem cells from the bone marrow in men with X-linked chronic granulomatous disease (X-CGD), a rare immune deficiency. The goal is to collect enough stem cells to potentially use in gene therapy. You may be eligible if... - You have a confirmed genetic diagnosis of X-CGD - You are a male between 18 and 45 years old - You are in reasonably good health (Karnofsky Index above 80%) - You have normal heart, kidney, liver, and lung function - You have no history of blood clots and test negative for clotting disorders You may NOT be eligible if... - You have had a bone marrow transplant or gene therapy previously - You have used other experimental drugs recently or are currently on interferon-gamma therapy - You have active inflammatory bowel disease or a recent serious infection - You have cancer, a blood disorder, or a history of uncontrolled seizures or deep vein thrombosis - You have HIV, hepatitis C, hepatitis B, or active alcohol/substance abuse - You have a contraindication to any of the study drugs Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGIbuprofen

Ibuprofen: 3 mg/kg tid (total daily dose: 9 mg/kg); administered orally from day 1 to day 5 and then from day 14 to the day before the last LP.

DRUGMyelostim

Myelostim (G-CSF): 5 µg/kg bid (total daily dose 10 µg/kg); administered subcutaneously from day 19 to the day of the last LP.

DRUGMozobil

Mozobil (Plerixafor): 0,24 mg/kg daily. When CD34+ are ≥ 10 /μL Plerixafor will be administered subcutaneously from the next day (or from day 24 if CD34+ are \< 10 /μL) to the day of the last LP.


Locations(2)

Ospedale Pediatrico Bambino Gesù

Rome, Lazio, Italy

Ospedale San Raffaele

Milan, Lombardy, Italy

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NCT03055247