Trial Outcomes & Findings for IGIV Study for Chronic ITP Patients Ages 3-70 (NCT NCT00511147)

NCT ID: NCT00511147

Last Updated: 2017-06-12

Results Overview

Defined by the percentage of treated patients in whom platelet counts increase from ≤ 20 x 10\^9/L to ≥ 50 x 10\^9/L by Day 8 ± 1 \[where the day of the first infusion is Day 1\]

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 64 participants
• Primary outcome timeframe: 8 days
• Results posted on: 2017-06-12

Participant Flow

Participant milestones

Measure	IGIV3I Grifols 10% (All Subjects) Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Overall Study STARTED	64
Overall Study COMPLETED	57
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Measure	IGIV3I Grifols 10% (All Subjects) Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Overall Study Withdrawal by Subject	1
Overall Study Disease progression	5
Overall Study Required platelet transfusion	1

Baseline Characteristics

IGIV Study for Chronic ITP Patients Ages 3-70

Baseline characteristics by cohort

Measure	IGIV3I Grifols 10% (All Subjects) n=64 Participants Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Age, Continuous	33.4 years STANDARD_DEVIATION 18.50 • n=5 Participants
Sex: Female, Male Female	43 Participants n=5 Participants
Sex: Female, Male Male	21 Participants n=5 Participants
Race/Ethnicity, Customized Asian	32 Participants n=5 Participants
Race/Ethnicity, Customized Black/African American	2 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic/Latino	6 Participants n=5 Participants
Race/Ethnicity, Customized White	24 Participants n=5 Participants

PRIMARY outcome

Timeframe: 8 days

Population: Modified ITT Population

Defined by the percentage of treated patients in whom platelet counts increase from ≤ 20 x 10^9/L to ≥ 50 x 10^9/L by Day 8 ± 1 [where the day of the first infusion is Day 1]

Outcome measures

Measure	IGIV3I Grifols 10% (All Subjects) n=64 Participants Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Response Rate	81.3 percentage of responders

SECONDARY outcome

Timeframe: 30 days

Population: Number of participants analyzed is based on the number of responding patients (52/64 \[81.3%\]) in the Modified ITT Population

Defined by the number of days elapsed from Day 1 (the day of the first infusion of the IP) to the day when the platelet count is first known to be ≥ 50 x 10^9/L at any moment during the clinical follow-up period ending on Day 30 ± 1

Outcome measures

Measure	IGIV3I Grifols 10% (All Subjects) n=52 Participants Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Time to Platelet Count Recovery	1.7 days Standard Deviation 0.85

SECONDARY outcome

Timeframe: 30 days

Population: Number of participants analyzed is based on the number of responding patients (52/64 \[81.3%\]) in the Modified ITT Population

Defined by the number of consecutive days for which the platelet count remains ≥ 50 x 10^9/L at any moment during the clinical follow-up period ending on Day 30 ± 1.

Outcome measures

Measure	IGIV3I Grifols 10% (All Subjects) n=52 Participants Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Duration of Response	10.8 days Standard Deviation 7.69

SECONDARY outcome

Timeframe: 15 days

Population: Number of participants analyzed is based on the number of patients with hemorrhage/bleeding at Day 1 in the Modified ITT Population

Defined by the percentage of treated patients with hemorrhage/bleedings at Day 1 (i.e., the day of the first infusion, pre-infusion) who improve their diathesis during the clinical follow-up period ending on Day 15 ± 1.

Outcome measures

Measure	IGIV3I Grifols 10% (All Subjects) n=44 Participants Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Regression of Hemorrhage/Bleedings	90.9 percent of subjects with regression

Adverse Events

IGIV3I Grifols 10% (All Subjects)

Serious events: 4 serious events

Other events: 59 other events

Deaths: 0 deaths

Serious adverse events

Measure	IGIV3I Grifols 10% (All Subjects) n=64 participants at risk Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Infections and infestations meningitis aseptic	1.6% 1/64 • 30 days follow-up
General disorders soft tissue inflammation	1.6% 1/64 • 30 days follow-up
Nervous system disorders Headache	3.1% 2/64 • 30 days follow-up

Other adverse events

Measure	IGIV3I Grifols 10% (All Subjects) n=64 participants at risk Intravenous immunoglobulin IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Gastrointestinal disorders Nausea	20.3% 13/64 • 30 days follow-up
Gastrointestinal disorders Vomiting	14.1% 9/64 • 30 days follow-up
General disorders Chills	18.8% 12/64 • 30 days follow-up
General disorders Pyrexia	20.3% 13/64 • 30 days follow-up
Investigations Body temperature increased	10.9% 7/64 • 30 days follow-up
Musculoskeletal and connective tissue disorders Back pain	10.9% 7/64 • 30 days follow-up
Nervous system disorders Dizziness	7.8% 5/64 • 30 days follow-up
Nervous system disorders Headache	64.1% 41/64 • 30 days follow-up
Vascular disorders Hypertension	6.2% 4/64 • 30 days follow-up
Gastrointestinal disorders Diarrhoea	6.2% 4/64 • 30 days follow-up
Injury, poisoning and procedural complications Contusion	12.5% 8/64 • 30 days follow-up
Respiratory, thoracic and mediastinal disorders Epistaxis	6.2% 4/64 • 30 days follow-up
Skin and subcutaneous tissue disorders Petechiae	6.2% 4/64 • 30 days follow-up
Vascular disorders Hypotension	9.4% 6/64 • 30 days follow-up

Additional Information

Henry Li, PhD

Grifols Therapeutics Inc

Phone: 1 + 919 316 6042

Email: henry.li@grifols.com

Results disclosure agreements

• Principal investigator is a sponsor employee Site may publish results from the study, after providing sponsor 30 days' notice prior to submitting a manuscript or other materials related to the study to any outside party. At sponsor's request, site will remove any confidential information (other than study results), and site will upon sponsor's request delay publication or presentation for a period of up to 120 days to allow sponsor to protect its interests in any sponsor inventions
• Publication restrictions are in place

Restriction type: OTHER