Ibuprofen with G‑CSF and Plerixafor to mobilize stem cells for people with X‑CGD
Official title: 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
Run by IRCCS San Raffaele · NCT03055247 · Data updated 2026-11-12
This trial will test whether adding ibuprofen to G‑CSF and plerixafor helps collect enough stem cells for adults with X‑linked chronic granulomatous disease.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 45 Years |
| Sex | Men only |
| Enrollment | 3 (estimated) |
| Sponsor | IRCCS San Raffaele Academic / other |
| Locations | 2 sites (Rome, Lazio and 1 other locations) |
| Status | Recruiting |
| Started | November 6, 2015 |
| Main results expected | July 13, 2023 |
| Study ends | July 18, 2026 |
| Registry record updated | September 18, 2025 |
| Trial ID | NCT03055247 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults 18–45 with genetically confirmed X‑CGD who have good performance status, adequate organ function, and no prior bone marrow transplant or gene therapy are ideal candidates.
✕Probably not a fit
Patients with prior transplantation or gene therapy, active infections, significant organ dysfunction, thrombophilic history, or contraindications to NSAIDs, G‑CSF, or plerixafor are unlikely to benefit or are excluded.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This phase II exploratory trial tests whether combining ibuprofen with standard G‑CSF and plerixafor mobilization improves collection of hematopoietic stem and progenitor cells (HSPCs) in adults with X‑CGD. Participants receive G‑CSF, plerixafor, and a short course of ibuprofen before leukapheresis, with monitoring of safety and stem cell yield. The rationale is that chronic inflammation in X‑CGD may impair HSPC mobilization and that a non‑steroidal anti‑inflammatory could reduce inflammation and increase harvest. If a synergistic effect is seen, the regimen could be used in future gene therapy protocols for X‑CGD.
Why it matters
Potential benefit: If successful, the regimen could enable reliable collection of enough stem cells to make gene therapy options feasible for more people with X‑CGD.
How similar studies have gone: G‑CSF plus plerixafor is an established method for HSPC mobilization in gene therapy programs, but adding ibuprofen for X‑CGD patients is a novel approach with limited prior evidence.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
-
Ospedale Pediatrico Bambino Gesù
Rome, Lazio, Italy Recruiting -
Ospedale San Raffaele
Milan, Lombardy, Italy Recruiting
Study contacts
- Principal investigator: Fabio Ciceri, MD, PhD — Ospedale San Raffaele
- Study coordinator: Fabio Ciceri, MD, PhD
- Email: ciceri.fabio@hsr.it
- Phone: 39 02.2643.3903
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Base editing of blood stem cells to fix the CYBB mutation in X-linked chronic granulomatous disease.
This trial will try base-editing a person's own blood stem cells to fix a specific CYBB mutation in adult men with X-linked chronic granulomatous disease.
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This study is testing how immune problems affect people with conditions like XSCID and CGD to help find better treatments and understand their health over time.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.