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Primary Immunodeficiency DiseasePhase 3NCT02810444

Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID

A Phase 3 study testing IgG Next Generation (BT595) in people with Primary Immunodeficiency Disease, run by Biotest at 19 sites in 5 countries.

Completed

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

Official record
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Trial summary

This Phase III clinical study is to test efficacy, safety and pharmacokinetics of BT595 in treating patients with Primary Immunodeficiency (PID) Read more about this study

Who is it for?
Ages 2 to 75 years
Conditions the study is for
Primary Immunodeficiency Disease
What is being tested?
IgG Next Generation (BT595)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
81 participants
Where
19 sitesin 5 countries
Study dates
4 Oct 2016to 1 Apr 2020
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 to 75 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Criteria for inclusion:

  1. Written informed consent/assent obtained from subjects/subjects' parent(s) or legally acceptable representative indicating that they understood the purpose of, and procedures required for the study and are willing to participate in it.
  2. Male or female, aged 2 through 75 years, inclusive.
  3. Diagnosis of PID with impaired antibody production, ie:
  • Diagnosis of common variable immunodeficiency (CVID) as defined by the European Society for Immunodeficiencies (ESID)/Pan American Group for Immunodeficiency (PAGID) diagnostic criteria. Or
  • X-linked agammaglobulinaemia (XLA) as defined by ESID/PAGID diagnostic criteria.
  1. Established replacement therapy with any immunoglobulin for intravenous administration (IVIg) reference preparation during the previous 6 months, including documentation of IgG trough levels.
  2. Established replacement therapy with a single IVIg reference preparation for ≥3 months prior to treatment start with BT595 at a 3 week (Q3W) or 4 week (Q4W) schedule with a constant IVIg dose that did not change by ±20% of the mean dose, regular dosage intervals, and at least 1 IgG trough level of ≥5 g/L during the previous 3 months.

Criteria for exclusion:

  1. Pregnancy or unreliable contraceptive measures or lactation period (females only).
  2. Known intolerance to immunoglobulins or comparable substances (eg, vaccination reaction).
  3. Known intolerance to proteins of human origin or known allergic reactions to components of the study product.
  4. Participation in another clinical study within 30 days before entering the study or during the study and/or previous participation in this study.
  5. Employee or direct relative of an employee of the contract research organization, the study site, or Biotest.
  6. Acquired medical conditions known to cause secondary immune deficiency, such as chronic lymphatic leukemia, lymphoma, multiple myeloma, as well as protein losing enteropathies and hypoalbuminemia.
  7. Other medical condition, laboratory finding, or physical examination finding that precludes participation.
  8. Recent febrile illness that precludes or delays participation.
  9. Active infection and receiving antibiotic therapy for the treatment of this infection at the time of screening. Note: if the subject was deemed to be a screen failure due to a nonserious active infection requiring antibiotic therapy, the subject may have been rescreened after the initial screening.
  10. Therapy with systemic steroids or other immunosuppressant drugs at the time of enrollment (current daily use of corticosteroids, ie, >10 mg prednisone equivalent/day for >30 days. Intermittent corticosteroid use during the study was allowable, if medically necessary).
  11. History of thrombotic events (including myocardial infarction, cerebral vascular accident [including stroke], pulmonary embolism, and deep vein thrombosis) within the 6 months before treatment start with BT595 or the presence of significant risk factors for thrombotic events.
  12. Therapy with live-attenuated virus vaccines within 3 months before start of the study.
  13. Selective, absolute immunoglobulin A (IgA) deficiency or known antibodies to IgA.
  14. Positive diagnosis of hepatitis B or hepatitis C.
  15. Positive human immunodeficiency virus (HIV) test.
  16. History of drug or alcohol abuse within the 12 months before treatment start with BT595.
  17. Inability or lacking motivation to participate in the study.

Trial locations and contacts

19 sites in 5 countries.

19 sites in 5 countries

United States9 sites
  • Investigational site # 0104

    Birmingham, Alabama, 35294

  • Investigational site # 0103

    Centennial, Colorado, 80112

  • Investigational site # 0111

    Chicago, Illinois, 60612

  • Investigational Site # 0102

    Dallas, Texas, 75231

  • Investigational site # 0116

    Los Angeles, California, 90027

  • Investigational site #0115

    Memphis, Tennessee, 38103-2800

  • Investigational site # 0106

    South Bend, Indiana, 46617

  • Investigational site # 0114

    Thornton, Colorado, 80233

  • Investigational site # 0105

    Toledo, Ohio, 43617

Other trials in United States

Germany3 sites
  • Investigational site # 4902

    Frankfurt am Main, 60528

  • Investigational site # 4904

    Freiburg im Breisgau, 79106

  • Investigational site #4905

    Leipzig, 04129

Other trials in Germany

Hungary3 sites
  • Investigational site # 3602

    Budapest, 1097

  • Investigational Site # 3605

    Miskolc

  • Investigational site #3603

    Nyíregyháza, 4400

Other trials in Hungary

Russia2 sites
  • Investigational site # 0702

    Moscow, 117198

  • Investigational site # 0704

    Yekaterinburg, 620102

Other trials in Russia

Spain2 sites
  • Investigational site # 3403

    Barcelona, 08035

  • Investigational site # 3405

    Madrid, 28007

Other trials in Spain

About this study

Official title: An Open-label, Prospective, Multicenter Study Investigating Clinical Efficacy, Safety, and Pharmacokinetic Properties of the Human Normal Immunoglobulin for Intravenous Administration BT595 as Replacement Therapy in Patients With Primary Immunodeficiency Disease (PID)

This Phase III clinical study is to test efficacy, safety and pharmacokinetics of BT595 in treating patients with Primary Immunodeficiency (PID)

Being tested
IgG Next Generation (BT595)
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.

  • IgG Next Generation (BT595)Biological

    Also called Immune Globulin Intravenous (Human), 10% Liquid

Study groups in NCT02810444
Study groupRoleWhat they receive
BT595ExperimentalSubjects received BT595 (100 mg/mL human normal immunoglobulin) at doses between 0.2 and 0.8 g per kg body weight (bw) (2 to 8 mL/kg bw), either at a Q3W or Q4W schedule, The initial doses and dosage interval had to be consistent with the subject's prestudy IVIg treatment.
Receives: Biological: IgG Next Generation (BT595)

What is being measured

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

  1. Rate of Acute Serious Bacterial Infections

    The primary efficacy endpoint was the rate of acute serious bacterial infections, ie, the mean number of acute serious bacterial infections [SBIs as defined by EMA and FDA] per subject-year.

    Measured: approx. 12 month treatment period

Secondary outcomes (8)
  1. IgG Trough Levels (Total IgG) Before Each Infusion

    Total IgG levels [g/L] before each infusion, mean (SD)

    Measured: approx. 12 month treatment period

  2. Rate of Any Infections

    The annual rate of infections was calculated as the number of all infections (serious plus nonserious) per subject-year

    Measured: approx. 12 month treatment period

  3. Rate of Nonserious Infections

    The annual rate of nonserious infections was calculated as the number of nonserious infections per subject-year

    Measured: approx. 12 month treatment period

  4. Time to Resolution of Infections

    Time to resolution of infections (days) was calculated as infection stop date - infection start date +1.

    Measured: approx. 12 month treatment period

  5. Antibiotic Treatment Information

    Median (min-max) number of days on antibiotics treatment per subject

    Measured: approx. 12 month treatment period

  6. Rate of Time Lost From School/Work Due to Infections

    Annual rates of the number of days subjects are not able to attend school/work due to infections and their treatment will be calculated per subject-year.

    Measured: approx. 12 month treatment period

  7. Hospitalization / Hospitalization Due to Infection

    Annual rates of the number of days of hospitalization (any hospitalization/ hospitalization due to infection) will be calculated per subject-year.

    Measured: approx. 12 month treatment period

  8. Fever Episodes

    The number of days with episodes of fever will be calculated as the number of fever episodes per subject-year. Fever is defined as a body temperature ≥38°C (≥100.4°F).

    Measured: approx. 12 month treatment period

Timeline

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

  1. First posted23 Jun 2016 (estimated)
  2. Study start4 Oct 2016
  3. Primary completion1 Apr 2020
  4. Study completion1 Apr 2020

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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
81 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02810444
Sponsor's protocol ID
991
Other IDs
2015-003652-52, 2015
Status last verified
Jul 2023
First submitted
15 Jun 2016
Last updated
20 Jul 2023
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Krivan G, Borte M, Harris JB, Lumry WR, Aigner S, Lentze S, Staiger C. Efficacy, safety and pharmacokinetics of a new 10% normal human immunoglobulin for intravenous infusion, BT595, in children and adults with primary immunodeficiency disease. Vox Sang. 2022 Oct;117(10):1153-1162. doi: 10.1111/vox.13337. Epub 2022 Aug 9.

    PubMed 35944615
  • Krivan G, Borte M, Soler-Palacin P, Church JA, Csurke I, Harris JB, Lieberman JA, Melamed IR, Moy JN, Simon R, Aigner S, Lentze S, Staiger C. BT595, a 10% Human Normal Immunoglobulin, for Replacement Therapy of Primary Immunodeficiency Disease: Results of a Subcohort Analysis in Children. J Clin Immunol. 2023 Apr;43(3):557-567. doi: 10.1007/s10875-022-01397-0. Epub 2022 Nov 16.

    PubMed 36383294

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing IgG Next Generation (BT595) in people with Primary Immunodeficiency Disease, run by Biotest at 19 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?

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 20 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States, Germany, Hungary, Russia, Spain. 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 phase mean?

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?

Yes. Results are posted in the registry (first posted 12 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Rate of Acute Serious Bacterial Infections (time frame: approx. 12 month treatment period). 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 NCT02810444, which also lists 2 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 20 Jul 2023.

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