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Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Phase 1, PHASE2 trial testing SB-318 in MPS I in 3 participants. Terminated before completion.
| Lead sponsor | Sangamo Therapeutics |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | sequential |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 3 |
| Start date | 24 May 2017 |
| Primary completion | 3 November 2021 |
| Estimated completion | 3 November 2021 |
| Sites | 1 location across United States |
Sangamo Therapeutics — full company profile →
5 and older, any sex, with MPS I. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants with Treatment-Emergent Adverse Events
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg | 1 | |
| Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg | 2 | |
| Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg | 0 |
Change from baseline clinical laboratory in measurement of IDUA activity measured in leukocytes.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg | -1.200 | ± NA |
| Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg | -2.515 | ± 5.9185 |
Change from baseline in total GAG, Dermatan Sulfate GAG, and Heparan Sulfate GAG measured in urine at Month 24
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg | 0.510 | ± NA |
| Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg | -0.090 | ± 0.3960 |
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg | 30.2000 | ± NA |
| Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg | 3.6005 | ± 8.22436 |
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg | 0.940 | ± NA |
| Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg | 0.240 | ± 0.0424 |
Subjects with AAV2/6 clearance in plasma, saliva, urine, stool, and semen by PCR by Week 24.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg | 1 | |
| Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg | 2 | |
| Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg | 0 |
Time frame: Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Cohort 1: SB-318: Starting… | Cohort 2: SB-318 at Next A… | Cohort 3: SB-318 at Next A… |
|---|---|---|---|---|
| Atrial flutter | Cardiac disorders | — | — | — |
| Adenovirus infection | Infections and infestations | — | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — | — |
| Haemoglobin decreased | Investigations | — | — | — |
| Reaction | System | Cohort 1: SB-318: Starting… | Cohort 2: SB-318 at Next A… | Cohort 3: SB-318 at Next A… |
|---|---|---|---|---|
| Cardiac failure | Cardiac disorders | — | — | — |
| Blood cortisol decreased | Investigations | — | — | — |
| Acne | Skin and subcutaneous tissue disorders | — | — | — |
| Upper Respiratory Infection | Infections and infestations | — | — | — |
| Aortic valve stenosis | Cardiac disorders | — | — | — |
| Atrial fibrillation | Cardiac disorders | — | — | — |
| Left atrial enlargement | Cardiac disorders | — | — | — |
| Oedema peripheral | General disorders | — | — | — |
| Bronchitis | Infections and infestations | — | — | — |
| Conjunctivitis | Infections and infestations | — | — | — |
| Excoriation | Injury, poisoning and procedural complications | — | — | — |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | — | — | — |
| Scoliosis | Musculoskeletal and connective tissue disorders | — | — | — |
| Dizziness | Nervous system disorders | — | — | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Tracheal stenosis | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Contusion | Injury, poisoning and procedural complications | — | — | — |
| Headache | Nervous system disorders | — | — | — |
| Renal Cyst | Renal and urinary disorders | — | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — | — |
Most-reported serious reactions: Atrial flutter, Adenovirus infection, Upper respiratory tract infection, Haemoglobin decreased.
Data from ClinicalTrials.gov NCT02702115 adverse events section.
The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the α-L-iduronidase (IDUA) gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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