PNH - Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT05731050
Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 2 study testing NM8074 in people with PNH - Paroxysmal Nocturnal Hemoglobinuria, run by NovelMed Therapeutics.
Trial summary
The proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects. 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?
- NM8074
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Jun 2026to 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 patients ≥ 18 years at the time of consent
- Confirmation of PNH diagnosis by flow cytometry evaluation of red blood cells (RBCs) and white blood cells (WBCs), with granulocyte or monocyte clone size of ~5%
- Presence of one or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia: hemoglobin < 10 g/dL, history of a major adverse vascular event (including thrombosis), dysphagia, erectile dysfunction, PNH-mediated pRBC transfusions
- PNH patients must be undergoing treatment with Soliris for at least 3 months prior to screening, and must have a lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (ULN) during Screening
- Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and comply with the study visit schedule.
- Female subjects of childbearing potential must have a negative pregnancy test at Screening, and must not be planning pregnancy throughout the extent of the study term
- Female subjects of child-bearing potential and all male subjects must agree to use of effective contraception during study
- Soliris treated individuals must be able to provide documentation of vaccination against meningococcal infections.
Who cannot join
- Platelet count < 30,000/µL at Screening
- Absolute neutrophil count (ANC) < 500 cells/µL at Screening
- Body weight < 85 lbs. (38 kg) at Screening
- Estimated glomerular filtration rate of < 30 mL/min/1.73m2 based on modification of diet in renal disease (MDRD) equation, creatinine clearance, or CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) at Screening
- Elevation of liver function tests: alanine aminotransferase (ALT) > 2xULN or direct bilirubin and alkaline phosphatase (ALP) both > 2xULN
- Has a known history of meningococcal disease or N. meningitidis infection
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- Has an immunological disorder, such as, but not limited to, human immunodeficiency virus (HIV) infection (as evident by HIV-1 or HIV-2 antibody titer) or any acute or chronic infection including, but not limited to, hepatitis B virus (HBV) or hepatitis C virus (HCV)
- Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection that requires antibiotic, antifungal, antiparasitic, or antiviral mediations
- Temperature > 38°C for more than two weeks prior to screening
- History of bone marrow or solid organ transplantation
- Pregnant, planning to become pregnant, or nursing female subjects
- Recent surgery requiring general anesthesia within the 2 weeks prior to Screening, or expected to have surgery requiring general anesthesia during the 12-week treatment period
- Active malignancy requiring surgery, chemotherapy, or radiation within the prior 12- months (subjects with a history of malignancy who have undergone curative resection or otherwise not requiring treatment for at least 12-months prior to screening with no detectable recurrences are allowed)
- History of any significant major medical conditions (cardiac, pulmonary, renal, e endocrine, or hepatic), or psychiatric disorder that, in the opinion of the Investigator, would make the subject unsuitable for participation in the study
- PNH patients currently under complement blocker treatments other than Soliris
- Concomitant use of anticoagulants is prohibited, if not on a stable regimen for at least 2 weeks prior to Day 1
- Participation in any experimental small molecule or non-antibody therapy within 60 days prior to dosing on Day 1 (participation in observational studies and/or registry studies is permitted)
- Known or suspected history of illegal recreational drug or alcohol abuse within 1 year prior to start of screening
- Hypersensitivity or history of allergy to excipients in NM8074 formulation
- Unable or unwilling to comply with the requirements of the study
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase II, Open Label, Multi Dose Study of NM8074 in Soliris-Treated Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects.
Read the detailed description
The proposed study, NM8074-PNH-101, will evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects. The drug will be administered by intravenous (IV) infusion to adult patients who qualify based on the inclusion/exclusion criteria. A total of at least 6 patients (maximum of 10) with documented PNH and at least 3 months of Soliris treatment prior to Screening will be enrolled at the study site. The total duration of study term participation for all subjects will be up to 22 weeks, including a screening period of up to 8 weeks, a 12-week treatment period, and 2 weeks of a washout period. All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period.
- Being tested
- NM8074
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NM8074Drug
NM8074 will be administered as an intravenous infusion. All enrolled subjects will receive a dose of 15 mg/kg NM8074 once every two weeks for a total of 6 doses from Day1 to Day 84.
For Soliris-treated PNH subjects, admission and dosing of NM8074 on Day 1 should be scheduled to coincide with the next scheduled dose of Soliris (i.e., 14 ± 2 days after the last Soliris dose). Soliris should NOT be administered on Day 1 or at any time thereafter during course of the study.
Subjects in the treatment groups will receive a dose of 15 mg/kg of NM8074 on Day 1, which will be administered during the study visit by the site staff. During the remainder of the treatment period, subjects will be given another 15 mg/kg dose every two weeks at approximately the same time. On days of scheduled study visits at the Investigation Site, the dose must be administered after blood work is completed.
| Study group | Role | What they receive |
|---|---|---|
| NM8074 | Experimental | All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period. Receives: Drug: NM8074 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Normalization of serum LDH(Lactate dehydrogenase) levels from baseline levels
Measured: Up to Week 12
Change from Baseline or Percent Change from Baseline in total hemoglobin levels (g/dL)
Measured: Up to Week 12
Change from Baseline or Percent Change from Baseline in total number of units of packed red blood cells (pRBCs) transfused through Week 12
Measured: Up to Week 12
Change from Baseline or Percent Change from Baseline in number of transfusions
Measured: Up to Week 12
Percent Change from Baseline in Levels of Membrane Attack Complex (MAC) via Alternative Pathway (AP) of Complement Activity as Compared to Percent Change from Baseline in Levels of MAC via Classical Pathway (CP) of Complement Activity
Measured: Up to Week 12
Secondary outcomes (3)
Normalization of anemia
Change from Baseline or Percent Change from Baseline in Reticulocyte Count
Measured: Up to Week 12
Normalization of anemia
Change from Baseline or Percent Change from Baseline in Bilirubin Levels
Measured: Up to Week 12
Change from Baseline or Percent Change from Baseline of C3b deposition on PNH Type II and Type III RBCs via Flow Cytometry
Measured: Up to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Feb 2023
- Study startJun 2026 (estimated)
- Primary completionSep 2027 (estimated)
- Study completionMar 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
- Not applicable
- Intervention model
- Sequential
- Model description
- A total of at least 6 patients (maximum of 10) with documented PNH and at least 3 months of Soliris treatment prior to Screening will be enrolled at the study site. The total duration of study term participation for all subjects will be up to 22 weeks, including a screening period of up to 8 weeks, a 12-week treatment period, and 2 weeks of a washout period. All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period. No more than two subjects will be dosed in a single day.
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NovelMed Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05731050
- Sponsor's protocol ID
- NM8074-PNH-101
- Status last verified
- Mar 2025
- First submitted
- 1 Feb 2023
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open Label, Multi Dose Study of NM8074 in Soliris-Treated Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT05731050
- Acronym / sponsor ID
- NM8074-PNH-101
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- NovelMed TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Jun 2026 (estimated)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Mar 2028 (estimated)
- First posted
- 16 Feb 2023 (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 proposed study, NM8074-PNH-101, is a phase II, open-label, multi-dose, unicenter trial to evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects.
Detailed description
The proposed study, NM8074-PNH-101, will evaluate the safety and efficacy of NM8074 in Soliris-treated PNH subjects. The drug will be administered by intravenous (IV) infusion to adult patients who qualify based on the inclusion/exclusion criteria. A total of at least 6 patients (maximum of 10) with documented PNH and at least 3 months of Soliris treatment prior to Screening will be enrolled at the study site. The total duration of study term participation for all subjects will be up to 22 weeks, including a screening period of up to 8 weeks, a 12-week treatment period, and 2 weeks of a washout period. All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A total of at least 6 patients (maximum of 10) with documented PNH and at least 3 months of Soliris treatment prior to Screening will be enrolled at the study site. The total duration of study term participation for all subjects will be up to 22 weeks, including a screening period of up to 8 weeks, a 12-week treatment period, and 2 weeks of a washout period. All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period. No more than two subjects will be dosed in a single day.
| Arm | Type | Receives |
|---|---|---|
| NM8074 | Experimental | Drug: NM8074All subjects will receive a dose of 15 mg/kg NM8074 every two weeks with a total of 6 doses, from Day 1 to Day 84 during the treatment period. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female patients ≥ 18 years at the time of consent
- Confirmation of PNH diagnosis by flow cytometry evaluation of red blood cells (RBCs) and white blood cells (WBCs), with granulocyte or monocyte clone size of ~5%
- Presence of one or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia: hemoglobin < 10 g/dL, history of a major adverse vascular event (including thrombosis), dysphagia, erectile dysfunction, PNH-mediated pRBC transfusions
- PNH patients must be undergoing treatment with Soliris for at least 3 months prior to screening, and must have a lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (ULN) during Screening
- Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and comply with the study visit schedule.
Key exclusion criteria (20 listed)
- Platelet count < 30,000/µL at Screening
- Absolute neutrophil count (ANC) < 500 cells/µL at Screening
- Body weight < 85 lbs. (38 kg) at Screening
- Estimated glomerular filtration rate of < 30 mL/min/1.73m2 based on modification of diet in renal disease (MDRD) equation, creatinine clearance, or CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) at Screening
- Elevation of liver function tests: alanine aminotransferase (ALT) > 2xULN or direct bilirubin and alkaline phosphatase (ALP) both > 2xULN
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 |
|---|---|---|
| NM8074NM8074 will be administered as an intravenous infusion. All enrolled subjects will receive a dose of 15 mg/kg NM8074 once every two weeks for a total of 6 doses from Day1 to Day 84. For Soliris-treated PNH subjects, admission and dosing of NM8074 on Day 1 should be scheduled to coincide with the next scheduled dose of Soliris (i.e., 14 ± 2 days after the last Soliris dose). Soliris should NOT be administered on Day 1 or at any time thereafter during course of the study. Subjects in the treatment groups will receive a dose of 15 mg/kg of NM8074 on Day 1, which will be administered during the study visit by the site staff. During the remainder of the treatment period, subjects will be given another 15 mg/kg dose every two weeks at approximately the same time. On days of scheduled study visits at the Investigation Site, the dose must be administered after blood work is completed. | Drug | NM8074 |
Primary outcome
Normalization of serum LDH(Lactate dehydrogenase) levels from baseline levels
Time frame: Up to Week 12
Change from Baseline or Percent Change from Baseline in total hemoglobin levels (g/dL)
Time frame: Up to Week 12
Change from Baseline or Percent Change from Baseline in total number of units of packed red blood cells (pRBCs) transfused through Week 12
Time frame: Up to Week 12
Change from Baseline or Percent Change from Baseline in number of transfusions
Time frame: Up to Week 12
Percent Change from Baseline in Levels of Membrane Attack Complex (MAC) via Alternative Pathway (AP) of Complement Activity as Compared to Percent Change from Baseline in Levels of MAC via Classical Pathway (CP) of Complement Activity
Time frame: Up to Week 12
3 secondary outcomes
Normalization of anemia
Time frame: Up to Week 12
Normalization of anemia
Time frame: Up to Week 12
Change from Baseline or Percent Change from Baseline of C3b deposition on PNH Type II and Type III RBCs via Flow Cytometry
Time frame: Up to Week 12
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 30 Mar 2025.
- Individual participant dataSharing planned: No
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NM8074 in people with PNH - Paroxysmal Nocturnal Hemoglobinuria, run by NovelMed Therapeutics.
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 8 inclusion and 20 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: opening soon, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
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?
Contact Rekha Bansal, PhD (2164402696, clinicalsae@novelmed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Normalization of serum LDH(Lactate dehydrogenase) levels from baseline levels (time frame: Up to Week 12). The study lists 5 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 30 Mar 2025.