NCT06848296

Brief Summary

The complement system is an important component of the innate immune system. Abnormal activation, inadequate regulation and control of the complement system, as well as impaired and dysfunctional effector functions, underlie complement mediated diseases including PNH. VSA012 targeting complement system has the potential to treat a variety of diseases associated with abnormal activation of the complement system.The purpose of VSA012-1002 is to evaluate the safety, tolerability, pharmacokinetic, pharmacodynamics and efficacy of VSA012 Injection in subjects with PNH.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P50-P75 for phase_1

Timeline
16mo left

Started Apr 2025

Typical duration for phase_1

Geographic Reach
1 country

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress45%
Apr 2025Aug 2027

First Submitted

Initial submission to the registry

February 17, 2025

Completed
10 days until next milestone

First Posted

Study publicly available on registry

February 27, 2025

Completed
2 months until next milestone

Study Start

First participant enrolled

April 15, 2025

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 28, 2027

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2027

Last Updated

May 4, 2026

Status Verified

April 1, 2026

Enrollment Period

1.9 years

First QC Date

February 17, 2025

Last Update Submit

April 28, 2026

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Outcome Measures

Primary Outcomes (2)

  • Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)

    up to Day 540

  • preliminary efficacy

    Percentage change from baseline in lactate dehydrogenase (LDH) by visit Change from baseline in hemoglobin (Hb) level by visit

    up to Day 540

Secondary Outcomes (11)

  • Pharmacokinetics (PK) of VSA012 (First dose): Maximum Observed Plasma Concentration (Cmax)

    Up to 48 hours post-dose

  • PK of VSA012(First dose): Time to Maximum Observed Plasma Concentration (Tmax)

    Up to 48 hours post-dose

  • PK of VSA012(First dose)：Area under the concentration-time curve during the dosing interval (AUC 0-tau)

    Up to 48 hours post-dose

  • PK of VSA012 (Multiple dose)：Trough concentration (C min)

    Up to 48 hours post-dose

  • PK of VSA012 (Multiple dose)：Accumulation ratio of C max

    Up to 48 hours post-dose

  • +6 more secondary outcomes

Study Arms (5)

VSA012 dose A

EXPERIMENTAL
Drug: VSA012

VSA012 dose B

EXPERIMENTAL
Drug: VSA012

VSA012 dose C

EXPERIMENTAL
Drug: VSA012

VSA012 dose D

EXPERIMENTAL
Drug: VSA012

VSA012 dose E

EXPERIMENTAL
Drug: VSA012

Interventions

VSA012DRUG

VSA012 injection

VSA012 dose AVSA012 dose BVSA012 dose CVSA012 dose DVSA012 dose E

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may not qualify if:

  • Participants voluntarily participate in this clinical study, and voluntarily sign the ICF;
  • BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age;
  • Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%;
  • Presence of one or more of PNH-related signs or symptoms within 3 months prior to screening;
  • Hb \< 100 g/L;
  • LDH value \> 1.5 × ULN;
  • One of the following criteria for prior drug therapy for PNH must be met:
  • Having never received any complement inhibitor therapy;
  • Having received C5, C3, or CFB complement inhibitors and having discontinued the complement inhibitor for more than 5 half-lives or 3 months prior to screening;
  • Participants are willing to receive meningococcal vaccine and pneumococcal vaccine at least 14 days prior to dosing.
  • Participants voluntarily participate in this clinical study, and voluntarily sign the ICF;
  • BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age;
  • Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%;
  • Participants who have been on a stable dose and interval of a C5 complement inhibitor (approved locally) for at least 3 months prior to the first dose of VSA012;
  • Within the 3 months prior to screening, have a documented Hb level of \< 105 g/L while on C5 complement inhibitor therapy;
  • +24 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Location

The First Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Officials

  • Bing Han

    Peking Union Medical College Hospital

    PRINCIPAL INVESTIGATOR

  • Hongyan Tong

    Zhejiang University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 17, 2025

First Posted

February 27, 2025

Study Start

April 15, 2025

Primary Completion (Estimated)

February 28, 2027

Study Completion (Estimated)

August 31, 2027

Last Updated

May 4, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

CN(2)