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

ConditionParoxysmal Nocturnal Hemoglobinuria

Trial statusNot yet recruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18+

SponsorNovelMed Therapeutics

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

NM8074
Drug
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.
Soliris
Drug
Complement C5 blocker administered intravenously

Treatment groups

12 Participants

are divided into 2 treatment groups

Group A: Cohort 1Experimental treatment 2 interventions

Group B: Cohort 2Experimental treatment 2 interventions

Trial outcomes

Primary outcomes

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

Time frame

Up to Study Day 105

Number of Participants with Antidrug Antibodies (ADAs) to NM8074

Time frame

Up to Study Day 105

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

Time frame

Up to Study Day 105

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

Time frame

Up to Study Day 105

Secondary outcomes

Change from Baseline or Percent Change from Baseline in Reticulocyte Count

Time frame

Up to Study Day 105

Change from Baseline or Percent Change from Baseline in Bilirubin Levels

Time frame

Up to Study Day 105

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.

Time frame

Up to Study Day 105

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.

Time frame

Up to Study Day 105

Other outcomes

Change from Baseline or Percent Change from Baseline in Complement Component Factor B Levels

Time frame

Up to Study Day 105

Change from Baseline or Percent Change from Baseline in Haptoglobin Levels

Time frame

Up to Study Day 105

Change from Baseline or Percent Change from Baseline in Platelet Count

Time frame

Up to Study Day 105

Change from Baseline or Percent Change from Baseline in PNH Cell Clone Size

Clone size will be measured via fluorescein-labeled proaerolysin (FLAER) staining of WBCs (granulocytes and monocytes)

Time frame

Up to Study Day 105

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

NovelMed Therapeutics

Lead sponsor

Source ClinicalTrials.gov

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