RecruitingPhase 2NCT03055247

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

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


Sponsor

IRCCS San Raffaele

Enrollment

3 participants

Start Date

Nov 6, 2015

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Sex: MALEMin Age: 18 YearsMax Age: 45 Years

Inclusion Criteria6

  • Genetic diagnosis of X-CGD
  • 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 Criteria12

  • 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

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Interventions

DRUGIbuprofen

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.

DRUGMyelostim

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.

DRUGMozobil

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.


Locations(2)

Ospedale Pediatrico Bambino Gesù

Rome, Lazio, Italy

Ospedale San Raffaele

Milan, Lombardy, Italy

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NCT03055247