A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

Related Clinical Trial
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria Compassionate Use of Pozelimab Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy Study of ARO-C3 in Adult Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria and Complement-Mediated Renal Disease A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria Pegcetacoplan Early Access Program for PNH A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC “GENERIUM”, Russian Federation) Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH A Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors. A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies Unrelated Donor Stem Cell Transplantation Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies Unrelated And Partially Matched Related Donor PSCT w/ TCR αβ Depletion for Patients With BMF Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH Study of Global Coagulation Tests in Patients With Paroxysmal Nocturnal Haemoglobinuria A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects With PNH Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. APL-2 in PNH Subjects Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH A Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Proof of Concept Study of ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH) REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Transfer of GPI-Linked Proteins to Transfused Patients With Paroxysmal Nocturnal Hemoglobinuria Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Brief Title

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

Official Title

A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study

Brief Summary

      This is a single-arm long-term extension study that will enroll participants with PNH who
      have completed participation in Alexion-sponsored clinical studies with danicopan as an add
      on therapy to a C5i.
    

Detailed Description

      The total duration of the study will be up to 3 years. Eligible participants must complete
      all study assessments on the parent protocol before starting this study. All participants
      entering this study will receive danicopan as an add-on to a background C5i therapy. The only
      allowed C5i therapies are eculizumab and ravulizumab.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs

Secondary Outcome

 Change In Hemoglobin Values

Condition

Paroxysmal Nocturnal Hemoglobinuria

Intervention

Danicopan

Study Arms / Comparison Groups

 Danicopan
Description:  Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

100

Start Date

May 16, 2022

Completion Date

December 25, 2025

Primary Completion Date

December 25, 2025

Eligibility Criteria

        Key Inclusion Criteria:

          -  All participants who completed their participation in an Alexion sponsored clinical
             study with danicopan as an add on to a C5i treatment.

          -  Patient is capable of giving signed informed consent, which includes compliance with
             the requirements and restrictions listed in the informed consent form and in this
             protocol.

          -  Documentation of vaccination for Neisseria meningitidis: All participants must be
             revaccinated as per national vaccination guidelines or local practice for vaccination
             use with complement inhibitors.

        Key Exclusion Criteria:

          -  Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or
             psychiatric) that, in the opinion of the Investigator, might interfere with
             participation in the study, pose any added risk to the participant, or confound the
             assessment of the participant.

          -  Patient has been permanently discontinued from danicopan in the parent study for any
             reason other than enrollment into this LTE study.

          -  Female participants who are pregnant, breastfeeding, or intending to conceive during
             the course of the study.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, +1 855-752-2356, [email protected]



Administrative Informations


NCT ID

NCT05389449

Organization ID

ALXN2040-PNH-303

Secondary IDs

2021-004253-22

Responsible Party

Sponsor

Study Sponsor

Alexion Pharmaceuticals


Study Sponsor

, , 


Verification Date

May 2022