PNH - Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT07212426
Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients.
A Phase 2 study testing LP-005 Injection in people with PNH - Paroxysmal Nocturnal Hemoglobinuria, run by Longbio Pharma at 1 site in China.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- PNH - Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- LP-005 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 14 Nov 2024to 31 Mar 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official 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.
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).
- Being tested
- LP-005 Injection
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LP-005 InjectionBiological
IV infusion, Q4W
| Study group | Role | What they receive |
|---|---|---|
| LP-005 900 mg | Experimental | Patients will receive intravenous infusion of LP-005 Injection at a dose of 900 mg once every 4 weeks. Receives: Biological: LP-005 Injection |
| LP-005 1200 mg | Experimental | Patients will receive intravenous infusion of LP-005 Injection at a dose of 1200 mg once every 4 weeks. Receives: Biological: LP-005 Injection |
| LP-005 1500 mg | Experimental | Patients will receive intravenous infusion of LP-005 Injection at a dose of 1500 mg once every 4 weeks. Receives: Biological: LP-005 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in serum lactate dehydrogenase (LDH) levels.
Measured: Baseline and at Week 12.
Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.
Measured: Baseline and at Week 24.
Secondary outcomes (11)
Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion.
Measured: Baseline and at Week 12 and 48
Proportion of participants who are transfusion-free.
Measured: Baseline to Week 12, 24 and 48
Change from Baseline in the number of red blood cell (RBC) transfusions.
Measured: Baseline to Week 24 and 48.
Change from Baseline in hemoglobin levels.
Measured: Baseline, Week 12, 24 and 48
Proportion of participants achieving hemoglobin levels ≥100 g/L in the absence of transfusion.
Measured: Baseline, Week 12, 24 and 48
Proportion of participants with LDH levels normalized.
Measured: Baseline, Week 12, 24 and 48
Changes in serum complement hemolytic activity.
Measured: Observation from Predose on Day 1 through 337 days post-administration.
Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F)
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.
Measured: Baseline and at Week 12, 36, and 48.
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Measured: Baseline up to approximately 13 months.
Serum concentrations of LP-005.
Measured: 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.
Measured: Observation from Predose on Day 1 through 337 days post-administration.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2025
- Study start14 Nov 2024
- Primary completion6 May 2026 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PNH - Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Longbio Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Guangsheng He, Principal investigator, The First Affiliated Hospital with Nanjing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07212426
- Sponsor's protocol ID
- P10-LP005-02
- Status last verified
- Jan 2026
- First submitted
- 30 Sep 2025
- Last updated
- 16 Jan 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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.
- ClinicalTrials.gov ID
- NCT07212426
- Acronym / sponsor ID
- P10-LP005-02
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Longbio PharmaIndustry (pharma, biotech, devices)
- Study start
- 14 Nov 2024 (actual)
- Primary completion
- 6 May 2026 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 8 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| LP-005 900 mg | Experimental | Biological: LP-005 InjectionPatients will receive intravenous infusion of LP-005 Injection at a dose of 900 mg once every 4 weeks. |
| LP-005 1200 mg | Experimental | Biological: LP-005 InjectionPatients will receive intravenous infusion of LP-005 Injection at a dose of 1200 mg once every 4 weeks. |
| LP-005 1500 mg | Experimental | Biological: LP-005 InjectionPatients will receive intravenous infusion of LP-005 Injection at a dose of 1500 mg once every 4 weeks. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LP-005 InjectionIV infusion, Q4W | Biological | LP-005 1200 mg, LP-005 1500 mg, LP-005 900 mg |
Primary outcome
Change from Baseline in serum lactate dehydrogenase (LDH) levels.
Time frame: 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.
11 secondary outcomes
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
Proportion of participants who are transfusion-free.
Time frame: 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.
Change from Baseline in hemoglobin levels.
Time frame: 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
Proportion of participants with LDH levels normalized.
Time frame: Baseline, Week 12, 24 and 48
Changes in serum complement hemolytic activity.
Time frame: Observation from Predose on Day 1 through 337 days post-administration.
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.
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Time frame: Baseline up to approximately 13 months.
Serum concentrations of LP-005.
Time frame: 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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Guangsheng He principal investigator
The First Affiliated Hospital with Nanjing Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LP-005 Injection in people with PNH - Paroxysmal Nocturnal Hemoglobinuria, run by Longbio Pharma at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 16 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in serum lactate dehydrogenase (LDH) levels. (time frame: Baseline and at Week 12.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jan 2026.