E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10008910 |
E.1.2 | Term | Chronic hepatitis B |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
• To evaluate the single-dose pharmacokinetics of LDT600 in pediatric and adolescent patients (2-18 years of age) with chronic hepatitis B (CHB) infection • To evaluate the safety and tolerability of LDT600 in pediatric and adolescent patients with CHB infection
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1 Patients or legal guardian must have read and signed the written informed consent form (ICF) after the nature of the study has been fully explained and all questions answered. 2 Patients must be between the ages of 2 to 18 years with body weight within 15th to 85th percentile of normal relative to age, based on either the Centers for Disease Control and Prevention (CDC) growth chart (Appendix 1) or national health service norms. 3 The patient must have documented chronic HBV (CHB) infection with positive HBsAg assay at Screening. 4 Patients must have a screening creatinine clearance (CLcr) ≥ 80 mL/min/1.73 m2 as estimated by the Schwartz formula (See Section 8.2.2.1). 5 All female patients of reproductive potential must have negative serum pregnancy test at Screening and negative urine pregnancy test on Day –1. 6 All patients of reproductive potential must agree to use appropriate double barrier method of birth control or agree to abstain from intercourse from Day –1 through Day 30 after dosing. 7 Patients must agree not to take any other medications during the course of the study, without the approval of the Investigator, who in consultation with the Sponsor, may permit its use, on a case-by-case basis, when it is judged not to jeopardize the patient’s safety or interfere with study endpoints. 8 Patients must agree not to consume alcohol within two days of reporting to the clinic on Day –1. |
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E.4 | Principal exclusion criteria |
1 Decompensated liver disease (Child-Turcotte-Pugh (CTP) Score≥7, Class B and C) 2 Other clinically significant disease, condition or abnormality, unrelated to their HBV infection, as assessed by the Investigator, based on medical history, physical examination, 12-lead electrocardiogram (ECG), or clinical laboratory testing, including: • Hemoglobin value <110 g/L (11.0 g/dL) for males and <100 g/L (10.0 g/dL) for females • Absolute neutrophil count (ANC) (<1,500/ x 10E3) (<1.5x10E9) • Platelet count (< 120,000/ x10E3) (<120 x10E9) • White blood cell count (WBC) (<3,000/ x10E3) (3.0 x10E9) • Prothrombin time/INR prolonged by more than 3 seconds, (based on the Upper Limit Normal [ULN]; of the reference value) • Serum amylase or lipase ≥1.5 x ULN; • Serum albumin (<3.5 g/dL); (<35 g/L) • Total bilirubin (≥2.0 mg/dL or ≥34.2 µmol/L); • Serum ALT level >10 x ULN. • Blood urea nitrogen (BUN) greater than the upper limit of normal. 3 Participation in a clinical drug study within 30 days of Screening. 4 Treatment with interferon within six months of Screening. 5 Treatment with nucleoside/nucleotide reverse transcriptase inhibitors within three months of Screening. 6 Treatment with any other anti-HBV drugs or other antiviral therapy (e.g., acyclovir ganciclovir) within 30 days of study drug dosing. 7 Treatment with antibiotics within 7 days of Screening. 8 Females who are pregnant or are breast-feeding. 9 Patients who are currently abusing alcohol or illicit drugs, or have a history of such abuse within the preceding two years. For the purposes of the present study, alcohol abuse is arbitrarily defined as frequent consumption of alcoholic beverages with an average daily intake of more than 40 g of alcohol. |
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E.5 End points |
E.5.1 | Primary end point(s) |
• LDT600 plasma concentration and pharmacokinetic (PK) parameters of exposure (Cmax and AUC) • Incidence of adverse events (AEs) and serious adverse events (SAEs).
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
First administration in the paediatric population |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |