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Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy

ConditionaHUS - Atypical Hemolytic Uremic Syndrome
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorNovelMed Therapeutics

About this trial

This is a Phase II, open-label study designed to determine if intravenously administered NM8074 results in remission from TMA in treatment-naïve aHUS patients.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients ≥ 18 years at the time of consent

Patients with evidence of resistant or relapsed complement-mediated aHUS with symptoms of Thrombocytopenia, hemolysis, ongoing Thrombotic Microangiopathy and acute kidney injury.

Evidence of ongoing Thrombotic Microangiopathy which includes Haptoglobin <LLN or undetectable and/or presence of schistocytes

Acute kidney injury (proteinuria/creatinuria > ULN and/or reduced eGFR)

Disqualifiers

History of bone marrow, hematopoietic stem cell, or solid organ transplantation

Treatment with complement blockers

Patients with infections

HUS due to ADAMTS-13 deficiency (<5%)

Trial design

Design model

Sequential

Treatments tested in this trial

  • NM8074

    Drug

    NM8074 will be administered as an intravenous infusion. In Cohort 1, all subjects will be administered 20 mg/kg of NM8074 intravenously every two weeks for a total of 7 doses from Day 1 to Day 85 of the Treatment Period. Patients in Cohort 2 will receive weekly doses of 10 mg/kg for a total of 4 doses from Day 1 to Day 22 followed by biweekly doses at 20 mg/kg for a total of 5 doses from Day 29 to Day 85.

Treatment groups

12 Participants
are divided into 2 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Normalization of platelet count (≥150 x 10^9/L)

Time frame
Up to Study Day 120
2

Normalization of LDH levels to below ULN

Time frame
Up to Study Day 120
3

Normalization of Schistocyte levels (<1%)

Time frame
Up to Study Day 120
4

Change from Baseline or Percent Change from Baseline in renal function

Assessed via the change from baseline or percent change from baseline in serum creatinine level.

Time frame
Up to Study Day 120

Secondary outcomes

1

Time to achieve complete TMA response

Time frame
Baseline through Study Day 120
2

Time to achieve higher hemoglobin from baseline

Time frame
Baseline through Study Day 120
3

Change from Baseline or Percent Change from Baseline in blood clots

Time frame
Up to Study Day 120
4

Change from Baseline or Percent Change from Baseline in the total number of plasma infusions or exchanges

Time frame
Baseline through Study Day 120

Other outcomes

1

Change from Baseline or Percent Change from Baseline in CP modulation

Time frame
Baseline through Study Day 120
2

Change from Baseline or Percent Change from Baseline in Factor B levels

Time frame
Baseline through Study Day 120
3

Change from Baseline or Percent Change from Baseline in plasma concentration of NM8074

Time frame
Baseline through Study Day 120
4

Maximum plasma concentration (Cmax)

Time frame
Baseline through Study Day 120

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

This trial is not recruiting at the moment. You can still explore other options: