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Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT05646524

Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

A Phase 2 study testing NM8074 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
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Trial summary

This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). 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
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
Apr 2026to Sep 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 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 of white blood cells (WBCs), with neutrophil, granulocyte and/or monocyte clone size of ≥10%
  • Evidence of ongoing hemolysis
  • ≥1 packed red blood cell (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
  • 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

  • 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
  • Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1
  • Participants with known or suspected hereditary or acquired complement deficiency
  • History of currently active primary or secondary immunodeficiency
Show all 15Show fewer
  • 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 (chronic kidney disease (CKD) stage 4, dialysis)
  • Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1
  • 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 refrain from donating 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 Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).

Read the detailed description

The proposed study will enroll a planned number of 12 treatment naïve PNH patients with amaximum of 18 PNH patients who have been diagnosed with hemolytic anemia and meet the inclusion criteria. There will be 2 cohorts with 6 to 9 patients each. Patients in Cohort 1 will be administered NM8074 at 20 mg/kg intravenously (IV) every 2 weeks over the treatment period. Cohort 2 patients will be administered a dose of 10 mg/kg NM8074 weekly for 4 weeks followed by a 20 mg/kg dose of NM8074 administered via IV every 2 weeks for the remainder of the treatment period. This study will determine if NM8074 will provide the desired inhibition of the alternative pathway (AP).

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. Doses will be administered over a treatment period of 13 weeks.

Study groups in NCT05646524
Study groupRoleWhat they receive
Cohort 1Experimental6 subjects will receive an intravenous (IV) infusion of NM8074 at 20 mg/kg every two weeks.
Receives: Drug: NM8074
Cohort 2Experimental6 subjects will receive an intravenous (IV) infusion of NM8074 at 10 mg/kg weekly for four weeks followed by a 20 mg/kg dose of NM8074 every two weeks for the remainder of the treatment period.
Receives: Drug: NM8074

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 startApr 2026 (estimated)
  3. Primary completionAug 2027 (estimated)
  4. Study completionSep 2028 (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
12 (18) patients will be divided into two cohorts of 6 (9) patients each. Patients will be dosed in parallel with either a 20 mg/kg NM8074 dose every two weeks for the entire duration of the treatment period or with an initial dose of 10 mg/kg NM8074 weekly for the first four weeks followed by the 20 mg/kg dose for the remainder of the treatment period.
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05646524
Sponsor's protocol ID
NM8074-PNH-105
Status last verified
Mar 2025
First submitted
30 Nov 2022
Last updated
11 Mar 2025
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 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 8 inclusion and 15 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 11 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 (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 NCT05646524.

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 11 Mar 2025.

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