Trial Outcomes & Findings for Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP) (NCT NCT03866798)

Results Overview

Number of subjects with an increase in platelet count at least once to ≥50 × 10\^9/L within 7 days after the first infusion, i.e., by Day 8 (increase must have occurred at least once on any day up to and including Day 8).

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

6 participants

Primary outcome timeframe

8 Days

Results posted on

2024-09-24

Participant Flow

Six subjects aged ≥1 year to \<18 years were recruited between 21-Jan-2020 and 19-Sep-2023. The subjects were enrolled at five research sites across the United States, with a total of eight research sites activated in the study.

Participant milestones

Participant milestones
Measure	Panzyga Panzyga Panzyga: Immune Globulin, intravenous, human-ifas
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Panzyga n=6 Participants Panzyga Panzyga: Immune Globulin, intravenous, human-ifas
Age, Categorical <=18 years	6 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants

PRIMARY outcome

Timeframe: 8 Days

Number of subjects with an increase in platelet count at least once to ≥50 × 10\^9/L within 7 days after the first infusion, i.e., by Day 8 (increase must have occurred at least once on any day up to and including Day 8).

Outcome measures

Outcome measures
Measure	Panzyga n=6 Participants Panzyga Panzyga: Immune Globulin, intravenous, human-ifas
Platelet Count Increase	4 Participants

SECONDARY outcome

Timeframe: Through study completion, up to 37 days

Number of days for subjects to reach Platelet Count of at least 50x10\^9/L after infusion

Outcome measures

Outcome measures
Measure	Panzyga n=6 Participants Panzyga Panzyga: Immune Globulin, intravenous, human-ifas
Days to Reach Platelet Count of at Least 50x10^9/L 2 days	3 Participants
Days to Reach Platelet Count of at Least 50x10^9/L 3 days	1 Participants
Days to Reach Platelet Count of at Least 50x10^9/L Did not reach Platelet Count of at least 50x10^9/L	2 Participants

SECONDARY outcome

Timeframe: Through study completion, up to 37 days

Number of days the platelet count remains above at least 50x10\^9/L

Outcome measures

Outcome measures
Measure	Panzyga n=6 Participants Panzyga Panzyga: Immune Globulin, intravenous, human-ifas
Duration of Platelet Response 6 days	1 Participants
Duration of Platelet Response 7 days	1 Participants
Duration of Platelet Response 33 days	1 Participants
Duration of Platelet Response 34 days	1 Participants
Duration of Platelet Response Did not reach Platelet Count of at least 50x10^9/L	2 Participants

SECONDARY outcome

Timeframe: Through study completion, up to 37 days

Maximum platelet count (10\^9/L) for each subjects

Outcome measures

Outcome measures
Measure	Panzyga n=6 Participants Panzyga Panzyga: Immune Globulin, intravenous, human-ifas
Maximum Platelet Count 99 (10^9/L)	1 Participants
Maximum Platelet Count 11 (10^9/L)	1 Participants
Maximum Platelet Count 516 (10^9/L)	1 Participants
Maximum Platelet Count 386 (10^9/L)	1 Participants
Maximum Platelet Count 204 (10^9/L)	1 Participants
Maximum Platelet Count 30 (10^9/L)	1 Participants

Adverse Events

Panzyga

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Panzyga n=6 participants at risk Panzyga Panzyga: Immune Globulin, intravenous, human-ifas
Nervous system disorders Headache	33.3% 2/6 • Number of events 2 • 3 years, 9 months
Musculoskeletal and connective tissue disorders Myalgia	16.7% 1/6 • Number of events 1 • 3 years, 9 months
Gastrointestinal disorders Nausea	33.3% 2/6 • Number of events 2 • 3 years, 9 months
Gastrointestinal disorders Vomiting	33.3% 2/6 • Number of events 2 • 3 years, 9 months
Blood and lymphatic system disorders Neutropenia	16.7% 1/6 • Number of events 1 • 3 years, 9 months
Respiratory, thoracic and mediastinal disorders Epistaxis	16.7% 1/6 • Number of events 1 • 3 years, 9 months
Gastrointestinal disorders Mouth haemorrhade	16.7% 1/6 • Number of events 2 • 3 years, 9 months
Gastrointestinal disorders Dental Caries	16.7% 1/6 • Number of events 1 • 3 years, 9 months
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	16.7% 1/6 • Number of events 1 • 3 years, 9 months
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	16.7% 1/6 • Number of events 1 • 3 years, 9 months

Additional Information

Patrick Murphy

Clinical Research Management Group

Phone: 4138210022

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place