Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 1NCT06294301
A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants
A Phase 1 study testing LP-005 Dose 1 (Single), LP-005 Dose 2 (Single) and LP-005 Dose 3 (Single) in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Longbio Pharma at 1 site in China.
Trial summary
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of LP-005 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of LP-005 and Part 2, multiple ascending… Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- LP-005 Dose 1 (Single), LP-005 Dose 2 (Single) and LP-005 Dose 3 (Single)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 1 siteChina
- Study dates
- 23 Nov 2023to 31 Aug 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy males or females aged 18 through 50 years
- Male subjects with a weight of ≥50 kg, female subjects with a weight of ≥45 kg, and BMI between 19.0 and 26.0 kg/m² (inclusive).
- Vaccination: Meningococcal Conjugate Vaccine, Serogroups A, C, W, Y (MPV-ACYW) meningococcal conjugate vaccine and Streptococcus pneumoniae vaccine should be given 14 days or more before randomisation.
- Male subjects and their partners or female subjects must agree to use one or more non-pharmaceutical contraceptive methods (such as total abstinence, condoms, Iuds, partner ligation, etc.) during the trial period and for 6 months after the trial, and do not plan to donate sperm or eggs.
- The subjects fully understand the purpose, nature, method and possible adverse reactions of the experiment, and voluntarily participate in the experiment and sign the informed consent.
- The subjects were able to communicate well with the researchers and complete the study according to the protocol.
Who cannot join
- Participants who are immunocompromised or have one of the following underlying diseases: anatomic absence of spleen (including sickle cell disease); congenital complement component deficiencies (complement component 3 and complement component 4).
- Any history of Neisseria gonorrhea, meningitis infection, and Guillain-Barré syndrome.
- Contraindications to meningococcal vaccination (previous medical history such as epilepsy or other brain disorders).
- Presence or suspicion of active viral, bacterial, fungal, or parasitic infection, including herpes, shingles, or cold sores, within 14 days prior to screening.
- History of unexplained recurrent infections, or use of systemic antibiotics within 90 days prior to dosing.
- Malignancy or history of malignancy, except non-melanoma skin cancer cured for more than 3 years.
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- Positive HIV test (HIV-Ab), positive hepatitis B virus (HBV) test (HBsAg), positive hepatitis C virus (HCV), positive anti-syphilis helix-specific antibodies.
- Participation in a clinical trial of any other drug within 3 months prior to screening or within 5 half-lives of other clinical trial drugs (selecting the longer time period).
- Women who are pregnant, breastfeeding, or at risk of pregnancy.
- Any condition deemed unsuitable for study participation by the investigator.
Trial locations and contacts
1 site in 1 country.
China1 site
Shanghai Public Health Clinical Center
Shanghai, Shanghai Municipality
About this study
Official title: To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of LP-005 in Healthy Volunteers
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of LP-005 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of LP-005 and Part 2, multiple ascending dose (MAD).
- Being tested
- LP-005 Dose 1 (Single), LP-005 Dose 2 (Single), LP-005 Dose 3 (Single), LP-005 Dose 4 (Single), LP-005 Dose 5 (Single)
- 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 Dose 1 (Single)Biological
A single dose of LP-005 (Dose 1) was administered intravenously.
LP-005 Dose 2 (Single)Biological
A single dose of LP-005 (Dose 2) was administered intravenously.
LP-005 Dose 3 (Single)Biological
A single dose of LP-005 (Dose 3) was administered intravenously.
LP-005 Dose 4 (Single)Biological
A single dose of LP-005 (Dose 4) was administered intravenously.
LP-005 Dose 5 (Single)Biological
A single dose of LP-005 (Dose 5) was administered intravenously.
LP-005 Dose 6 (Single)Biological
A single dose of LP-005 (Dose 6) was administered intravenously.
Placebo (Single)Biological
A single dose of placebo was administered intravenously.
LP-005 Dose 7 (Multiple)Biological
LP-005 (Dose 7) was administered multiple times intravenously.
LP-005 Dose 8 (Multiple)Biological
LP-005 (Dose 8) was administered multiple times intravenously.
LP-005 Dose 9 (Multiple)Biological
LP-005 (Dose 9) was administered multiple times intravenously.
Placebo (Multiple)Biological
Placebo was administered multiple times intravenously.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: LP-005 Dose 1 (Single) | Experimental | Not described Receives: Biological: LP-005 Dose 1 (Single) |
| Cohort 2: LP-005 Dose 2 (Single) | Experimental | Not described Receives: Biological: LP-005 Dose 2 (Single) |
| Cohort 3: LP-005 Dose 3 (Single) | Experimental | Not described Receives: Biological: LP-005 Dose 3 (Single) |
| Cohort 4: LP-005 Dose 4 (Single) | Experimental | Not described Receives: Biological: LP-005 Dose 4 (Single) |
| Cohort 5: LP-005 Dose 5 (Single) | Experimental | Not described Receives: Biological: LP-005 Dose 5 (Single) |
| Cohort 6: LP-005 Dose 6 (Single) | Experimental | Not described Receives: Biological: LP-005 Dose 6 (Single) |
| Cohort 7: Placebo (Single) | Placebo comparator | Not described Receives: Biological: Placebo (Single) |
| Cohort 8: LP-005 Dose 7 (Multiple) | Experimental | Not described Receives: Biological: LP-005 Dose 7 (Multiple) |
| Cohort 9: LP-005 Dose 8 (Multiple) | Experimental | Not described Receives: Biological: LP-005 Dose 8 (Multiple) |
| Cohort 10: LP-005 Dose 9 (Multiple) | Experimental | Not described Receives: Biological: LP-005 Dose 9 (Multiple) |
| Cohort 11: Placebo (Multiple) | Placebo comparator | Not described Receives: Biological: Placebo (Multiple) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Number of subjects with treatment-related Treatment Emergent Adverse Events (TEAEs).
Measured: Observation for 78 days after administration
Secondary outcomes (8)
Time to peak concentration (Tmax) of LP-005
The time when the blood drug concentration reaches its peak after a single dose of medication.
Measured: Observation for 78 days after administration
Maximum concentration (Cmax) of LP-005
The maximum concentration of LP-005 in the bloodstream after administration.
Measured: Observation for 78 days after administration
Elimination half-life (t1/2) of LP-005
The time required for the concentration of LP-005 in the bloodstream to decrease by half.
Measured: Observation for 78 days after administration
Area under the concentration-time curve (AUC0-t) of LP-005
The area under the concentration-time curve (AUC) from time zero to the last chosen time point represents the integral of the drug concentration in the bloodstream over the specified duration.
Measured: Observation for 78 days after administration
Apparent clearance rate (CL/F) of LP-005
The ratio of drug clearance to drug concentration, represents the apparent clearance of a drug after administration, adjusted for bioavailability.
Measured: Observation for 78 days after administration
Assessment of immunogenicity
The proportion of anti drug antibody (ADA) positive subjects at different detection time points.
Measured: Observation for 78 days after administration
Assessment of complement C5 activity
Evaluate complement C5 hemolytic activity and serum concentration of C5 changes from baseline at various time points of assessment.
Measured: Observation for 78 days after administration
Assessment of complement C3b activity
Evaluate C3b deposition on red blood cells and serum concentration of C3b changes from baseline at various time points of assessment.
Measured: Observation for 78 days after administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2024
- Study start23 Nov 2023
- Primary completion31 Aug 2024
- Study completion31 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Longbio Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06294301
- Sponsor's protocol ID
- P10-LP005-01
- Status last verified
- Oct 2025
- First submitted
- 3 Feb 2024
- Last updated
- 15 Dec 2025
- 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 15 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of LP-005 in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT06294301
- Acronym / sponsor ID
- P10-LP005-01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Longbio PharmaIndustry (pharma, biotech, devices)
- Study start
- 23 Nov 2023 (actual)
- Primary completion
- 31 Aug 2024 (actual)
- Study completion
- 31 Aug 2024 (actual)
- First posted
- 5 Mar 2024 (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.
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of LP-005 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of LP-005 and Part 2, multiple ascending dose (MAD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 11
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: LP-005 Dose 1 (Single) | Experimental | Biological: LP-005 Dose 1 (Single) |
| Cohort 2: LP-005 Dose 2 (Single) | Experimental | Biological: LP-005 Dose 2 (Single) |
| Cohort 3: LP-005 Dose 3 (Single) | Experimental | Biological: LP-005 Dose 3 (Single) |
| Cohort 4: LP-005 Dose 4 (Single) | Experimental | Biological: LP-005 Dose 4 (Single) |
| Cohort 5: LP-005 Dose 5 (Single) | Experimental | Biological: LP-005 Dose 5 (Single) |
| Cohort 6: LP-005 Dose 6 (Single) | Experimental | Biological: LP-005 Dose 6 (Single) |
| Cohort 7: Placebo (Single) | Placebo comparator | Biological: Placebo (Single) |
| Cohort 8: LP-005 Dose 7 (Multiple) | Experimental | Biological: LP-005 Dose 7 (Multiple) |
| Cohort 9: LP-005 Dose 8 (Multiple) | Experimental | Biological: LP-005 Dose 8 (Multiple) |
| Cohort 10: LP-005 Dose 9 (Multiple) | Experimental | Biological: LP-005 Dose 9 (Multiple) |
| Cohort 11: Placebo (Multiple) | Placebo comparator | Biological: Placebo (Multiple) |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Healthy males or females aged 18 through 50 years
- Male subjects with a weight of ≥50 kg, female subjects with a weight of ≥45 kg, and BMI between 19.0 and 26.0 kg/m² (inclusive).
- Vaccination: Meningococcal Conjugate Vaccine, Serogroups A, C, W, Y (MPV-ACYW) meningococcal conjugate vaccine and Streptococcus pneumoniae vaccine should be given 14 days or more before randomisation.
- Male subjects and their partners or female subjects must agree to use one or more non-pharmaceutical contraceptive methods (such as total abstinence, condoms, Iuds, partner ligation, etc.) during the trial period and for 6 months after the trial, and do not plan to donate sperm or eggs.
- The subjects fully understand the purpose, nature, method and possible adverse reactions of the experiment, and voluntarily participate in the experiment and sign the informed consent.
Key exclusion criteria (10 listed)
- Participants who are immunocompromised or have one of the following underlying diseases: anatomic absence of spleen (including sickle cell disease); congenital complement component deficiencies (complement component 3 and complement component 4).
- Any history of Neisseria gonorrhea, meningitis infection, and Guillain-Barré syndrome.
- Contraindications to meningococcal vaccination (previous medical history such as epilepsy or other brain disorders).
- Presence or suspicion of active viral, bacterial, fungal, or parasitic infection, including herpes, shingles, or cold sores, within 14 days prior to screening.
- History of unexplained recurrent infections, or use of systemic antibiotics within 90 days prior to dosing.
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 Dose 1 (Single)A single dose of LP-005 (Dose 1) was administered intravenously. | Biological | Cohort 1: LP-005 Dose 1 (Single) |
| LP-005 Dose 2 (Single)A single dose of LP-005 (Dose 2) was administered intravenously. | Biological | Cohort 2: LP-005 Dose 2 (Single) |
| LP-005 Dose 3 (Single)A single dose of LP-005 (Dose 3) was administered intravenously. | Biological | Cohort 3: LP-005 Dose 3 (Single) |
| LP-005 Dose 4 (Single)A single dose of LP-005 (Dose 4) was administered intravenously. | Biological | Cohort 4: LP-005 Dose 4 (Single) |
| LP-005 Dose 5 (Single)A single dose of LP-005 (Dose 5) was administered intravenously. | Biological | Cohort 5: LP-005 Dose 5 (Single) |
| LP-005 Dose 6 (Single)A single dose of LP-005 (Dose 6) was administered intravenously. | Biological | Cohort 6: LP-005 Dose 6 (Single) |
| Placebo (Single)A single dose of placebo was administered intravenously. | Biological | Cohort 7: Placebo (Single) |
| LP-005 Dose 7 (Multiple)LP-005 (Dose 7) was administered multiple times intravenously. | Biological | Cohort 8: LP-005 Dose 7 (Multiple) |
| LP-005 Dose 8 (Multiple)LP-005 (Dose 8) was administered multiple times intravenously. | Biological | Cohort 9: LP-005 Dose 8 (Multiple) |
| LP-005 Dose 9 (Multiple)LP-005 (Dose 9) was administered multiple times intravenously. | Biological | Cohort 10: LP-005 Dose 9 (Multiple) |
| Placebo (Multiple)Placebo was administered multiple times intravenously. | Biological | Cohort 11: Placebo (Multiple) |
Primary outcome
Adverse events
Time frame: Observation for 78 days after administration
Number of subjects with treatment-related Treatment Emergent Adverse Events (TEAEs).
8 secondary outcomes
Time to peak concentration (Tmax) of LP-005
Time frame: Observation for 78 days after administration
Maximum concentration (Cmax) of LP-005
Time frame: Observation for 78 days after administration
Elimination half-life (t1/2) of LP-005
Time frame: Observation for 78 days after administration
Area under the concentration-time curve (AUC0-t) of LP-005
Time frame: Observation for 78 days after administration
Apparent clearance rate (CL/F) of LP-005
Time frame: Observation for 78 days after administration
Assessment of immunogenicity
Time frame: Observation for 78 days after administration
Assessment of complement C5 activity
Time frame: Observation for 78 days after administration
Assessment of complement C3b activity
Time frame: Observation for 78 days after administration
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Dec 2025.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LP-005 Dose 1 (Single), LP-005 Dose 2 (Single) and LP-005 Dose 3 (Single) in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), 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 6 inclusion and 10 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: finished, as of the sponsor's last update on 15 Dec 2025. 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. Some participants in this study may receive a placebo. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adverse events (time frame: Observation for 78 days after administration). 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 15 Dec 2025.