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

• Status: Recruiting
• Trial end date: 2023-07-18
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: IRCCS San Raffaele
• Collaborator: Fondazione Telethon
• Treatments: Ibuprofen

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