Phase 2
Completed N=15
A Gene Therapy Study for Hemophilia B
Source: ClinicalTrials.gov NCT02484092 ↗Enrolled (actual)
15
Serious AEs
0.0%
Results posted
May 2020
Primary outcomePrimary: Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings — 0 Participants
Summary
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings |
— | — |
| PRIMARY Number of Participants With Clinically Significant Change From Baseline in Vital Signs |
— | — |
| PRIMARY Number of Participants With Clinical Laboratory Abnormalities Reported as TEAE |
2 | — |
| PRIMARY Number of Participants With Drug -Related TEAEs and Serious Adverse Events (SAEs) |
2; 0 | — |
| PRIMARY Number of Participants With Positive Immune Reponses Against Adeno-associated Virus Vector (AAV) Capsid |
2 | — |
| PRIMARY Number of Participants Who Reached > 150% Vector-derived FIX:C Activity Level After SPK-9001 Infusion |
— | — |
| PRIMARY Number of Participants With FIX Inhibitor |
— | — |
| PRIMARY Incremental Recovery of FIX Product |
0.0100; 0.0162 | — |
| SECONDARY FIX:C Activity |
22.9; 29.1 | — |
| SECONDARY Change From Baseline in FIX:C Antigen Level at Steady State |
-4.4; -4.7; -4.7; -5.8; -4.6; -6.6 | — |
Eligibility Criteria
Inclusion Criteria
- Able to provide informed consent and comply with requirements of the study
- Males ≥18 y.o. with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2% endogenous factor IX)
- Received ≥50 exposure days to factor IX products
- A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions
- No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein
- Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences
Exclusion Criteria
- Evidence of active hepatitis B or C
- Currently on antiviral therapy for hepatitis B or C
- Have significant underlying liver disease
- Have serological evidence* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (* subjects who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll)
- Neutralizing antibodies reactive with AAV-Spark100 above and/or below a defined titre
- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 12 weeks
- Unable or unwilling to comply with study assessments
Data sourced from ClinicalTrials.gov (NCT02484092). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.