NCT05731050

Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase II, Open Label, Multi Dose Study of NM8074 in Soliris-Treated Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: NovelMed Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects.

Detailed description

The proposed study, NM8074-PNH-101, will evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects. The drug will be administered by intravenous (IV) infusion to adult patients who qualify based on the inclusion/exclusion criteria. A total of at least 6 patients (maximum of 10) with documented PNH and at least 3 months of Soliris treatment prior to Screening will be enrolled at the study site. The total duration of study term participation for all subjects will be up to 22 weeks, including a screening period of up to 8 weeks, a 12-week treatment period, and 2 weeks of a washout period. All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period.

Conditions

• PNH - Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
DRUG	NM8074	NM8074 will be administered as an intravenous infusion. All enrolled subjects will receive a dose of 15 mg/kg NM8074 once every two weeks for a total of 6 doses from Day1 to Day 84. For Soliris-treated PNH subjects, admission and dosing of NM8074 on Day 1 should be scheduled to coincide with the next scheduled dose of Soliris (i.e., 14 ± 2 days after the last Soliris dose). Soliris should NOT be administered on Day 1 or at any time thereafter during course of the study. Subjects in the treatment groups will receive a dose of 15 mg/kg of NM8074 on Day 1, which will be administered during the study visit by the site staff. During the remainder of the treatment period, subjects will be given another 15 mg/kg dose every two weeks at approximately the same time. On days of scheduled study visits at the Investigation Site, the dose must be administered after blood work is completed.

Timeline

• Start date: 2026-06-01
• Primary completion: 2027-09-01
• Completion: 2028-03-01
• First posted: 2023-02-16
• Last updated: 2025-03-30

Regulatory

• FDA-regulated drug study