Studio di fase III su efficacia e sicurezza di NXT007 vs simoctocog alfa (fattore VIII) nella profilassi di persone con emofilia A senza inibitori

Join the study

After giving informed consent, the Patient becomes a participant in the trial.

The Patient receives a copy of the study information and a Schedule of required visits.

Baseline assessments

A series of examinations are performed, including medical history, physical Examination, laboratory tests and quality‑of‑life questionnaires.

Blood samples are taken to Measure baseline plasma levels of the study drugs and to test for anti‑drug antibodies.

Randomization

The Patient is assigned by the study system to one of two treatment arms:

NXT007 (subcutaneous injection) or factor VIII (intravenous administration).

First dose administration

The Patient receives the first dose of the assigned medication under medical supervision.

The dose is recorded as "0 df dosage form" in the protocol; the actual amount is calculated by the Investigator according to body weight and clinical guidelines.

Regular prophylactic dosing

The Patient continues to self‑administer the assigned medication at the frequency determined by the Investigator.

For NXT007 the route is subcutaneous injection; for factor VIII the route is intravenous administration.

Each injection is performed according to the Schedule set in the study protocol and the dose remains individualized throughout the 6‑month treatment period.

Scheduled Clinic visits

The Patient attends Clinic visits at predefined timepoints (e.g., month 1, month 3, month 6 and month 7).

During each visit, the Investigator records the number of bleeds, evaluates safety, collects blood samples for pharmacokinetic analysis and checks for adverse events.

Bleed reporting

The Patient documents any bleeding episode in a diary, noting the date, location and whether treatment WAS required.

Treated bleeds are recorded together with the amount of medication used to stop the bleed.

Safety monitoring

At each visit and whenever needed, the Patient undergoes laboratory tests to monitor Organ function and to detect possible adverse events such as injection‑site reactions, hypersensitivity or thrombotic complications.

The Investigator assesses the Severity of any adverse event according to the National cancer Institute Common Terminology Criteria.

End of 6‑month treatment period

After six months of prophylaxis, the Patient completes the final treatment assessments.

The annualized number of treated bleeds, total bleeds and other efficacy endpoints are calculated.

Final follow‑up

A post‑treatment visit is performed (month 7) to evaluate quality of life, collect final blood samples for plasma concentration of NXT007 and assess the presence of anti‑drug antibodies.

The Patient receives a summary of the study findings and any necessary medical recommendations.