Health TrialsbyHealthPanel

Efficacy and Safety of Lanoracopan in the Treatment of PNH

ConditionParoxysmal Nocturnal Hemoglobinuria, PNH
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-75
SponsorPeking Union Medical College Hospital

About this trial

This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age 18-75 years.

Newly diagnosed PNH with active hemolysis (hemoglobin [Hb] <100 g/L, lactate dehydrogenase [LDH] >1.5×upper limit of normal [ULN]);

Receiving a stable regimen of complement C5 monoclonal antibody (eculizumab) at standard dose and interval for ≥3 months, with Hb still <120 g/L despite treatment;

Intolerant to eculizumab therapy;

Disqualifiers

Previous bone marrow or hematopoietic stem cell transplantation.

Previous splenectomy.

Known or suspected hereditary complement deficiency.

History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.

Trial population

Patients who visited Peking Union Medical College Hospital

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Lanoracopan Hydrochloride

    Drug

    400mg bid

Treatment groups

No treatment groups listed

Trial outcomes

Primary outcomes

1

rate of adverse events (AEs)

According to CTCAE V5.0

Time frame
During treatment, an average of 24 weeks

Secondary outcomes

1

Proportion of subjects maintaining Hb ≥120 g/L

During the treatment observation period, assess the proportion of subjects maintaining Hb ≥120 g/L every 12 weeks without red blood cell transfusion

Time frame
12 weeks, 24 weeks
2

changes in hemoglobin (Hb) levels

During the treatment observation period, evaluate changes in Hb levels every 12 weeks without red blood cell transfusion.

Time frame
12 weeks, 24 weeks
3

incidence of clinically significant hemolysis

During the treatment observation period, assess the incidence of clinically significant hemolysis every 12 weeks.

Time frame
12 weeks, 24 weeks
4

proportion of subjects experiencing a major adverse vascular event (MAVE)

During the treatment observation period, assess the proportion of subjects experiencing a MAVE every 12 weeks.

Time frame
12 weeks, 24 weeks

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

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

Peking Union Medical College Hospital

Lead sponsor

Source ClinicalTrials.gov

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