A Phase 1/2 interventional study of SIG-001 in Hemophilia A, sponsored by Eli Lilly and Company. Terminated at 6 sites in 2 countries. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-09-04.
Sponsored by Eli Lilly and Company · Phase 1/2, Interventional, and Treatment
SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A without inhibitors. Up to three dose cohorts (3 patients each) are planned. Cohort expansions (up to 3 additional patients) may be triggered to collect additional information about safety and efficacy.
885 studies on the registry are indexed under Hemophilia A; 139 are open to participants now.
This study's enrollment of 3 is below the median of 27 across 523 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →Eli Lilly and Company is the lead sponsor of 2,095 studies on the registry; 142 are open to participants now.
Of its 535 completed or terminated interventional studies of FDA-regulated products, 356 (67%) have results posted.
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Exclusion Criteria:
Participants received a single dose of 50 milliliter (mL), 78.5 mL and 133 mL of SIG-001 spheres \[an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce B-Domain Deleted Human Factor VIII (BDD-hFVIII) producing Spheres\] administered laparoscopically into the peritoneal cavity.
Combination Product: SIG-001
Laparoscopic administration of SIG-001 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce BDD-hFVIII.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Number of Participants with at least one TEAEs are reported. A summary of other nonserious adverse events (AEs), and all serious adverse events (SAE's), regardless of causality, is located in the Reported Adverse Events section.
Time frame: Baseline Up to 115 Weeks
Number of Participants With Serious Treatment Emergent Adverse Events (TEAEs)
Number of Participants with at least one serious TEAEs are reported. A summary of other nonserious AEs, and all SAE's, regardless of causality, is located in the Reported Adverse Events section.
Time frame: Baseline Up to 115 Weeks
Number of Participants With Inhibitor Titer Values Assessed by Nijmegen Bethesda Assay
Development of FVIII inhibitors is measured using the Nijmegen Bethesda inhibitor assay. The assay measures inhibitors to BDD-FVIII and also other forms of FVIII in the plasma, although rFVIII-BDD was used as a calibrator.
Time frame: Baseline Up to 115 Weeks
Change From Baseline in FVIII Activity Levels Assessed by One-stage and Chromogenic Assays
The change from baseline in FVIII activity, as measured by one-stage and chromogenic assays) is summarized.
Time frame: Baseline Up to 115 Weeks
Number of Bleeding Events [Annualized Bleeding Rate (ABR)] for All Bleeds Following SIG-001 Administration
The annualized number of bleeds per participant (annualized bleeding rate) is calculated as the number of bleeding events divided by length of time on study product follow-up, in years. The duration of assessment for this outcome measure was two years and three months, and Year 3 includes only the three-month part of this study period.
Time frame: Time Frame: Pre-infusion (bleeding events in 12 months prior to sphere placement), 1 year, 2 year and 3-year post-infusion (post sphere placement) from SIG-001 administration annualized up to 115 Weeks.
Total Number of Replacement FVIII Therapies
Number of doses after prophylaxis discontinued is reported.
Time frame: Baseline Up to 115 weeks
| Milestone | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Started | 1 | 1 | 1 |
| Received at least one dose of study drug | 1 | 1 | 1 |
| Completed | 0 | 0 | 0 |
| Not completed | 1 | 1 | 1 |
| Withdrew: Study terminated | 1 | 1 | 1 |
Number of Participants with at least one TEAEs are reported. A summary of other nonserious adverse events (AEs), and all serious adverse events (SAE's), regardless of causality, is located in the Reported Adverse Events section.
| Participants | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 1 | 1 | 1 |
Number of Participants with at least one serious TEAEs are reported. A summary of other nonserious AEs, and all SAE's, regardless of causality, is located in the Reported Adverse Events section.
| participants | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Number of Participants With Serious Treatment Emergent Adverse Events (TEAEs) | 0 | 0 | 1 |
Development of FVIII inhibitors is measured using the Nijmegen Bethesda inhibitor assay. The assay measures inhibitors to BDD-FVIII and also other forms of FVIII in the plasma, although rFVIII-BDD was used as a calibrator.
| participants | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Number of Participants With Inhibitor Titer Values Assessed by Nijmegen Bethesda Assay | 0 | 0 | 1 |
The change from baseline in FVIII activity, as measured by one-stage and chromogenic assays) is summarized.
| International Unit/deciliter (IU/dL) | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Chromogenic Assay | 13.03 ± NA | 223.78 ± NA | -122.71 ± NA |
| One-stage | 15.46 ± NA | 118.28 ± NA | 189.63 ± NA |
The annualized number of bleeds per participant (annualized bleeding rate) is calculated as the number of bleeding events divided by length of time on study product follow-up, in years. The duration of assessment for this outcome measure was two years and three months, and Year 3 includes only the three-month part of this study period.
| Bleeding events per year | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Pre-infusion | 4 | 0 | 4 |
| Year 1 | 7 | 1 | 10 |
| Year 2 | 5 | 0 | 0 |
| Year 3 | 0 | 0 | NA |
Number of doses after prophylaxis discontinued is reported.
| Number of doses | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Total Number of Replacement FVIII Therapies | 401 | 327 | 1558 |
Collected over Baseline Up to 115 Weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1 | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Cohort 2 | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Cohort 3 | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Event | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Factor viii inhibitionBlood and lymphatic system disorders | 0/1 | 0/1 | 1/1 |
| Drug ineffectiveGeneral disorders | 0/1 | 0/1 | 1/1 |
| Anti factor viii antibody increasedInvestigations | 0/1 | 0/1 | 1/1 |
| Event | Cohort 1 | Cohort 2 | Cohort 3 |
|---|---|---|---|
| Abdominal distensionGastrointestinal disorders | 1/1 | 1/1 | 1/1 |
| Abdominal pain upperGastrointestinal disorders | 1/1 | 0/1 | 0/1 |
| Abdominal tendernessGastrointestinal disorders | 0/1 | 1/1 | 0/1 |
| ConstipationGastrointestinal disorders | 1/1 | 1/1 | 0/1 |
| Faeces hardGastrointestinal disorders | 0/1 | 1/1 | 0/1 |
| HaemorrhoidsGastrointestinal disorders | 0/1 | 1/1 | 0/1 |
| Axillary painGeneral disorders | 0/1 | 1/1 | 0/1 |
| Non-cardiac chest painGeneral disorders | 0/1 | 1/1 | 0/1 |
| Peripheral swellingGeneral disorders | 0/1 | 0/1 | 1/1 |
| Vaccination site painGeneral disorders | 0/1 | 1/1 | 0/1 |
All participants who received at least one dose of study drug.
| Age, Categorical(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Total |
|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 1 | 1 | 1 | 3 |
| >=65 years | 0 | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Total |
|---|---|---|---|---|
| Female | 0 | 0 | 0 | 0 |
| Male | 1 | 1 | 1 | 3 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Total |
|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 |
| Not Hispanic or Latino | 1 | 1 | 1 | 3 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 |
| White | 1 | 1 | 1 | 3 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Region of Enrollment(participants) | Cohort 1 | Cohort 2 | Cohort 3 | Total |
|---|---|---|---|---|
| United States | 0 | 0 | 1 | 1 |
| United Kingdom | 1 | 1 | 0 | 2 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
Ran from Sep 2020 to primary completion in Oct 2022, 25 months.
Results were first posted in Sep 2024, 1 year 10 months after primary completion.
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