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NCT Number: NCT07212426

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

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).

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This study is active but is not currently recruiting participants.

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Key information

Conditions

PNH - Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The First Affiliated Hospital of Nanjing Medical University

Nanjing, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

LP-005 Injection

Biological

IV infusion, Q4W

Primary outcomes

  1. Change from Baseline in serum lactate dehydrogenase (LDH) levels.

    Time frame: Baseline and at Week 12.

  2. 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.

Secondary outcomes

  1. 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

  2. Proportion of participants who are transfusion-free.

    Time frame: Baseline to Week 12, 24 and 48

  3. Change from Baseline in the number of red blood cell (RBC) transfusions.

    Time frame: Baseline to Week 24 and 48.

  4. Change from Baseline in hemoglobin levels.

    Time frame: Baseline, Week 12, 24 and 48

  5. Proportion of participants achieving hemoglobin levels ≥100 g/L in the absence of transfusion.

    Time frame: Baseline, Week 12, 24 and 48

  6. Proportion of participants with LDH levels normalized.

    Time frame: Baseline, Week 12, 24 and 48

  7. Changes in serum complement hemolytic activity.

    Time frame: Observation from Predose on Day 1 through 337 days post-administration.

  8. 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.

  9. Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).

    Time frame: Baseline up to approximately 13 months.

  10. Serum concentrations of LP-005.

    Time frame: Observation from Predose on Day 1 through 337 days post-administration.

  11. 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.

Sponsors and collaborators

Lead sponsor

Longbio Pharma

Industry

Registry information

Official study title

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.

Important dates

Study start
2024
Primary completion
2026
Study completion
2028
First posted
Oct 8, 2025
Registry last updated
Jan 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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