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A study of SGX-001 in patients with chronic granulomatous disease caused by p47phox deficiency (p47-CGD)
EU CTIS ID: 2025-524423-50-00
What this study is testing
To evaluate the safety and tolerability of a single administration of autologous CD34⁺ HSPCs transduced with a lentiviral vector (SGX-001) in participants with p47-CGD following myeloablative conditioning
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 01 Properly completed and signed informed consent or assent (participant/LAR)
- 10 Willingness and ability of the participant (or LAR, as applicable) to comply with long-term follow-up requirements for a total duration of up to 15 years after administration of SGX-001 through participation in a dedicated LTFU study.
- 02 Confirmed diagnosis of CGD due to p47phox deficiency (confirmed mutation in the NCF1 gene by molecular genetic testing).
- 03 Absent or > 95% reduced biochemical activity of NADPH oxidase in a dihydrorhodamine flow cytometric test.
- 04 Male or female aged ≥ 18 months and have a body weight ≥ 10 kg the time of signing the informed consent or assent.
- 05 One or more ongoing or recurrent severe infectious and/or inflammatory complications, at the discretion of the Investigator.
You likely can't join if
- 01 Participant or parent/legal guardian is unable or unwilling to comply with the protocol requirements
- 10 Inability to stop using interferon-gamma at least 30 days prior to administration of the study intervention.
- 11 Participation in another interventional clinical study within 6 months prior to enrolment.
- 12 Presence of any condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or would interfere with interpretation of the study results.
- 02 Availability of a willing 10/10 HLA-matched (A, B, C, DR, DQ) sibling donor unless there is an unacceptable risk associated with an allogeneic HSCT procedure.
- 03 Previous allogeneic HSCT.
See the full eligibility criteria
Who can join
- 01 Properly completed and signed informed consent or assent (participant/LAR)
- 10 Willingness and ability of the participant (or LAR, as applicable) to comply with long-term follow-up requirements for a total duration of up to 15 years after administration of SGX-001 through participation in a dedicated LTFU study.
- 02 Confirmed diagnosis of CGD due to p47phox deficiency (confirmed mutation in the NCF1 gene by molecular genetic testing).
- 03 Absent or > 95% reduced biochemical activity of NADPH oxidase in a dihydrorhodamine flow cytometric test.
- 04 Male or female aged ≥ 18 months and have a body weight ≥ 10 kg the time of signing the informed consent or assent.
- 05 One or more ongoing or recurrent severe infectious and/or inflammatory complications, at the discretion of the Investigator.
- 06 Lack of an available 10/10 HLA-matched (A, B, C, DR, DQ) sibling donor suitable for HSCT
- 07 Ability to return to the study site for follow-up during the 1-year on-study, and to the local HSCT site during the off-protocol monitoring period.
- 08 Female participants of childbearing potential must have a negative serum pregnancy test result performed within 3 days prior to starting each cycle of mobilisation and within 5 days prior to infusion of XXX and must not be pregnant, lactating, or planning a pregnancy from Screening to XXX after SGX-001 administration.
- 09 Male participants with female partners of childbearing potential must use highly effective methods of birth control during their participation in the study and for XXX after the administration of SGX-001.
What rules you out
- 01 Participant or parent/legal guardian is unable or unwilling to comply with the protocol requirements
- 10 Inability to stop using interferon-gamma at least 30 days prior to administration of the study intervention.
- 11 Participation in another interventional clinical study within 6 months prior to enrolment.
- 12 Presence of any condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or would interfere with interpretation of the study results.
- 02 Availability of a willing 10/10 HLA-matched (A, B, C, DR, DQ) sibling donor unless there is an unacceptable risk associated with an allogeneic HSCT procedure.
- 03 Previous allogeneic HSCT.
- 04 Pregnancy or lactation.
- 05 Contraindications to any of the following: CD34+ cell mobilisation procedure (haemoglobin < 8 g/dL, cardiovascular instability, severe coagulopathy). b. Apheresis procedure. c. Conditioning regimen.
- 06 Contraindication for administration of XXX, or any component of the study intervention
- 07 Concomitant human immunodeficiency virus (HIV1 or HIV2), hepatitis B virus, hepatitis C virus, adenovirus, parvovirus B19, human T-lymphotropic virus, or toxoplasmosis infection.
- 08 Evidence of active metastatic or locoregionally advanced malignancy (including haematologic malignancy) for which survival is anticipated to be less than 3 years.
- 09 Significant organ dysfunction/co-morbidity, including but not limited to: mechanical ventilation, shortening fraction on echocardiogram < 25%, renal failure (defined as dialysis dependence), uncontrolled seizure disorder, major congenital anomaly, expected survival < 6 months.
The study team makes the final eligibility decision.
Where it's taking place
- Switzerland
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Switzerland. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.