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Clinical Trials in Italy / NCT03055247
Recruiting Phase 2

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

NCT03055247 · tracked via the Priya Life Science Italy tracker
Sponsor
IRCCS San Raffaele
Phase
Phase 2
Started
2015-11-06
Last updated
2025-09-18

Condition(s) studied

Chronic Granulomatous Disease X-linked (X-CGD)

Investigational drug(s) / intervention(s)

Ibuprofen →MyelostimMozobil

Ibuprofen: 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.

Myelostim: 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.

Mozobil: 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.

Study 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
MALE
Min age
18 Years
Max age
45 Years
Healthy volunteers
No
Inclusion Criteria: * Genetic diagnosis of X-CGD * 18-45 years of age * Karnofsky Index \> 80 % * Adequate cardiac, renal, hepatic and pulmonary function. * Negative thrombophilic screen and negative history for previous thrombotic events * Written informed consent Exclusion Criteria: * Previous Bone Marrow Transplantation or previous Gene Therapy. * Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents). * Ongoing IFN-γ treatment (within 4 weeks). * Symptomatic inflammatory bowel disease. * Symptomatic viral, bacterial, or fungal infection within 6 weeks of eligibility * Neoplasia (except local skin cancer) or history of "familial" cancer * Myelodysplasia or other serious hematological disorder * History of uncontrolled seizures and deep venous thrombosis * Other systemic disease judged as incompatible with the procedure * Positivity for HIV and/or HCV RNA and/or HbsAg and/or HBV DNA * Active alcohol or substance abuse within 6 months of the study. * Contraindications to IBU, G-CSF, Plerixafor or Pantoprazole administration

Primary outcome measure(s)

  • Percentage of patients experiencing adverse events — up to 30 days after the last LP
    Percentage of patients experiencing adverse events, as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse events (CTCAe v3.0, 2006) (all grades).
  • Number of CD34+ collected per body weight after the last LP — Day 21-24
    Cytofluorimetric analysis for CD34 on PB and on collected PBSC to calculate the number of CD34+ cells collected per kg body weight. The analysis will be performed at the end of the LP(s) (Day 21-24)

Trial sites (2)

FacilityCityRegionStatus
Ospedale Pediatrico Bambino Gesù Rome Lazio Recruiting
Ospedale San Raffaele Milan Lombardy Recruiting

On this site

📄 Advil (ibuprofen) drug profile →

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03055247 on ClinicalTrials.gov ↗ ← All trials in Italy