Clinical study to evaluate the safety and tolerability of immunoglobulin intravenous (human) 10% (NewGam) administered at high infusion rates to patients with primary immunodeficiency diseases
| ISRCTN | ISRCTN11002924 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN11002924 |
| EudraCT/CTIS number | 2011-005015-82 |
| ClinicalTrials.gov number | NCT01313507 |
| Secondary identifying numbers | NGAM-05 (extension of study NGAM 01) |
- Submission date
- 08/03/2011
- Registration date
- 16/03/2011
- Last edited
- 15/03/2019
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Other
Plain English summary of protocol
Background and study aims
This study aims to find a treatment for Primary Immunodeficiency Diseases (PID). According to the leading experts in immunology, when part of the immune system (body mechanism that protects the body against infection caused by bacteria, viruses, fungi or parasites) is either absent or not functioning properly, it can result in an immune deficiency disease. When the cause of this deficiency is hereditary or genetic, it is called a primary immunodeficiency disease (PID). One of the most common signs of PID is an increased susceptibility to infections. You may have infections that are more frequent, longer lasting or harder to treat than are the infections of someone with a normal immune system. You may also get infections that a person with a healthy immune system likely wouldn't get (called opportunistic infections).
One of the common treatments for PID is Immunoglobulin therapy. Immunoglobulin consists of antibody proteins needed for the immune system to fight infections. It can be injected into a vein through an intravenous (IV) line. Treatment with IV immunoglobulin is needed every few weeks to maintain sufficient levels of antibodies in your blood.
Who can participate?
You may only participate in the NGAM-05 study if you completed the NGAM-01 study and did not notice any side effects during your last few IV immunoglobulin infusions of that study.
What does the study Involve?
This study, named NGAM-05, is an extension of the NGAM-01 study that was testing a new formulation of IV Immunoglobulin called NewGam. In the NGAM-05 study we are testing the same NewGam and you will receive the same dose as in NGAM-01. The difference is that the NewGam is given to you at a faster speed and during a shorter time than the usual medicine is given to you. You will receive either four infusions if on a 4 week infusion schedule, or five infusions if on a 3-week infusion schedule, and a follow-up visit.
What are the possible benefits and risks of participating?
The benefit of being in this study is that your infusions will go faster than normal, and your visit to the doctors office will take less time than normal.
The risks of being in this study are that you may experience more side effects than normal. It is felt that the faster you get the medicine, usually the risk of side effects goes up. The most common side effects when people take IV immunoglobulin are headache, chills, migraine, dizziness, fever, nausea/vomiting, fatigue, faster heart rate, Itching, upper abdominal pain, rash/hives, increased blood pressure and cough.
Where is the study run from?
This study is being run at six different centers in the USA.
When is the study starting and how long is it expected to run for?
This study started in May 2011 and is expected to last until September 2012.
Who is funding the study?
Octapharma.
Who is the main contact?
Barbara Pyringer
barbara.pyringer@octapharma.com
Contact information
Scientific
Oberlaaer Str. 235
Vienna
1100
Austria
Study information
| Study design | Prospective open-label non-controlled non-randomised multicentre Phase III study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Other |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details below to request a patient information sheet |
| Scientific title | Clinical study to evaluate the safety and tolerability of immunoglobulin intravenous (human) 10% (NewGam) administered at high infusion rates to patients with primary immunodeficiency diseases: a prospective, open label, non-controlled, non-randomised, multicentre, phase III study |
| Study objectives | To assess the safety and tolerability of NewGam when administered at infusion rates from 0.08 mL/kg/min to 0.14 mL/kg/min Extension of study NGAM 01 registered under http://www.controlled-trials.com/ISRCTN05425999 |
| Ethics approval(s) | 1. Research and Clinical Trials Administration Office, Rush University Medical Parkway, approval granted on 04/03/2011 2. Western IRB, approval granted on 16/03/2011 3. St. Louis University Biomedical Institutional Review Board, approval granted on 05/04/2011 4. Institutional Review board, Seattle Childrens Hospital, approval granted on 22/12/2011 5. Office of Research Administration, University of California Irvine, approval granted on 08/04/2011 |
| Health condition(s) or problem(s) studied | Primary immunodeficiency (PID) diseases |
| Intervention | Patients will receive NewGam every 3 or 4 weeks (+/-3 days) following the same dosing interval as in the main study NGAM 01. Patients who complete the present study will receive either 5 (at 3-week intervals) or 4 (at 4-week intervals) infusions of NewGam. |
| Intervention type | Other |
| Primary outcome measure | 1. Occurrence of adverse events (AEs) 2. Occurrence of AEs temporally associated with the study treatment 3. Proportion of infusions with one or more temporally associated AEs 4. AEs by infusion rate 5. Short-term tolerance parameters including vital signs (blood pressure, heart rate, temperature, respiratory rate) 6. Laboratory parameters (haematology, clinical chemistry, direct Coombs' test, urinalysis, and tests for viral safety) |
| Secondary outcome measures | Quality of Life: For QoL assessments, each patient will continue using the same questionnaire as before. That is, the parent or guardian of patients who were below 14 years of age when they entered the main study NGAM 01 will continue using the Child Health Questionnaire-Parent Form (CHQ-PF50), and patients who were ≥ 14 years of age when they entered the main study will continue using the SF-36 Health Survey |
| Overall study start date | 15/03/2011 |
| Completion date | 30/06/2012 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 20-35 |
| Key inclusion criteria | 1. Completion of the main study NGAM 01 2. At each of the last three infusions in the main study NGAM 01, administration of NewGam at the maximum infusion rate of 0.08 mL/kg/min and without the need for premedication 3. For adult patients: freely given written informed consent. For patients below the legal age of majority: freely given written informed consent from parents/legal guardians and written informed assent from the child/adolescent in accordance with the applicable approvals 4. For female patients of child-bearing potential, a negative result in a urine pregnancy test conducted at the screening visit 5. Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study |
| Key exclusion criteria | 1. Any condition or circumstance that would have led to the exclusion of the subject from the NGAM 01 study 2. Administration of any immunoglobulin infusion other than NewGam between conclusion of the NGAM 01 study and the beginning of the present study 3. A deviation of the subjects treatment interval of more than 7 days between the last infusion of NewGam in the NGAM 01 study and the first infusion of NewGam in the present study |
| Date of first enrolment | 15/03/2011 |
| Date of final enrolment | 30/06/2012 |
Locations
Countries of recruitment
- Austria
- United States of America
Study participating centre
1100
Austria
Sponsor information
Industry
Seidenstrasse 2
Lachen
8853
Switzerland
"ROR" |
https://ror.org/002k5fe57 |
|---|
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Basic results | No | No | |||
| Basic results | No | No |
Editorial Notes
15/03/2019: The following changes were made to the trial record:
1. Added clinicaltrials.gov link to basic results (scientific).
2. Added EudraCT link to basic results (scientific).
