Evaluation of safety and efficacy of the combination of Ibuprofen (IBU), G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by XCGD

• Conditions: X-linked chronic granulomatous disease; MedDRA version: 18.0Level: PTClassification code 10008906Term: Chronic granulomatous diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2015-002356-27-IT
• Lead Sponsor: Ospedale San Raffaele

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-07-29
• Last Update Posted: 7 December 2015

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 3

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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•Previous Bone Marrow Transplantation or previous Gene Therapy.
•Use of other investigational agents within 4 weeks prior to study en-rolment (within 6 weeks if use of long-acting agents).
•Ongoing IFN-? treatment (within 4 weeks).
•Symptomatic inflammatory bowel disease.
•Symptomatic viral, bacterial, or fungal infection within 6 weeks of eli-gibility evaluation or active infection (including fever of unknown origin)
•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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified