Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorNovelMed Therapeutics
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Sequential
Treatments tested in this trial
NM8074
DrugNM8074 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.
Treatment groups
6 Participants
are divided into 1 treatment groupGroup A: NM8074Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Normalization of serum LDH(Lactate dehydrogenase) levels from baseline levels
Time frame
Up to Week 122
Change from Baseline or Percent Change from Baseline in total hemoglobin levels (g/dL)
Time frame
Up to Week 123
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 124
Change from Baseline or Percent Change from Baseline in number of transfusions
Time frame
Up to Week 12Secondary outcomes
1
Normalization of anemia
Change from Baseline or Percent Change from Baseline in Reticulocyte Count
Time frame
Up to Week 122
Normalization of anemia
Change from Baseline or Percent Change from Baseline in Bilirubin Levels
Time frame
Up to Week 123
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 12Locations
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: