Phase 1 Open‑Label Dose‑Escalation Study of Inno8 in Adults and Adolescents with Haemophilia A, With or Without Inhibitors, Assessing Pharmacokinetics and Safety

The primary objective is to characterize the pharmacokinetic profile and assess dose proportionality of Inno8 in individuals with Hemophilia A, thereby providing the pharmacologic basis for optimal prophylactic dosing. Secondary objectives include:

• Evaluation of safety and tolerability across the dose range.
• Quantification of annualized bleeding rates to determine clinical efficacy.
• Monitoring of factor VIII activity and related biomarkers.
• Assessment of inhibitor development.
• Measurement of health‑related quality of life using validated instruments.

The trial enrolled a total of 4 participants, all of whom were male patients diagnosed with Haemophilia A and meeting the study’s indication for prevention of bleeding and long‑term prophylaxis irrespective of inhibitor status or severity. Subjects were selected based on the defined age range corresponding to code 3 and were required to be in generally stable health; no specific dietary, physical activity, or other lifestyle restrictions were stipulated in the provided information. Inclusion criteria emphasized male gender, confirmed diagnosis of the target condition, and classification as a patient; further exclusion parameters were not disclosed.

The trial is a phase 3, category 1, randomized, double‑blind, placebo‑controlled study evaluating multiple dose levels of Inno8 in participants diagnosed with haemophilia A (with or without inhibitors). Recruitment is planned from October 2025 to May 2026. After an initial screening visit to confirm eligibility, subjects are randomized to a dose cohort and begin the treatment period. Scheduled study visits occur at weeks 2, 4, 8, 12, 24, and 36 for pharmacokinetic sampling, safety monitoring, and efficacy assessment, followed by an end‑of‑study visit at week 48. Participant involvement lasts approximately 12 months, including the screening phase. Early termination may be required for serious treatment‑related adverse events, withdrawal of consent, or non‑compliance with protocol‑defined visit windows.

Efficacy will be evaluated in accordance with predefined endpoints specified in the Phase 3 study protocol for the investigational therapy in patients with haemophilia A, regardless of inhibitor status or disease severity. Assessments are scheduled at designated time points during the treatment period and will be analyzed using appropriate statistical methods.