Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD (XCGD-MOBI)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03055247|
Recruitment Status : Recruiting
First Posted : February 16, 2017
Last Update Posted : May 30, 2017
|Condition or disease||Intervention/treatment||Phase|
|Chronic Granulomatous Disease X-linked (X-CGD)||Drug: Ibuprofen Drug: Myelostim Drug: Mozobil||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Basic Science|
|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|
|Study Start Date :||November 2015|
|Estimated Primary Completion Date :||September 2017|
|Estimated Study Completion Date :||August 2018|
Experimental: XCGD mobilization
Treatment with combination of Ibuprofen, Myelostim and Mozobil
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 (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 (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.
- Percentage of patients experiencing adverse events [ Time Frame: up to 30 days after the last LP ]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).
- Number of CD34+ collected per body weight after the last LP [ Time Frame: Day 21-24 ]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)
- Change in number of CD34+ cells in PB before and after administration of Ibuprofen [ Time Frame: Day 6 and day 7 ]Cytofluorimetric analysis to determine the number of CD34+ cells present in PB on day 6 and 7 compared to before the administration of Ibuprofen
- Transduction efficiency [ Time Frame: Through study completion, an average of 1 year ]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.
- DHR (dihydrorhodamine) test in myeloid progeny [ Time Frame: Through study completion, an average of 1 year ]Correction of the functional defects in the differentiated myeloid progeny
- Functional characterization of mobilized CD34+ cells. [ Time Frame: Through study completion, an average of 1 year ]Phenotype analysis (FACS).
- Functional characterization of mobilized CD34+ cells. [ Time Frame: Through study completion, an average of 1 year ]Clonogenic activity (CFU-C) before and after transduction.
- Functional characterization of mobilized CD34+ cells. [ Time Frame: Through study completion, an average of 1 year ]Repopulating activity of mobilized CD34+ cells in immunodeficient mice.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03055247
|Contact: Fabio Ciceri, MD, PhD||39 email@example.com|
|Contact: Alessandro Aiuti, MD, PhDfirstname.lastname@example.org|
|Ospedale Pediatrico Bambino Gesù||Recruiting|
|Rome, Lazio, Italy, 00165|
|Contact: Franco Locatelli, MD, PhD|
|Sub-Investigator: Andrea Finocchi, MD|
|Ospedale San Raffaele||Recruiting|
|Milan, Lombardia, Italy, 20132|
|Contact: Fabio Ciceri, MD, PhD|
|Sub-Investigator: Alessandro Aiuti, MD, PhD|
|Sub-Investigator: Bernhard Gentner, MD, PhD|
|Sub-Investigator: Maddalena Migliavacca, MD|
|Sub-Investigator: Laura Bellio, MD|
|Principal Investigator:||Fabio Ciceri, MD, PhD||Ospedale San Raffaele|
|Principal Investigator:||Franco Locatelli, MD, PhD||Ospedale Pediatrico Bambino Gesù|