An open-label, single-arm, multicenter, phase 3 study to assess pharmacokinetics, safety and tolerability of iptacopan in pediatric PNH patients aged 2 to <18 years of age

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Brief summary

Safety evaluations, including AEs, SAEs, laboratory parameters, vital signs, and cardiovascular parameters., PK parameters Cmax, AUClast, AUCtau (and other PK parameters in plasma, as appropriate), and Ctrough concentrations.

Detailed description

Change in hemoglobin (Hb) from baseline ≥ 1 g/dL at Week 26 (in the absence of red blood cell (RBC) transfusions from Day 14). Change in Hb from baseline ≥ 1 g/dL at Week 52 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 26 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 52 (in the absence of RBC transfusions from Day 14)., Normal Hb at Weeks 26 in the absence of RBC transfusions from Day 14. Normal Hb at Week 52 in the absence of RBC transfusions from Day 14., Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 26. Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 52., Change from baseline in Hb and LDH at Week 26. Change from baseline in Hb and LDH at Week 52., Safety evaluations (including AEs, SAEs, lab parameters, vital signs, and cardiovascular parameters).

Related papers

No related papers found yet.

Papers published before 2007-09-01 may not appear yet. Historical indexing is in progress.

Interventions

DRUGIPTACOPAN HYDROCHLORIDE

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Safety evaluations, including AEs, SAEs, laboratory parameters, vital signs, and cardiovascular parameters., PK parameters Cmax, AUClast, AUCtau (and other PK parameters in plasma, as appropriate), and Ctrough concentrations.	—

Secondary

Measure	Time frame
Change in hemoglobin (Hb) from baseline ≥ 1 g/dL at Week 26 (in the absence of red blood cell (RBC) transfusions from Day 14). Change in Hb from baseline ≥ 1 g/dL at Week 52 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 26 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 52 (in the absence of RBC transfusions from Day 14)., Normal Hb at Weeks 26 in the absence of RBC transfusions from Day 14. Normal Hb at Week 52 in the absence of RBC transfusions from Day 14., Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 26. Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 52., Change from baseline in Hb and LDH at Week 26. Change from baseline in Hb and LDH at Week 52., Safety evaluations (including AEs, SAEs, lab parameters, vital signs, and cardiovascular parameters).	—

Measure

Time frame

Change in hemoglobin (Hb) from baseline ≥ 1 g/dL at Week 26 (in the absence of red blood cell (RBC) transfusions from Day 14). Change in Hb from baseline ≥ 1 g/dL at Week 52 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 26 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 52 (in the absence of RBC transfusions from Day 14)., Normal Hb at Weeks 26 in the absence of RBC transfusions from Day 14. Normal Hb at Week 52 in the absence of RBC transfusions from Day 14., Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 26. Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 52., Change from baseline in Hb and LDH at Week 26. Change from baseline in Hb and LDH at Week 52., Safety evaluations (including AEs, SAEs, lab parameters, vital signs, and cardiovascular parameters).

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Countries

France, Germany, Italy, Netherlands, Spain

Outcome results

None listed