Bezpieczeństwo i skuteczność autologicznych komórek CD34+ z wektorem lentivirusowym NCF1 u pacjentów z przewlekłą chorobą ziarniniakową (p47‑CGD)

Enrollment and baseline assessments

After you agree to participate, you will sign an informed consent form and undergo initial medical examinations.

These examinations include physical Examination, vital signs, 12‑lead ecg, laboratory tests, and Assessment of your chronic granulomatous disease status.

Collection of autologous CD34+ cells

Blood is drawn from you to obtain stem cells called CD34+ cells.

The cells are collected in a sterile procedure and sent to a specialized laboratory.

Laboratory processing of cells

In the laboratory, your CD34+ cells are genetically modified with a lentiviral vector that carries the human NCF1 gene, creating the investigational product sgx-001.

The modified cells are prepared as a dispersion for infusion.

Myeloablative conditioning

Before receiving the investigational product, you will receive a high‑intensity chemotherapy regimen known as myeloablative conditioning.

This treatment clears space in your bone marrow to allow the infused cells to engraft.

Infusion of sgx-001

Once conditioning is complete, you will receive a single infusion of the modified autologous CD34+ cells (sgx-001).

The dose is a single administration; the exact volume is determined by the laboratory based on your cell count.

The infusion is given intravenously over a short period of time.

Immediate post‑infusion monitoring

After the infusion you will stay in the hospital for close observation.

Monitoring includes regular checks of vital signs, repeat laboratory tests, 12‑lead ecg, and physical examinations.

The monitoring period continues until neutrophil recovery is documented (typically by day 42) and platelet recovery is confirmed.

Discharge and scheduled follow‑up visits

Once you are stable, you will be discharged and begin a follow‑up schedule.

Follow‑up visits are planned at week 2, month 1, month 2, month 3, month 6, month 9, and month 12 after the infusion.

Follow‑up assessments at each visit

At every visit you will undergo safety assessments, including reporting of any adverse events, physical Examination, vital signs, and laboratory testing.

Blood samples are taken to measure the level of the introduced gene (transgene) and to assess nadph oxidase activity in granulocytes.

The presence of infections, the need for antibacterial or antifungal prophylaxis, and any changes in quality of life are also recorded.

Final evaluation at month 12

The month 12 visit includes the primary efficacy Assessment: response is defined as nadph oxidase activity in at least 10 % of peripheral blood granulocytes.

Secondary outcomes such as overall survival, infection rates, use of antimicrobial therapy, and quality‑of‑life measures are also evaluated.