A phase 2a, multicentre trial to evaluate the efficacy, safety and pharmacokinetics of RBD1016 in participants with chronic hepatitis D virus infection, including a randomized, single-blinded, placebo-controlled exploratory part

Phase 1

• Conditions: Chronic hepatitis D virus infection; MedDRA version: 20.1Level: PTClassification code: 10019762Term: Hepatitis D Class: 100000004862; Therapeutic area: Diseases [C] - Virus Diseases [C02]

• Registration Number: CTIS2023-509007-33-00
• Lead Sponsor: Ribocure Pharmaceuticals AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-12-20
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Willing and able to give written informed consent for participation in the trial., Female participants of childbearing potential must also be willing to practice abstinence from heterosexual intercourse (only allowed when this is the preferred and usual lifestyle of the participant) or be willing to use a highly effective method of contraception (i.e., with a failure rate of <1%/year) to prevent pregnancy from at least 2 weeks prior to the first administration of IMP to 4 weeks after the last administration of IMP. The following are considered highly effective contraceptive methods: • Combined (oestrogen and progestogen-containing) or progestogen-only hormonal contraception associated with the inhibition of ovulation (oral, transdermal, intravaginal, injectable, or implantable). • Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS). Female participants of non-childbearing potential are defined as pre-menopausal females who have undergone any of the following surgical procedures; hysterectomy, bilateral salpingectomy or bilateral oophorectomy, or who are post-menopausal defined as 12 months of amenorrhoea (in questionable cases a blood sample with detection of follicle stimulating hormone [FSH] >25 IU/mL will be confirmatory). Male participants must be willing, unless they have undergone vasectomy, to practice sexual abstinence from heterosexual intercourse (only allowed when this is the preferred and usual lifestyle of the participant) or use condoms from the first administration of IMP and until 3 months after the last administration of IMP to prevent pregnancy and drug exposure of a female partner., Male or female participant aged 18 to 65 years, inclusive., Body mass index (BMI) = 18 and = 35 kg/m2 at the time of the screening visit., Documented evidence of HDV infection in medical history, i.e., HDV antibodies (HDVAb) and/or HDV RNA positive test results within at least 6 months prior to screening., Documented evidence of HBV infection in medical history, i.e., HBsAg and/or HBV DNA positive test results within at least 6 months prior to screening., Documented absence of liver cirrhosis, defined as an LSM = 10 kPa measured on FibroScan® elastography at screening., Female participants of childbearing potential only: Negative pregnancy test (urine dip-stick) at screening and upon confirmation of eligibility. If urine pregnancy tests are positive, blood/serum pregnancy test will be confirmatory.

Exclusion Criteria

Laboratory results at screening as follows, or any clinically significant laboratory parameter outliers that may interfere with the evaluation of efficacy and/or safety in the trial, at the discretion of the Investigator: • a-fetoprotein (AFP) > 50 µg/L. • Albumin concentration < 3.0 g/dL. • International normalized ratio (INR) > 1.5. • Platelet count < 90 × 109/L. • Direct bilirubin > 2 × ULN, Gilbert syndrome excluded. • Creatinine concentration > 1.5 × ULN. • Creatinine clearance < 60 mL/min, according to the Cockcroft-Gault equation. • ALT > 5 × ULN (see inclusion criterion no. 7)., Active clinically significant disease or disorder other than liver disease which, in the opinion of the Investigator, may either put the participant at risk because of participation in the trial, or influence the results or the participant’s ability to participate in the trial, e.g., any uncontrolled renal, cardiovascular, pulmonary, thyroid, neurogenic, digestive, endocrine and/or metabolic disease or disorder, at the discretion of the Investigator., Active severe mental illness or uncontrolled mental disorders, e.g., schizophrenia, bipolar disorder or depression, which make the participant unsuited for trial participation in the opinion of the Investigator., Major surgery within 6 months prior to screening., Clinically significant infection (i.e., that required treatment with antibiotics), trauma and/or medical/surgical procedure within 4 weeks of the (first) administration of IMP., Planned surgical procedure(s) during the course of the trial, i.e., from the screening visit to the follow-up/end-of-trial visit., Participants who are pregnant, currently breastfeeding, or intend to become pregnant during the course of the trial., History of or active severe allergy/hypersensitivity (i.e., allergic/hypersensitive to several drug substances, foods and/or other allergens), as judged by the Investigator and/or history of hypersensitivity to drugs with a similar chemical structure or class to RBD1016, NAs, or any of their preparation ingredients., History of severe hypersensitivity to subcutaneous injections. History of mild reactions, such as localised swelling or redness, is allowed., Use of pegylated interferon alpha (PegIFNa) and/or immunomodulators (e.g., thymosin, interleukin-2, levamisole) and/or systemic corticosteroids and/or cytotoxic drugs, within 6 months prior to screening, or planned treatment with such drugs during the course of the trial, i.e., from the screening visit to the follow-up/end-of-trial visit. See also Section 9.6.2.2., Planned treatment or treatment with another investigational drug within 1 month prior to the first IMP administration, or within 5 half-lives of the other investigational drug, whichever is longer. Participants consented and screened but not dosed in previous clinical trials will not be excluded., Positive result at screening for hepatitis C virus (HCV) and/or human immunodeficiency virus (HIV) and/or prior diagnosis of syphilis, acute hepatitis A and/or acute hepatitis E., Previous participation in clinical trials of similar anti-HBV siRNA or antisense oligonucleotide drugs within 6 months prior to screening., Positive screening result for alcohol during the trial. Positive results that are expected given the participant’s medical history and prescribed medications can be disregarded as judged by the Investigator after conferring with the Sponsor., Presence or history of drug abuse within 6 months prior to screening

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified