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Primary Immunodeficiency Diseases (PID)Phase 2/3NCT01412385

Immune Globulin Subcutaenous (Human), 20%

A Phase 2/3 study testing Immune Globulin Subcutaneous (Human), 20%, Immune Globulin Intravenous (Human), 10% and Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin) in people with Primary Immunodeficiency Diseases (PID), run by Baxalta now part of Shire at 12 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The purpose of the study is to develop a 20% subcutaneous immunoglobulin treatment option for patients with primary immunodeficiency (PID) diseases. Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Primary Immunodeficiency Diseases (PID)
What is being tested?
Immune Globulin Subcutaneous (Human), 20%, Immune Globulin Intravenous (Human), 10% and Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
55 participants
Where
12 sitesin 5 countries
Study dates
20 Jun 2011to 13 May 2014
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Subject must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement, as defined according to the IUIS Scientific Committee 2009, and by diagnostic criteria according to Conley ME, Notarangelo LD, Etzioni A. Diagnostic criteria for primary immunodeficiencies. Clin Immunol 1999; 93:190-197. The diagnosis must be confirmed by the Medical Director prior to enrollment.
  • Subject is 2 years or older at the time of screening
  • Written informed consent is obtained from either the subject or the subject's legally authorized representative prior to any study-related procedures and study product administration
  • Subject has been receiving a consistent dose of IgG over a period of at least 3 months prior to screening at an average minimum dose over that interval equivalent to 300 mg/kg body weight (BW)/4 weeks and a maximum dose equivalent to 1.0 gram/kg BW/4 weeks at a dosing frequency as follows:
  • intravenously (IV) at mean intervals of approximately 3 or 4 weeks or
  • subcutaneously (SC) at mean intervals of approximately 1 or 2 weeks
Show all 9Show fewer
  • Subject has a serum trough level of IgG > 5 g/L at screening
  • Subject has not had a serious bacterial infection within the 3 months prior to screening
  • Subject is willing and able to comply with the requirements of the protocol

Who cannot join

  • Subject has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2
  • Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):
  • Persistent alanine aminotransferase (ALT) and aspartate amino transferase (AST) > 2.5 times the upper limit of normal for the testing laboratory
  • Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] <= 500 /mm3)
  • Subject has creatinine clearance (CLcr) value that is < 60% of normal for age and gender
  • Subject has been diagnosed with or has a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix), unless the disease-free period prior to screening exceeds 5 years
Show all 23Show fewer
  • Subject is receiving anti-coagulation therapy or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within 12 months prior to screening or a history of thrombophilia
  • Subject has abnormal protein loss (protein losing enteropathy, nephrotic syndrome)
  • Subject has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site
  • Subject has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IV immunoglobulin, SC immunoglobulin, and/or Immune Serum Globulin (ISG) infusions
  • Subject has immunoglobulin A (IgA) deficiency (IgA less than 0.07g/L) and known anti IgA antibodies
  • Subject is on preventative (prophylactic) systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and cannot stop these antibiotics at the time of screening
  • Subject has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening
  • Subject has a bleeding disorder or a platelet count less than 20,000/μL, or who, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of subcutaneous therapy
  • Subject has total protein >9 g/dL or myeloma, or macroglobulinemia (IgM) or paraproteinemia
  • Women of childbearing potential meeting any one of the following criteria
  • subject presents with a positive pregnancy test
  • subject is breast feeding
  • subject intends to begin nursing during the course of the study
  • subject does not agree to employ adequate birth-control measures (e.g. intrauterine device, diaphragm or condom [for male partner] with spermicidal jelly or foam, or birth control pills/patches) throughout the course of the study
  • Subject has participated in another clinical study and has been exposed to an investigational product (IP) or device within 30 days prior to study enrollment (exception: treatment with immunoglobulin pre-study)
  • Subject is scheduled to participate in another (non-Baxter) non-observational (interventional) clinical study involving an IP or device during the course of the study
  • Subject has severe dermatitis that would preclude adequate sites for safe product administration

Trial locations and contacts

12 sites in 5 countries.

12 sites in 5 countries

Germany5 sites

  • Universitätsklinikum Erlangen, Medizinische Klinik 3

    Erlangen, 91054

  • University Medical Centre Freiburg, Centre of Chronic Immunodeficiency, Divison of Rheumatology and Clinical Immunology

    Freiburg im Breisgau, 79106

  • Universitätsklinikum Hamburg-Eppendorf, Kinderklinik

    Hamburg, 20246

  • Medizinische Hochschule Hannover, Klinik für Immunologie und Rheumatologie

    Hanover, 30625

  • Klinikum St. Georg GmbH, Klinik für Kinder- und Jugendmedizin

    Leipzig, 04129

Other trials in Germany

United Kingdom3 sites

  • Birmingham Heartlands Hospital, Heart of England NHS Foundation Trust, Immunology Department

    Birmingham, B9 5SS

  • Addenbrooke´s Hospital, Department of Clinical Immunology

    Cambridge, CB2 2QQ

  • Royal London Hospital, Barts and the London NHS Trust, Department of Immunology

    London, E1 2ES

Other trials in United Kingdom

Hungary2 sites

  • Fővárosi Önkormányzat Egyesített Szent István és Szent László Kórház, Gyermekhematológiai és Őssejt-transzplantációs Osztály

    Budapest, 1097

  • University of Debrecen, Medical and Health Science Center, Department of Infectious and Pediatric Immunology

    Debrecen, 4012

Other trials in Hungary

Austria1 site

  • Medizinische Universität Wien / AHK Wien (General Hospital Vienna), Universitätsklinik für Kinder- und Jugendheilkunde

    Vienna, 1090

Other trials in Austria

Sweden1 site

  • The Queen Silvia Children´s Hospital

    Gothenburg, 416 85

Other trials in Sweden

About this study

Official title: A Clinical Study of Immune Globulin Subcutaneous (Human) (IGSC), 20% for the Evaluation of Efficacy, Safety, and Pharmacokinetics in Subjects With Primary Immunodeficiency Diseases

The purpose of the study is to develop a 20% subcutaneous immunoglobulin treatment option for patients with primary immunodeficiency (PID) diseases.

Being tested
Immune Globulin Subcutaneous (Human), 20%, Immune Globulin Intravenous (Human), 10%, Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin)
Medical subject headings
Primary Immunodeficiency Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Immune Globulin Subcutaneous (Human), 20%Biological

    Also called IGSC, 20%

    Subcutaneous infusion (regulated via a pump), Epoch 2 only (all subjects)

  • Immune Globulin Intravenous (Human), 10%Biological

    Also called GAMMAGARD LIQUID (tradename in the US and Canada), KIOVIG (trademark in Europe)

    Intravenous infusion (regulated via a pump)

  • Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin)Biological

    Also called SUBCUVIA

    Subcutaneous infusion (regulated via a pump)

Study groups in NCT01412385
Study groupRoleWhat they receive
Epoch 1 (intravenous pre-study treatment) + Epoch 2ExperimentalStudy Epoch 1 (13 weeks): treatment with KIOVIG (once every 3 or 4 weeks, dose as during pre-study period) + Study Epoch 2 (same for all subjects, 51 weeks): treatment with IGSC, 20% (every week, dose to be calculated on the basis of weekly equivalents)
Receives: Biological: Immune Globulin Subcutaneous (Human), 20%; Biological: Immune Globulin Intravenous (Human), 10%
Epoch 1 (subcutaneous pre-study treatment) + Epoch 2ExperimentalStudy Epoch 1 (12 weeks): treatment with SUBCUVIA (once every week or once every two weeks, dose as during pre-study period) + Study Epoch 2 (same for all subjects, 51 weeks): treatment with IGSC, 20% (every week, dose to be calculated on the basis of weekly equivalents)
Receives: Biological: Immune Globulin Subcutaneous (Human), 20%; Biological: Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Acute serious bacterial infection rate defined as the mean number of acute serious bacterial infections per subject per year in the intent-to-treat population

    Acute serious bacterial infections will include bacteremia / sepsis, bacterial meningitis, osteomyelitis / septic arthritis, bacterial pneumonia, and visceral abscess, diagnosed according to the Diagnostic Criteria for Serious Acute Bacterial Infections

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Aug 2011 (estimated)
  2. Study start20 Jun 2011
  3. Primary completion13 May 2014
  4. Study completion13 May 2014

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
55 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01412385
Sponsor's protocol ID
170903
Other IDs
2010-019459-23
Status last verified
Apr 2021
First submitted
8 Aug 2011
Last updated
5 May 2021
Results posted
No
Data monitoring committee
No
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Publications

  • Borte M, Krivan G, Derfalvi B, Marodi L, Harrer T, Jolles S, Bourgeois C, Engl W, Leibl H, McCoy B, Gelmont D, Yel L. Efficacy, safety, tolerability and pharmacokinetics of a novel human immune globulin subcutaneous, 20%: a Phase 2/3 study in Europe in patients with primary immunodeficiencies. Clin Exp Immunol. 2017 Jan;187(1):146-159. doi: 10.1111/cei.12866. Epub 2016 Oct 18.

    PubMed 27613250

  • Li Z, McCoy B, Engl W, Yel L. Steady-State Serum IgG Trough Levels Are Adequate for Pharmacokinetic Assessment in Patients with Immunodeficiencies Receiving Subcutaneous Immune Globulin. J Clin Immunol. 2021 Aug;41(6):1331-1338. doi: 10.1007/s10875-021-00990-z. Epub 2021 May 26.

    PubMed 34036490

  • Suez D, Krivan G, Jolles S, Stein M, Gupta S, Paris K, van Hagen PM, Brodszki N, Engl W, Leibl H, McCoy B, Yel L. Safety and tolerability of subcutaneous immunoglobulin 20% in primary immunodeficiency diseases from two continents. Immunotherapy. 2019 Aug;11(12):1057-1065. doi: 10.2217/imt-2019-0057. Epub 2019 Jul 3.

    PubMed 31268374

  • Paris K, Haddad E, Borte M, Brodszki N, Derfalvi B, Marodi L, Hussain I, Darter A, Engl W, Leibl H, McCoy B, Yel L. Tolerability of subcutaneous immunoglobulin 20%, Ig20Gly, in pediatric patients with primary immunodeficiencies. Immunotherapy. 2019 Apr;11(5):397-406. doi: 10.2217/imt-2018-0088. Epub 2019 Jan 9.

    PubMed 30626238

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Immune Globulin Subcutaneous (Human), 20%, Immune Globulin Intravenous (Human), 10% and Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin) in people with Primary Immunodeficiency Diseases (PID), run by Baxalta now part of Shire at 12 sites in 5 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in Germany, United Kingdom, Hungary, Austria, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Acute serious bacterial infection rate defined as the mean number of acute serious bacterial infections per subject per year in the intent-to-treat population (time frame: 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01412385, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2021.

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