Trial Outcomes & Findings for A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency (NCT NCT01289847)

NCT ID: NCT01289847

Last Updated: 2014-12-23

Results Overview

Number of subjects with serious, acute, bacterial infections as a measure of efficacy

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 25 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2014-12-23

Participant Flow

First enrollment: 06 April 2011; Last Subject completed 23 April 2014; Nine recruiting sites globally: United States (US) (seven sites), Chile (one site) and Israel (one site)

This was a Phase IV, multicentre, open-label, non-randomised study. All enrolled subjects received study medication.

Participant milestones

Measure	Gammaplex Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Study STARTED	25
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Gammaplex Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Study Patient could not comply with visits	1

Baseline Characteristics

A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency

Baseline characteristics by cohort

Measure	Gammaplex n=25 Participants Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Age, Customized 2 to 5 year age group	3 participants n=5 Participants
Age, Customized 6 to 11 year age group	12 participants n=5 Participants
Age, Customized 12 to 16 year age group	10 participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Region of Enrollment United States	22 participants n=5 Participants
Region of Enrollment Israel	2 participants n=5 Participants
Region of Enrollment Chile	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: Intent to Treat (ITT)

Number of subjects with serious, acute, bacterial infections as a measure of efficacy

Outcome measures

Measure	Gammaplex n=25 Participants Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Adverse Events	2 participants

SECONDARY outcome

Timeframe: From week 15 onwards

Population: Seven subjects (28.0%) maintained trough IgG levels at all visits that were at least as high as the average of the two previous levels before the first infusion

Number and proportion of subjects who maintain trough IgG levels at least as high as the average of the 2 previous trough levels before the first Gammaplex infusion

Outcome measures

Measure	Gammaplex n=25 Participants Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Therapeutic Efficacy	7 participants

SECONDARY outcome

Timeframe: 12 months

Number of days off school

Outcome measures

Measure	Gammaplex n=25 Participants Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Therapeutic Efficacy	7.8 days Standard Deviation 12.06

SECONDARY outcome

Timeframe: 12 months

Number of days in hospital

Outcome measures

Measure	Gammaplex n=25 Participants Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Therapeutic Efficacy	0.3 days Standard Deviation 0.87

SECONDARY outcome

Timeframe: 12 months

Visits to physicians and/or emergency room

Outcome measures

Measure	Gammaplex n=25 Participants Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Therapeutic Efficacy	4.0 visits Standard Deviation 4.67

SECONDARY outcome

Timeframe: 12 months

Number of days on therapeutic antibiotics

Outcome measures

Measure	Gammaplex n=25 Participants Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Therapeutic Efficacy	32.0 days Standard Deviation 28.28

Adverse Events

Gammaplex

Serious events: 3 serious events

Other events: 25 other events

Deaths: 0 deaths

Serious adverse events

Measure	Gammaplex n=25 participants at risk Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Respiratory, thoracic and mediastinal disorders left lower lobe pneumonia	4.0% 1/25 • Number of events 1 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Gastrointestinal disorders Acute Gastroenterirtis (AGE)	4.0% 1/25 • Number of events 1 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.

Other adverse events

Measure	Gammaplex n=25 participants at risk Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Infections and infestations Nasopharyngitis	32.0% 8/25 • Number of events 11 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Acute sinusitis	28.0% 7/25 • Number of events 9 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Upper respiratory tract infection	24.0% 6/25 • Number of events 8 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Viral upper respiratory tract infection	20.0% 5/25 • Number of events 7 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Pharyngitis streptococcal	16.0% 4/25 • Number of events 4 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Sinusitis	12.0% 3/25 • Number of events 7 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Gastroenteritis viral	12.0% 3/25 • Number of events 4 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Pharyngitis	12.0% 3/25 • Number of events 4 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Bronchitis	8.0% 2/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Lobar pneumonia	8.0% 2/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Bronchitis acute	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Influenza	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Otitis media	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Otitis media acute	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Pneumonia	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Infections and infestations Rhinitis	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Cough	32.0% 8/25 • Number of events 19 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Nasal congestion	32.0% 8/25 • Number of events 17 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	16.0% 4/25 • Number of events 4 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Dyspnoea	12.0% 3/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal pain	12.0% 3/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Epistaxis	8.0% 2/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Nasal oedema	8.0% 2/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Respiratory, thoracic and mediastinal disorders Wheezing	8.0% 2/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
General disorders Pyrexia	36.0% 9/25 • Number of events 15 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
General disorders Fatigue	20.0% 5/25 • Number of events 11 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
General disorders Malaise	20.0% 5/25 • Number of events 7 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
General disorders Chest discomfort	12.0% 3/25 • Number of events 6 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
General disorders Infusion site erythema	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
General disorders Pain	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Nervous system disorders Headache	52.0% 13/25 • Number of events 39 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Gastrointestinal disorders Nausea	24.0% 6/25 • Number of events 8 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Gastrointestinal disorders Vomiting	20.0% 5/25 • Number of events 8 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Gastrointestinal disorders Diarrhoea	12.0% 3/25 • Number of events 4 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Gastrointestinal disorders Abdominal pain upper	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Injury, poisoning and procedural complications Joint sprain	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Injury, poisoning and procedural complications Skin laceration	8.0% 2/25 • Number of events 2 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Musculoskeletal and connective tissue disorders Arthralgia	16.0% 4/25 • Number of events 4 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Musculoskeletal and connective tissue disorders Myalgia	8.0% 2/25 • Number of events 13 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Vascular disorders Hypotension	16.0% 4/25 • Number of events 12 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Vascular disorders Diastolic hypertension	12.0% 3/25 • Number of events 5 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Skin and subcutaneous tissue disorders Eczema	8.0% 2/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Cardiac disorders Tachycardia	12.0% 3/25 • Number of events 6 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Blood and lymphatic system disorders Lymphadenopathy	8.0% 2/25 • Number of events 3 • AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.

Additional Information

Head of Medical Affairs

Bio Products Laboratory

Phone: +44 20 8957 2200

Email: medinfo@bpl.co.uk

Results disclosure agreements

• Principal investigator is a sponsor employee There is an agreement between the PI and Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results. Publication of Study results will be allowed only with prior written approval from Sponsor. At the request of Sponsor, the Institution and/or Investigator shall delete any Confidential Information pertaining to Sponsor's Inventions from any proposed publications prior to submitting or presenting the materials.
• Publication restrictions are in place

Restriction type: OTHER