Chronic Granulomatous Disease X-linked (X-CGD) Clinical Trial
— XCGD-MOBIOfficial 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
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.
| Status | Recruiting |
| Enrollment | 3 |
| Est. completion date | July 18, 2023 |
| Est. primary completion date | July 13, 2023 |
| Accepts healthy volunteers | No |
| Gender | Male |
| Age group | 18 Years to 45 Years |
| Eligibility | 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-? 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 |
| Country | Name | City | State |
|---|---|---|---|
| Italy | Ospedale San Raffaele | Milan | Lombardia |
| Italy | Ospedale Pediatrico Bambino Gesù | Rome | Lazio |
| Lead Sponsor | Collaborator |
|---|---|
| IRCCS San Raffaele | Fondazione Telethon |
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* Note: There are 26 references in all — Click here to view all references
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Percentage of patients experiencing adverse events | 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). | up to 30 days after the last LP | |
| Primary | Number of CD34+ collected per body weight after the last LP | 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) | Day 21-24 | |
| Secondary | Change in number of CD34+ cells in PB before and after administration of Ibuprofen | Cytofluorimetric analysis to determine the number of CD34+ cells present in PB on day 6 and 7 compared to before the administration of Ibuprofen | Day 6 and day 7 | |
| Secondary | Transduction efficiency | Efficient transduction of mobilized HSPC with a lentiviral vector encoding for a corrective cDNA of the human gp91phox gene. Frequency and Vector Copy Number tested by PCR. | Through study completion, an average of 1 year | |
| Secondary | DHR (dihydrorhodamine) test in myeloid progeny | Correction of the functional defects in the differentiated myeloid progeny | Through study completion, an average of 1 year | |
| Secondary | Functional characterization of mobilized CD34+ cells. | Phenotype analysis (FACS). | Through study completion, an average of 1 year | |
| Secondary | Functional characterization of mobilized CD34+ cells. | Clonogenic activity (CFU-C) before and after transduction. | Through study completion, an average of 1 year | |
| Secondary | Functional characterization of mobilized CD34+ cells. | Repopulating activity of mobilized CD34+ cells in immunodeficient mice. | Through study completion, an average of 1 year |