Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients.

January 14, 2026 updated by: Longbio Pharma

A Phase II, Multicenter, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria.

This is a multicenter, randomized, open-label, Phase II clinical study to evaluate the efficacy, safety, and pharmacokinetics of LP-005 injection in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Nanjing, China
        • The First Affiliated Hospital of Nanjing Medical University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male or female, aged 18 to 65 years.
  • Diagnosis of PNH based on flow cytometry with clone size > 10% by granulocytes.
  • Presence of one or more PNH-related signs or symptoms within 3 months prior to screening or a history of transfusion due to PNH.
  • LDH level ≥2.0×upper limit of the normal range(ULN).
  • Hemoglobin level <100 g/L at screening.

Exclusion Criteria:

  • Active or suspected active viral, bacterial, fungal, or parasitic infection within 14 days prior to screening.
  • History of meningococcal infection.
  • History of splenectomy or congenital asplenia.
  • History of systemic autoimmune disease or known/suspected immunodeficiency.
  • History of hematopoietic stem cell transplantation.
  • Use of any complement inhibitor within 3 months prior to screening or within 5 drug half-lives (whichever is longer).
  • Pregnant or breastfeeding women, or women planning to become pregnant during the study or follow-up period.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: LP-005 1200 mg
Patients will receive intravenous infusion of LP-005 Injection at a dose of 1200 mg once every 4 weeks.
Biological: LP-005 Injection
IV infusion, Q4W
Experimental: LP-005 1500 mg
Patients will receive intravenous infusion of LP-005 Injection at a dose of 1500 mg once every 4 weeks.
Biological: LP-005 Injection
IV infusion, Q4W
Experimental: LP-005 900 mg
Patients will receive intravenous infusion of LP-005 Injection at a dose of 900 mg once every 4 weeks.
Biological: LP-005 Injection
IV infusion, Q4W

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change from Baseline in serum lactate dehydrogenase (LDH) levels.
Time Frame: Baseline and at Week 12.
Baseline and at Week 12.
Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.
Time Frame: Baseline and at Week 24.
Baseline and at Week 24.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Changes in serum complement hemolytic activity.
Time Frame: Observation from Predose on Day 1 through 337 days post-administration.
Observation from Predose on Day 1 through 337 days post-administration.
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Time Frame: Baseline up to approximately 13 months.
Baseline up to approximately 13 months.
Serum concentrations of LP-005.
Time Frame: Observation from Predose on Day 1 through 337 days post-administration.
Observation from Predose on Day 1 through 337 days post-administration.
Number of Participants with Treatment-emergent Anti-Drug Antibodies (ADA) and neutralizing antibodies (Nab) Response to LP-005.
Time Frame: Observation from Predose on Day 1 through 337 days post-administration.
Observation from Predose on Day 1 through 337 days post-administration.
Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.
Time Frame: Baseline and at Week 12 and 48
Baseline and at Week 12 and 48
Proportion of participants who are transfusion-free.
Time Frame: Baseline to Week 12, 24 and 48
Baseline to Week 12, 24 and 48
Change from Baseline in the number of red blood cell (RBC) transfusions.
Time Frame: Baseline to Week 24 and 48.
Baseline to Week 24 and 48.
Change from Baseline in hemoglobin levels.
Time Frame: Baseline, Week 12, 24 and 48
Baseline, Week 12, 24 and 48
Proportion of participants achieving hemoglobin levels ≥100 g/L in the absence of transfusion.
Time Frame: Baseline, Week 12, 24 and 48
Baseline, Week 12, 24 and 48
Proportion of participants with LDH levels normalized.
Time Frame: Baseline, Week 12, 24 and 48
Baseline, Week 12, 24 and 48
Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F)
Time Frame: Baseline and at Week 12, 36, and 48.
FACIT-F is a 40-item measure that assesses self-reported fatigue and its impact upon daily activities and function. The higher the score, the better the QOL.
Baseline and at Week 12, 36, and 48.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Longbio Pharma

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Guangsheng He, The First Affiliated Hospital with Nanjing Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 14, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 6, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 31, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 30, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 30, 2025

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 8, 2025

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 16, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 14, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • P10-LP005-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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