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Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT05646563

Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris

A Phase 2 study testing NM8074 and Soliris in people with Paroxysmal Nocturnal Hemoglobinuria, run by NovelMed Therapeutics.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Paroxysmal Nocturnal Hemoglobinuria
What is being tested?
NM8074 and Soliris
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
No sites listed yet
Study dates
Jan 2027to Aug 2030
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients ≥ 18 years (males and females), weight ≥ 45 kg at the time of consent.
  • Confirmation of PNH diagnosis by flow cytometry evaluation white blood cells (WBCs), with neutrophil, granulocyte and/or monocyte clone size of ≥10%.
  • Evidence of ongoing hemolysis.
  • ≥1 pRBC transfusion within 12 months prior to screening.
  • Anemia (Hemoglobin ≤10.5 g/dL).
  • Lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (xULN) during Screening.
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  • Treatment with Soliris
  • All patients must be vaccinated prior to dosing with MenACWY Menactra® polysaccharide diphtheria toxoid conjugate vaccination against Neisseria meningitidis serogroups A, C, Y, and W-135 and MenB meningococcal serogroup B vaccine (Bexsero®). If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics.
  • 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.

Who cannot join

  • Subjects currently or previously under other complement inhibitor treatments other than Soliris less than 3 months prior to study Day 1
  • History of bone marrow, hematopoietic stem cell, or solid organ transplantation
  • History of splenectomy
  • Participation in any other investigational drug trial within 5 elimination half-lives of enrollment, or within 30 days, whichever is longer
  • Participants with known or suspected hereditary or acquired complement deficiency
  • History of currently active primary or secondary immunodeficiency
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  • Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infections
  • Has a known history of meningococcal disease or N. meningitidis infection
  • Patients on immunosuppressive agents or systemic corticosteroids less than 8 weeks prior to dosing
  • Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study.
  • Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (CKD stage 4, dialysis)
  • Pregnant, planning to become pregnant, or nursing female subjects. Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for 1 week after stopping the investigational drug.
  • Females who have a positive pregnancy test result at Screening or on Day 1.
  • Male patients and partners of child-bearing potential must agree to use contraceptives and male patients must agree to not donate sperm for the duration 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.

Rekha Bansal contact

216-440-2696

clinicalsae@novelmed.com

About this study

Official title: A Phase II, Open-Label Study of NM8074 in Soliris®-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.

Read the detailed description

The proposed study, NM8074-PNH-106, will enroll a planned number of 12 Soliris-treated PNH patients who have been diagnosed with hemolytic anemia and meet the defined inclusion criteria. This study will evaluate the safety, efficacy, and immunogenicity of NM8074 as both a mono- and combination therapy with complement component C5 blocker Soliris. Patients will be evenly divided into two cohorts.

Being tested
NM8074, Soliris
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 is an anti-Factor Bb humanized monoclonal antibody that will be administered as an intravenous infusion. Doses will be administered over a treatment period of 13 weeks.

  • SolirisDrug

    Also called Eculizumab

    Complement C5 blocker administered intravenously

Study groups in NCT05646563
Study groupRoleWhat they receive
Cohort 1Experimental6 Soliris-treated patients will receive an intravenous (IV) dose of NM8074 at 10 mg/kg weekly for 4 weeks. Patients will then discontinue Soliris treatment and be administered NM8074 at 20 mg/kg IV every 2 weeks for the remainder of the treatment period (8 weeks). At the end of the treatment period, patients will resume Soliris monotherapy as prescribed.
Receives: Drug: NM8074; Drug: Soliris
Cohort 2Experimental6 Soliris-treated patients will receive an intravenous (IV) dose of NM8074 at 10 mg/kg for 4 weeks. Patients will then continue receiving Soliris while being administered NM8074 as a combination therapy at 20 mg/kg IV every 2 weeks for the remainder of the treatment period (8 weeks). At the end of the treatment period, patients will resume Soliris monotherapy as prescribed.
Receives: Drug: NM8074; Drug: Soliris

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Monitoring of Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Adverse events will be graded according to the CTCAE v4.03. If the AE term is not described in the grading scales, the AE severity shall be reported according to the following:

    Grade I: Mild (awareness of sign or symptom, but easily tolerated)

    Grade II: Moderate (discomfort sufficient to cause interference with normal activities)

    Grade III: Severe (incapacitating, with inability to perform normal activities)

    Grade IV: Life threatening

    Grade V: Fatal

    Measured: Up to Study Day 105

  2. Number of Participants with Antidrug Antibodies (ADAs) to NM8074

    Measured: Up to Study Day 105

  3. Change from Baseline or Percent Change from Baseline in Hemoglobin (Hgb) Levels

    Measured: Up to Study Day 105

  4. Change from Baseline or Percent Change from Baseline in Lactate Dehydrogenase (LDH) Levels

    Measured: Up to Study Day 105

  5. Change from Baseline or Percent Change from Baseline in Number of Packed Red Blood Cell (pRBC) Transfusions

    Measured: Up to Study Day 105

  6. 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 Study Day 105

  7. Percent Change from Baseline in Levels of Complement Component C3b via Alternative Pathway (AP) of Complement Activity as Compared to Percent Change from Baseline in Levels of C3b via Classical Pathway (CP) of Complement Activity

    Measured: Up to Study Day 105

Secondary outcomes (8)

  1. Change from Baseline or Percent Change from Baseline in Reticulocyte Count

    Measured: Up to Study Day 105

  2. Change from Baseline or Percent Change from Baseline in Bilirubin Levels

    Measured: Up to Study Day 105

  3. Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) Survey Assessed via the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale, Version 4.

    The FACIT-fatigue scale is a 13-item patient-reported measure of fatigue with a 7-day recall period. Items are scored on a 0 - 4 response scale ranging from "Not at all" to "Very much so". All items are summed to create a single fatigue score with a range from 0 to 52 with a better quality of life indicated by a higher score.

    Measured: Up to Study Day 105

  4. Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) Survey Assessed via the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 Scale (QLQ- C30), Version 3.0.

    All EORTC QLQ-C30 scales and single-item measures range from 0 to 100. This includes 3 symptom scales (fatigue, pain, nausea and vomiting), 5 functional scales (physical, role, cognitive, emotional, and social), single-item questions addressing symptoms like insomnia, dyspnea, loss of appetite, and others that are commonly reported by cancer patients, and the perceived financial impact of the disease. A higher score is associated with a greater quality of life for global health status.

    Measured: Up to Study Day 105

  5. Changes in plasma concentration of NM8074

    Measured: Up to Study Day 105

  6. Maximum plasma concentration (Cmax)

    Measured: Up to Study Day 105

  7. Time corresponding to Cmax (tmax)

    Measured: Up to Study Day 105

  8. Area Under the Drug Concentration-Time Curves (AUC0-t)

    Measured: Up to Study Day 105

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Dec 2022
  2. Study startJan 2027 (estimated)
  3. Primary completionJul 2029 (estimated)
  4. Study completionAug 2030 (estimated)

Resources

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
Non-randomised
Intervention model
Parallel groups
Model description
Patients will be divided evenly into two cohorts that will be evaluated in parallel. Both cohorts will evaluate NM8074 as a combination therapy with Soliris and Cohort 1 will also evaluate NM8074 as a monotherapy.
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05646563
Sponsor's protocol ID
NM8074-PNH-106
Status last verified
Apr 2026
First submitted
30 Nov 2022
Last updated
13 Apr 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NM8074 and Soliris in people with 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 9 inclusion and 14 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 13 Apr 2026. 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 (216-440-2696, clinicalsae@novelmed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Monitoring of Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Up to Study Day 105). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05646563.

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 13 Apr 2026.

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