This is an open-label study with single- and multiple-ascending dose arms followed by a dose expansion arm. The primary objective of the study is to determine the safety and tolerability of CRMA-1001 in adult participants with Chronic Hepatitis B. In addition, the pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of CRMA-1001 will be evaluated. CRMA-1001 is an epigenetic gene therapy delivered via intravenous (IV) infusion. Up to four dose levels will be tested. Participants will receive a single or multiple doses of CRMA-1001 and will remain on antiviral therapy during the dosing process.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
66
Epigenetic gene silencing therapy delivered by intravenous (IV) infusion
Queen Mary Hospital, The University of Hong Kong
Hong Kong, Hong Kong
RECRUITINGNew Zealand Clinical Research
Auckland, New Zealand
RECRUITINGSafety and tolerability of single and multiple doses of CRMA-1001
Incidence and severity of treatment-emergent adverse events
Time frame: 6 months
Long-term safety of single and multiple doses of CRMA-1001
Incidence and severity of treatment emergent adverse events
Time frame: 60 Months
Pharmacokinetics of CRMA-1001 components (Cmax)
Maximum concentration (Cmax) in plasma
Time frame: 6 Months
Pharmacokinetics of CRMA-1001 components (Tmax)
Time of maximum concentration (Tmax) in plasma
Time frame: 6 Months
Pharmacokinetics of CRMA-1001 components (terminal clearance rate)
Clearance rate in terminal clearance phase (CL) in plasma
Time frame: 6 Months
Pharmacokinetics of CRMA-1001 components (Vd)
Volume of distribution (Vd) in plasma
Time frame: 6 Months
To evaluate the immunogenicity of CRMA-1001
Incidence and characterization of anti-drug antibodies
Time frame: 6 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBsAg)
Change from baseline in HBsAg
Time frame: 6 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBs)
Change from baseline in anti-HBs antibody titier
Time frame: 6 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBV DNA)
Change from baseline in HBV DNA
Time frame: 6 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBeAg)
Change from baseline in HBeAg
Time frame: 6 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBe)
Change from baseline in anti-HBe antibody titer
Time frame: 6 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBsAg)
Change from baseline in HBsAg
Time frame: 60 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBs)
Change from baseline in anti-HBs antibody titer
Time frame: 60 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBV DNA)
Change from baseline in HBV DNA
Time frame: 60 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (HBeAg)
Change from baseline in HBeAg
Time frame: 60 Months
To evaluate the effect of CRMA-1001 on circulating HBV biomarkers (anti-HBe)
Change from baseline in anti-HBe antibody titer
Time frame: 60 Months
To evaluate the rate of antiviral therapy discontinuation after treatment with CRMA-1001
Proportion of participants able to discontinue antiviral therapy
Time frame: 60 Months
To evaluate the effect of CRMA-1001 on the incidence of functional cure
Incidence of functional cure
Time frame: 60 Months
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