NCT03814798

Study Evaluating IGSC 20% Flexible Dosing in Treatment-Experienced and Treatment-Naive Subjects With Primary Immunodeficiency

A Multicenter, Randomized, Cross-over, Open-label Study to Evaluate IGSC 20% Flexible Dosing Including Daily Push Dosing In Treatment-Experienced Subjects With Primary Immunodeficiency (PI) and Evaluation of Loading/Maintenance IGSC 20% in Treatment-Naïve Subjects With PI

• Status: Withdrawn
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Grifols Therapeutics LLC · Industry
• Sex: All
• Age: 2 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, multi-center, randomized, open-label, 2-period cross-over study (16 weeks per treatment period) to evaluate flexible dosing and daily push dosing of IGSC 20% in treatment-experienced subjects with PI. An additional, separate cohort of treatment-naïve, non-randomized subjects who will not be part of the crossover are included and will receive a loading dose of 5 consecutive daily doses of IGSC 20% followed by weekly infusions of IGSC 20% starting Week 1 (Day 8) through Week 32 (end of Treatment Phase). For treatment-experienced subjects, the study consists of a Screening Visit, Baseline Visit, 16-week Treatment Period 1, 16-week Treatment Period 2, and Final Visit/Early Termination Visit. For treatment-naïve subjects, the study consists of a Screening Visit, a Baseline Visit, a 32-week Treatment Phase, and Final Visit/Early Termination Visit. Approximately 54 treatment-experienced subjects and approximately 6 treatment-naïve subjects will be enrolled at study centers in the United States (US) and European Union (EU).

Detailed description

This is a prospective, multi-center, randomized, open-label, 2-period cross-over study (16 weeks per treatment period) to evaluate flexible dosing and daily push dosing of IGSC 20% in treatment-experienced subjects with PI. There is an additional, separate cohort of treatment-naïve, non-randomized subjects with PI who will not be part of the crossover. Treatment-experienced subjects entering the study will maintain the same IgG dose (mg/kg) received prestudy that will be normalized to the study-assigned dose-time interval (ie, there will be 1:1 correspondence regardless of prestudy route of administration \[intravenous \[IV\] or SC\]). Treatment-experienced subjects will be randomized in a 1:1:1 ratio into 1 of 3 parallel cohorts consisting of 18 subjects each as follows: Cohort 1 (daily push compared with every 2 weeks \[biweekly\] pump) will evaluate syringe daily push dosing versus every 2 weeks \[biweekly\] ambulatory pump administration. Subjects will be randomized in a 1:1 ratio to 1 of 2 sequences, either beginning with syringe daily push dosing for 16 weeks and then crossing over to ambulatory pump administration every 2 weeks for 16 weeks or the reverse sequence. Cohort 2 (daily push compared with once weekly pump) will evaluate syringe daily push dosing versus once weekly ambulatory pump administration. Subjects will be randomized in a 1:1 ratio to 1 of 2 sequences, either beginning with syringe daily push dosing for 16 weeks and then crossing over to ambulatory pump administration once every week for 16 weeks or the reverse sequence. Cohort 3 (daily push compared with 2 times/week pump) will evaluate syringe daily push dosing versus 2 times/week ambulatory pump administration. Ambulatory pump dosing will take place on preselected days (eg, Monday and Thursday) not less than 3 days apart. The dosing days should remain constant for the duration of the study. Subjects will be randomized in a 1:1 ratio to 1 of 2 sequences, either beginning with syringe daily push dosing for 16 weeks and then crossing over to ambulatory pump administration 2 times/week for 16 weeks or the reverse sequence. The treatment-naïve cohort will receive a loading dose of 5 consecutive daily doses of IGSC 20% 150 mg/kg/day (Week 0, Days 1-5) followed by weekly infusions of 150 mg/kg starting Week 1 (Day 8) through Week 32 (end of Treatment Phase).

Conditions

• Primary Immunodeficiency

Interventions

Type	Name	Description
BIOLOGICAL	IGSC 20% daily push versus every 2 weeks pump	IGSC 20% syringe daily push dosing for 16 weeks followed by every 2 weeks pump dosing for 16 weeks or the reverse sequence
BIOLOGICAL	IGSC 20% daily push versus once a week pump	IGSC 20% syringe daily push dosing for 16 weeks followed by once weekly ambulatory pump administration dosing for 16 weeks or the reverse sequence
BIOLOGICAL	IGSC 20% daily push versus 2 times per week pump	IGSC 20% syringe daily push dosing for 16 weeks followed by 2 times/week ambulatory pump administration on preselected days (eg, Monday and Thursday) not less than 3 days apart for 16 weeks or the reverse sequence
BIOLOGICAL	IGSC 20% 150 mg/kg	loading dose of IGSC 20% consisting of 5 consecutive daily doses of 150 mg/kg/day (Week 0, Days 1-5) followed by weekly infusions of IGSC 20% 150 mg/kg starting Week 1 (Day 8) through Week 32 using an infusion SC pump

Timeline

• Start date: 2019-12-01
• Primary completion: 2020-09-01
• Completion: 2020-09-01
• First posted: 2019-01-24
• Last updated: 2020-04-10

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study