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

NCT ID: NCT03055247

Last Updated: 2025-09-18

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-11-06
• Study Completion Date: 2026-07-18

Brief 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.

Detailed Description

We designed a mobilization trial with the aim of collecting a sufficient number of HSPC in X-CGD patients; it is well known that this procedure is challenging for these patients, potentially due to functional defects induced by their chronic inflammatory state.

The combination of G-CSF and Plerixafor is considered state of the art for HSPC harvest in gene therapy trials; we considered to add a non-steroidal inflammatory drug to increase HSPC mobilization and reduce inflammation that could have a role in altering HSPC content.

If this trial confirms the synergistic effect of the three drugs under investigation, such a regimen will be considered for a HSPC mobilization in future gene therapy trial for X-CGD patients.

Conditions

Chronic Granulomatous Disease X-linked (X-CGD)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: BASIC_SCIENCE
• Blinding Strategy: NONE

Study Groups

XCGD mobilization

Treatment with combination of Ibuprofen, Myelostim and Mozobil

Group Type: EXPERIMENTAL

Ibuprofen

Intervention Type: DRUG

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

Intervention Type: DRUG

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

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

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.

Intervention Type: DRUG

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.

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Fondazione Telethon

OTHER

Sponsor Role: collaborator

IRCCS San Raffaele

OTHER

Sponsor Role: lead

Responsible Party

Ciceri Fabio

Director Hematology and BMT Unit

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Fabio Ciceri, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Ospedale San Raffaele

Franco Locatelli, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Ospedale Pediatrico Bambino Gesù

Locations

Ospedale Pediatrico Bambino Gesù

Rome, Lazio, Italy

Site Status: RECRUITING

Ospedale San Raffaele

Milan, Lombardy, Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Fabio Ciceri, MD, PhD

Role: CONTACT

ciceri.fabio@hsr.it

39 02.2643.3903

Alessandro Aiuti, MD, PhD

Role: CONTACT

aiuti.alessandro@hsr.it

+390226434875

Facility Contacts

Franco Locatelli, MD, PhD

Role: primary

Fabio Ciceri, MD, PhD

Role: primary

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Other Identifiers

2015-002356-27

Identifier Type: -

Identifier Source: org_study_id