NCT01485796

Tolerability and Safety of IGI, 10% With rHuPH20 in PIDD

Completed Phase 2, PHASE3 Results posted Last updated 19 May 2021

What this trial tests

Phase 2, PHASE3 trial testing Immune Globulin Infusion (Human), 10% in Primary Immunodeficiency Diseases (PID) in 54 participants. Completed in 1 January 2013.

Timeline

29 December 2011

Primary endpoint
1 January 2013

1 January 2013

Quick facts

Lead sponsor	Baxalta now part of Shire
Phase	Phase 2, PHASE3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	54
Start date	29 December 2011
Primary completion	1 January 2013
Estimated completion	1 January 2013
Sites	10 locations across United States

Drugs / interventions tested

• Immune Globulin Infusion (Human), 10% — full drug profile →
• Recombinant human hyaluronidase — full drug profile →

Conditions studied

• Primary Immunodeficiency Diseases (PID) — all drugs for Primary Immunodeficiency Diseases (PID) →

Sponsor

Baxalta now part of Shire — full company profile →

Who can join

2 and older, any sex, with Primary Immunodeficiency Diseases (PID). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Related Systemic Adverse Events (Excluding Infections) Primary · 7 months (per subject)

Group	Value	95% CI
Safety Analysis Set (n=37)	59

Rate of Related Systemic Adverse Events (Excluding Infections) Per Infusion Primary · 7 months (per subject)

A point estimate and 95% confidence interval for the rate of related systemic adverse events per infusion was derived using a Poisson model.

Epoch 1

Group	Value	95% CI
Safety Analysis Set (n=37)	0.250	0.140 – 0.406
Epoch 2 Data Set (n=36)	0.253	0.141 – 0.413

Epoch 2

Group	Value	95% CI
Safety Analysis Set (n=37)	0.377	0.205 – 0.626
Epoch 2 Data Set (n=36)	0.377	0.205 – 0.626

Epoch 1+2

Group	Value	95% CI
Safety Analysis Set (n=37)	0.324	0.187 – 0.517
Epoch 2 Data Set (n=36)	0.326	0.186 – 0.522

Proportion of Subjects Who Achieve a Treatment Interval of 3 or 4 Weeks in Epoch 2 Secondary · 6 months (per subject)

3 or 4-week treatment interval

Group	Value	95% CI
Epoch 2 Data Set (n=36)	36

3-week treatment interval

Group	Value	95% CI
Epoch 2 Data Set (n=36)	6

4-week treatment interval

Group	Value	95% CI
Epoch 2 Data Set (n=36)	30

Proportion of Subjects Who Maintain a Treatment Interval of 3 or 4 Weeks in Epoch 2 for 24 Weeks Secondary · 6 months (per subject)

Group	Value	95% CI
Epoch 2 Data Set (n=36)	1

Number of Related Local Adverse Events (Excluding Infections) Secondary · 7 months (per subject)

Group	Value	95% CI
Safety Analysis Set (n=37)	153

Rate of Related Local Adverse Events (Excluding Infections) Per Infusion Secondary · 7 months (per subject)

A point estimate and 95% confidence interval for the rate of related local adverse events per infusion was derived using a Poisson model.

Epoch 1

Group	Value	95% CI
Safety Analysis Set (n=37)	0.882	0.581 – 1.271
Epoch 2 Data Set (n=36)	0.880	0.575 – 1.278

Epoch 2

Group	Value	95% CI
Safety Analysis Set (n=37)	0.811	0.541 – 1.160
Epoch 2 Data Set (n=36)	0.811	0.541 – 1.160

Epoch 1+2

Group	Value	95% CI
Safety Analysis Set (n=37)	0.841	0.587 – 1.159
Epoch 2 Data Set (n=36)	0.840	0.582 – 1.164

Number of All Related Adverse Events (Excluding Infections) Secondary · 7 months (per subject)

Group	Value	95% CI
Safety Analysis Set (n=37)	212

Rate of All Adverse Events (Excluding Infections) Per Infusion Secondary · 7 months (per subject)

A point estimate and 95% confidence interval for the rate of adverse events per infusion was derived using a Poisson model.

Epoch 1

Group	Value	95% CI
Safety Analysis Set (n=37)	1.645	1.221 – 2.157
Epoch 2 Data Set (n=36)	1.653	1.221 – 2.178

Epoch 2

Group	Value	95% CI
Safety Analysis Set (n=37)	1.642	1.164 – 2.235
Epoch 2 Data Set (n=36)	1.642	1.164 – 2.235

Epoch 1+2

Group	Value	95% CI
Safety Analysis Set (n=37)	1.643	1.235 – 2.132
Epoch 2 Data Set (n=36)	1.646	1.232 – 2.145

Number of Subjects Who Develop Neutralizing Antibodies to rHuPH20 Secondary · 7 months (per subject)

Group	Value	95% CI
Safety Analysis Set (n=37)	0

Trough Levels of Immunoglobulin G (IgG) Secondary · 7 months (per subject)

IgG trough levels at the beginning of Study Epoch 1 (previous immunoglobulin treatment) and at the end of Study Epoch 2 were analyzed.

At screening

Group	Value	95% CI
Epoch 2 Data Set	10.53	9.46 – 11.73

At end of Epoch 2

Group	Value	95% CI
Epoch 2 Data Set	9.21	8.28 – 10.25

Number of Infusions Per Month in Epoch 1 and Epoch 2 Secondary · 7 months (per subject)

Non-parametric descriptive statistics (median, range) are provided.

Epoch 1

Group	Value	95% CI
Safety Analysis Set (n=37)	2.90	2.5 – 10.1

Epoch 2

Group	Value	95% CI
Safety Analysis Set (n=37)	1.09	0.6 – 2.0

Number of Infusion Sites (Needle Sticks) Per Month in Epoch 1 and Epoch 2 Secondary · 7 months (per subject)

Non-parametric descriptive statistics (median, range) are provided.

Epoch 1

Group	Value	95% CI
Safety Analysis Set (n=37)	2.90	2.5 – 10.1

Epoch 2

Group	Value	95% CI
Safety Analysis Set (n=37)	1.12	0.6 – 3.8

Adverse events — posted to ClinicalTrials.gov

Time frame: Overall: Approximately 1 year Per subject: 7 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Epochs 1+2 (SC Treatment w/ IGI,10% and rHuPH20, n=37)

Serious: 0/37 (0%)

Deaths: —

Safety Follow-up (n=26)

Serious: 1/26 (4%)

Deaths: —

Serious adverse events (1 terms)

Reaction	System	Epochs 1+2 (SC Treatment w…	Safety Follow-up (n=26)
Hypoglycemia	Metabolism and nutrition disorders	—	—

Other adverse events (33 terms — click to expand)

Reaction	System	Epochs 1+2 (SC Treatment w…	Safety Follow-up (n=26)
Infusion site pain	General disorders	—	—
Infusion site erythema	General disorders	—	—
Headache	Nervous system disorders	—	—
Infusion site swelling	General disorders	—	—
Fatigue	General disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Infusion site pruritus	General disorders	—	—
Pyrexia	General disorders	—	—
Sinusitis	Infections and infestations	—	—
Infusion site discoloration	General disorders	—	—
Local swelling	General disorders	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—
Diarrhea	Gastrointestinal disorders	—	—
Pain	General disorders	—	—
Upper respiratory tract infection	Infections and infestations	—	—
Hemosiderinuria	Renal and urinary disorders	—	—
Upper abdominal pain	Gastrointestinal disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Infusion site hematoma	General disorders	—	—
Injection site pain	General disorders	—	—
Injection site reaction	General disorders	—	—
Bronchitis	Infections and infestations	—	—
Pharyngitis	Infections and infestations	—	—
Vulvovaginal mycotic infection	Infections and infestations	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—
Dizziness	Nervous system disorders	—	—
Insomnia	Psychiatric disorders	—	—
Vulvovaginal swelling	Reproductive system and breast disorders	—	—
Nasal congestion	Respiratory, thoracic and mediastinal disorders	—	—
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	—	—
Respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	—	—

Most-reported serious reactions: Hypoglycemia.

Data from ClinicalTrials.gov NCT01485796 adverse events section.

Sponsor's own description

The purpose of the study is to acquire additional data on safety and tolerability of recombinant human hyaluronidase (rHuPH20) facilitated subcutaneous treatment of Immune Globulin Infusion (Human), 10% (IGI, 10%) and to assess the mode of product administration.

Following a discussion with the FDA at the end of July 2012, all participants still active in the study stopped treatment with rHuPH20 to assure safety of the participants participating in the study and went into a safety follow-up.

During this safety follow-up period, participants underwent treatment with the licensed product IGI, 10% (Gammagard Liquid). The intravenous or subcutaneous administration route was at the discretion of the participant and the investigator.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.