A study of SGX-001 in patients with chronic granulomatous disease caused by p47phox deficiency (p47-CGD)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Somagenetix AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Decision date (initial)

2026-05-29

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

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

Secondary objectives 15

1. To assess the overall and event-free survival of participants
2. To quantify NADPH oxidase function restoration from baseline as measured by DHR test in participants treated with SGX-001
3. To evaluate the rate and nature of new CGD typic infections in participants treated with SGX-001
4. To evaluate the need for antibacterial prophylaxis, frequency, duration and indication for antibacterial therapy in participants treated with SGX-001
5. To evaluate the need for antifungal prophylaxis, frequency, duration and indication for antifungal therapy in participants treated with SGX-001
6. To evaluate the need for IFN-gamma in participants treated with SGX-001
7. To evaluate the need for systemic immunosuppression in participants treated with SGX-001
8. To evaluate the inflammatory diseases, including IBD, in participants treated with SGX-001
9. To evaluate the recovery from pre-existing infections in participants treated with SGX-001
10. To evaluate the effect of treatment with SGX-001 on HRQoL
11. To assess the engraftment in participants treated with SGX-001
12. Neutrophil recovery in participants treated with SGX-001
13. Platelet recovery in participants treated with SGX-001
14. To assess the transgene integration profile
15. To assess the incidence of SGX-001-related RCL detection

Conditions and MedDRA coding

Chronic granulomatous disease (CGD) caused by p47phox deficiency (p47-CGD)

Regulatory references

Scientific advice from competent authorities

Spanish Agency Of Medicines And Medical Devices, European Medicines Agency, Swissmedic Swiss Agency for Therapeutic Products, U.S. Food And Drug Administration, Paul-Ehrlich-Institut

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. 01 Properly completed and signed informed consent or assent (participant/LAR)
2. 02 Confirmed diagnosis of CGD due to p47phox deficiency (confirmed mutation in the NCF1 gene by molecular genetic testing).
3. 03 Absent or > 95% reduced biochemical activity of NADPH oxidase in a dihydrorhodamine flow cytometric test.
4. 04 Male or female aged ≥ 18 months and have a body weight ≥ 10 kg the time of signing the informed consent or assent.
5. 05 One or more ongoing or recurrent severe infectious and/or inflammatory complications, at the discretion of the Investigator.
6. 06 Lack of an available 10/10 HLA-matched (A, B, C, DR, DQ) sibling donor suitable for HSCT
7. 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.
8. 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.
9. 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.
10. 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.

Exclusion criteria 12

1. 01 Participant or parent/legal guardian is unable or unwilling to comply with the protocol requirements
2. 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.
3. 03 Previous allogeneic HSCT.
4. 04 Pregnancy or lactation.
5. 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.
6. 06 Contraindication for administration of XXX, or any component of the study intervention
7. 07 Concomitant human immunodeficiency virus (HIV1 or HIV2), hepatitis B virus, hepatitis C virus, adenovirus, parvovirus B19, human T-lymphotropic virus, or toxoplasmosis infection.
8. 08 Evidence of active metastatic or locoregionally advanced malignancy (including haematologic malignancy) for which survival is anticipated to be less than 3 years.
9. 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.
10. 10 Inability to stop using interferon-gamma at least 30 days prior to administration of the study intervention.
11. 11 Participation in another interventional clinical study within 6 months prior to enrolment.
12. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Safety endpoints until Month 12 presented overall and/or by study stage or visit (as applicable), including: • TEAEs • SAEs • SAEs related to SGX-001 • AESIs • TEAEs by intensity grade • Discontinuations due to TEAEs • Laboratory assessments • Vital signs • 12 lead ECGs • Physical examinations
2. Participants with response to the treatment with SGX-001 (defined as NADPH oxidase activity in ≥ 10% of peripheral blood granulocytes) at Month 12

Secondary endpoints 27

1. Overall survival at Month 12
2. % ox-DHR+ in peripheral blood granulocytes and change in stimulation index from screening to Month 12
3. Number and type of new CGD-typic infections to Month 12
4. Number of participants off antibacterial prophylaxis at Month 12
5. Number of participants on antibacterial therapy in participants treated with SGX 001
6. Duration of antibacterial therapy in participants treated with SGX-001
7. Indication for antibacterial therapy in participants treated with SGX-001
8. Number of participants off antifungal prophylaxis at Month 12
9. Number of participants on antifungal therapy in participants treated with SGX 001
10. Duration of antifungal therapy in participants treated with SGX 001
11. Indication for antifungal therapy in participants treated with SGX-001
12. Number of participants not requiring treatment with IFN gamma at Month 12
13. Number of participants not requiring treatment with systemic immunosuppression at Month 12
14. Change from baseline in the presence or severity of IBD
15. Change from baseline to Month 12 in the presence or severity of inflammatory manifestations
16. Number of participants recovered from pre-existing active infection at Month 12
17. Number of participants with improved HRQoL at Month 12
18. Incidence of primary graft failure
19. Incidence of secondary graft failure
20. Transgene level in bone marrow cells at Months 6 and 12
21. Transgene level in peripheral blood cells at Months 1, 2, 3, 6, 9, and 12
22. Time to neutrophil recovery
23. Incidence of neutrophil recovery by D 42 after SGX 001 infusion
24. Time to platelet recovery
25. Incidence of platelet recovery at Month 12
26. Incidence of clonal dominance events in blood and bone marrow at Months 6 and 12
27. Incidence of detectable RCL at Months 3, 6, and 12

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Somagenetix AG

Sponsor organisation

Somagenetix AG

Address

Bahnhofstrasse 1

City

Altendorf

Postcode

8852

Country

Switzerland

Scientific contact point

Organisation

Somagenetix AG

Contact name

Oleksandr Pastukhov

Public contact point

Organisation

Somagenetix AG

Contact name

Oleksandr Pastukhov

Third parties 3

Locations

2 EU/EEA countries · 2 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 24 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications