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

San Donato Group (GSD)

Status and phase

Enrolling

Phase 2

Conditions

Chronic Granulomatous Disease X-linked (X-CGD)

Treatments

Drug: Myelostim

Drug: Ibuprofen

Drug: Mozobil

Study type

Interventional

Funder types

Other

Identifiers

NCT03055247

2015-002356-27

Details and patient eligibility

About

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.

Full description

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.

Enrollment

3 estimated patients

Sex

Male

Ages

18 to 45 years old

Volunteers

No Healthy Volunteers

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