Chronic Granulomatous Disease (CGD)Phase 1/2NCT07826767
Open-label Gene Therapy Study in p47-CGD
A Phase 1/2 study testing SGX-001 in people with Chronic Granulomatous Disease (CGD), run by Somagenetix AG at 3 sites in 3 countries.
Trial summary
Chronic granulomatous disease (CGD) caused by p47phox deficiency (p47-CGD) is a life-threatening genetic disorder causing nicotinamide adenine dinucleotide phosphate (NADPH) oxidase deficiency in phagocytes. Read more about this study
- Who is it for?
- Ages 18 months and older
- Conditions the study is for
- Chronic Granulomatous Disease (CGD)
- What is being tested?
- SGX-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 18 Aug 2026to Sep 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants are eligible to be included in the study only if all of the following criteria apply:
- Properly completed and signed informed consent or assent (participant/legally authorised representative).
- Confirmed diagnosis of CGD due to p47phox deficiency (confirmed mutation in the NCF1 gene by molecular genetic testing).
- Absent or > 95% reduced biochemical activity of NADPH oxidase in a dihydrorhodamine (DHR) flow cytometric test.
- Male or female aged ≥ 18 months and have a body weight ≥ 10 kg at the time of signing the informed consent or assent.
- One or more ongoing or recurrent severe infectious and/or inflammatory complications, at the discretion of the Investigator.
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- Note: Participants must not have an uncontrolled active infection at the time of screening or apheresis. Infectious or inflammatory complications should be clinically stable (e.g., afebrile, hemodynamically stable, and on appropriate antimicrobial/anti-inflammatory therapy if indicated) before initiation of screening procedures and prior to leukapheresis.
- Lack of an available 10/10 HLA-matched (A, B, C, DR, DQ) sibling donor suitable for HSCT.
- 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.
- 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 busulfan and must not be pregnant, lactating, or planning a pregnancy from Screening to Month 12 after SGX-001 administration.
- Note: Female participants of childbearing potential will be included if they are either sexually inactive (abstinent) for 90 days prior to starting the first cycle of mobilisation, or are using a highly effective birth control methods (i.e., results in < 1% failure rate when used consistently and correctly).
- Note: Sexual abstinence or use of contraceptive measures must continue throughout the study and for 12 months after the administration of SGX-001.
- Male participants with female partners of childbearing potential must use highly effective methods of birth control during their participation in the study and for 12 months after the administration of SGX-001.
- Willingness and ability of the participant (or a legally authorised representative, 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.
Who cannot join
- Participants are excluded from the study if any of the following criteria apply:
- Participant or parent/legal guardian is unable or unwilling to comply with the protocol requirements.
- 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.
- Previous allogeneic HSCT.
- Pregnancy or lactation.
- Contraindications to any of the following:
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- CD34+ cell mobilisation procedure (haemoglobin < 8 g/dL, cardiovascular instability, severe coagulopathy).
- Apheresis procedure.
- Conditioning regimen.
- Contraindication for administration of filgrastim, lenograstim, plerixafor, busulfan, or any component of the study intervention.
- Concomitant human immunodeficiency virus (HIV1 or HIV2), hepatitis B virus (HBV), hepatitis C virus (HCV), adenovirus, parvovirus B19, human T-lymphotropic virus (HTLV1 2), or toxoplasmosis infection.
- Evidence of active metastatic or locoregionally advanced malignancy (including haematologic malignancy) for which survival is anticipated to be less than 3 years.
- 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.
- Inability to stop using IFN gamma at least 30 days prior to administration of the study intervention.
- Participation in another interventional clinical study within 6 months prior to enrolment.
- 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.
Trial locations and contacts
3 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 3 countries
Germany1 site, 1 recruiting
- Not yet recruiting
Universitaetsklinikum Ulm
Ulm, 89075
Ansgar Schulz, Prof. Dr., +4973150057174, ansgar.schulz@uniklinik-ulm.de
Spain1 site, 1 recruiting
- Not yet recruiting
Hospital Universitari Vall D Hebron
Barcelona, 08035
Pere Soler Palacín, Dr., +34934893140, pere.soler@vallhebron.cat
Switzerland1 site, 1 recruiting
- Recruiting
University Children's Hospital Zurich
Zurich, 8008
Tayfun Güngör, Prof. Dr. med., +41 44 249 59 79, tayfun.guengoer@kispi.uzh.ch
About this study
Official title: An Open-label, Single-arm, Phase 1/2 First-in-human Study to Assess the Safety and Efficacy of Autologous CD34+ Cells Transduced With a Lentiviral Vector Encoding the Human NCF1 Gene (SGX-001) in Paediatric and Adult Patients With Chronic Granulomatous Disease Caused by p47phox Deficiency
Chronic granulomatous disease (CGD) caused by p47phox deficiency (p47-CGD) is a life-threatening genetic disorder causing nicotinamide adenine dinucleotide phosphate (NADPH) oxidase deficiency in phagocytes. This leads to severe bacterial and fungal infections as well as hyperinflammatory complications that significantly reduces life expectancy.
Standard of care includes daily antimicrobial prophylactic treatment by conventional pharmacotherapy. This aims to prevent or reduce the frequency and severity of infections and other disease manifestations. However, the underlying genetic defect in neutrophil cytosolic factor 1 (NCF1) cannot be cured by pharmacotherapy, and many p47-CGD patients suffer from significant morbidity, impaired quality of life, and early mortality. Allogeneic haematopoietic stem cell transplant (HSCT), the only established curative treatment and carries substantial risks when using non-sibling donors. Risks include graft failure and graft-versus-host disease.
Read more
Treatment with SGX-001 aims to cure the underlying genetic defect using autologous haematopoietic stem and progenitor cells (HSPCs) transduced with a lentiviral self-inactivating vector to express transgenic p47phox protein and restore NADPH oxidase function in phagocytes. This treatment eliminates the need for allogeneic HSCT and could offer a safer alternative to allogeneic transplantation for patients without ideal donors.
In this study, safety and efficacy of SGX-001 will be investigated in participants with p47-CGD who have an indication for allogeneic HSCT but lack a human leukocyte antigen-matched suitable sibling donor.
- Conditions
- Chronic Granulomatous Disease (CGD)
- Being tested
- SGX-001
- Keywords
- p47-CGD
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SGX-001Biological
Autologous CD34+ cell-enriched population that contains HSPCs transduced with a lentiviral vector encoding the human NCF1 gene
| Study group | Role | What they receive |
|---|---|---|
| SGX-001 | Experimental | All eligible participants will be assigned to a single active treatment group and receive infusion of SGX-001. Receives: Biological: SGX-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of safety events following a single administration of SGX-001
Safety and tolerability of a single administration of autologous CD34⁺ HSPCs transduced with a lentiviral vector (SGX-001) in participants with p47-CGD will be assessed based on the incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), SAEs related to SGX-001, adverse events of special interest (AESIs), TEAEs by intensity grade, and discontinuations due to TEAEs, as well as clinical laboratory abnormalities, vital signs, 12-lead ECGs, and physical examination findings. Safety endpoints will be presented overall and/or by study stage or visit, as applicable. Laboratory abnormalities will be graded according to NCI CTCAE version 6.0.
Measured: 12 months
Number of participants with a response to SGX-001 based on NADPH oxidase activity in peripheral blood granulocytes
Efficacy of a single administration of autologous CD34⁺ HSPCs transduced with a lentiviral vector (SGX-001) in participants with p47-CGD will be assessed based on the proportion of peripheral blood granulocytes with detectable NADPH oxidase activity. Participants will be considered responders if NADPH oxidase activity is present in ≥10% of peripheral blood granulocytes and non-responders if NADPH oxidase activity is present in <10% of peripheral blood granulocytes. The outcome will be summarized as the number and percentage of participants with a response.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2026
- Study start18 Aug 2026
- Primary completionJul 2028 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Granulomatous Disease (CGD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Somagenetix AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07826767
- Sponsor's protocol ID
- SGX-001-CT-01
- Other IDs
- 2025-524423-50-00, 702243 SGX-001-CT-01, 2025
- Status last verified
- Sep 2026
- First submitted
- 7 Sep 2026
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Phase 1/2 First-in-human Study to Assess the Safety and Efficacy of Autologous CD34+ Cells Transduced With a Lentiviral Vector Encoding the Human NCF1 Gene (SGX-001) in Paediatric and Adult Patients With Chronic Granulomatous Disease Caused by p47phox Deficiency
- ClinicalTrials.gov ID
- NCT07826767
- Acronym / sponsor ID
- SGX-001-CT-01
- Conditions
- Chronic Granulomatous Disease (CGD)
- Keywords
- p47-CGD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Somagenetix AGIndustry (pharma, biotech, devices)
- Study start
- 18 Aug 2026 (actual)
- Primary completion
- Jul 2028 (estimated)
- Study completion
- Sep 2028 (estimated)
- First posted
- 17 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Chronic granulomatous disease (CGD) caused by p47phox deficiency (p47-CGD) is a life-threatening genetic disorder causing nicotinamide adenine dinucleotide phosphate (NADPH) oxidase deficiency in phagocytes. This leads to severe bacterial and fungal infections as well as hyperinflammatory complications that significantly reduces life expectancy.
Standard of care includes daily antimicrobial prophylactic treatment by conventional pharmacotherapy. This aims to prevent or reduce the frequency and severity of infections and other disease manifestations. However, the underlying genetic defect in neutrophil cytosolic factor 1 (NCF1) cannot be cured by pharmacotherapy, and many p47-CGD patients suffer from significant morbidity, impaired quality of life, and early mortality. Allogeneic haematopoietic stem cell transplant (HSCT), the only established curative treatment and carries substantial risks when using non-sibling donors. Risks include graft failure and graft-versus-host disease.
Treatment with SGX-001 aims to cure the underlying genetic defect using autologous haematopoietic stem and progenitor cells (HSPCs) transduced with a lentiviral self-inactivating vector to express transgenic p47phox protein and restore NADPH oxidase function in phagocytes. This treatment eliminates the need for allogeneic HSCT and could offer a safer alternative to allogeneic transplantation for patients without ideal donors.
In this study, safety and efficacy of SGX-001 will be investigated in participants with p47-CGD who have an indication for allogeneic HSCT but lack a human leukocyte antigen-matched suitable sibling donor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SGX-001 | Experimental | Biological: SGX-001All eligible participants will be assigned to a single active treatment group and receive infusion of SGX-001. |
Who may qualify
- Ages
- 18 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- Participants are eligible to be included in the study only if all of the following criteria apply:
- Properly completed and signed informed consent or assent (participant/legally authorised representative).
- Confirmed diagnosis of CGD due to p47phox deficiency (confirmed mutation in the NCF1 gene by molecular genetic testing).
- Absent or > 95% reduced biochemical activity of NADPH oxidase in a dihydrorhodamine (DHR) flow cytometric test.
- Male or female aged ≥ 18 months and have a body weight ≥ 10 kg at the time of signing the informed consent or assent.
Key exclusion criteria (16 listed)
- Participant or parent/legal guardian is unable or unwilling to comply with the protocol requirements.
- 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.
- Previous allogeneic HSCT.
- Pregnancy or lactation.
- Contraindications to any of the following:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SGX-001Autologous CD34+ cell-enriched population that contains HSPCs transduced with a lentiviral vector encoding the human NCF1 gene | Biological | SGX-001 |
Primary outcome
Incidence of safety events following a single administration of SGX-001
Time frame: 12 months
Safety and tolerability of a single administration of autologous CD34⁺ HSPCs transduced with a lentiviral vector (SGX-001) in participants with p47-CGD will be assessed based on the incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), SAEs related to SGX-001, adverse events of special interest (AESIs), TEAEs by intensity grade, and discontinuations due to TEAEs, as well as clinical laboratory abnormalities, vital signs, 12-lead ECGs, and physical examination findings. Safety endpoints will be presented overall and/or by study stage or visit, as applicable. Laboratory abnormalities will be graded according to NCI CTCAE version 6.0.
Number of participants with a response to SGX-001 based on NADPH oxidase activity in peripheral blood granulocytes
Time frame: 12 months
Efficacy of a single administration of autologous CD34⁺ HSPCs transduced with a lentiviral vector (SGX-001) in participants with p47-CGD will be assessed based on the proportion of peripheral blood granulocytes with detectable NADPH oxidase activity. Participants will be considered responders if NADPH oxidase activity is present in ≥10% of peripheral blood granulocytes and non-responders if NADPH oxidase activity is present in <10% of peripheral blood granulocytes. The outcome will be summarized as the number and percentage of participants with a response.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
University Children's Hospital Zurich
Zurich, Switzerland
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SGX-001 in people with Chronic Granulomatous Disease (CGD), run by Somagenetix AG at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Germany, Spain, Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of safety events following a single administration of SGX-001 (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.