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

NCT ID: NCT02810444

Last Updated: 2023-07-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 81 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-10-04
• Study Completion Date: 2020-04-01

Brief Summary

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

Conditions

Primary Immunodeficiency Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

BT595

Subjects 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.

Group Type: EXPERIMENTAL

IgG Next Generation (BT595)

Intervention Type: BIOLOGICAL

Interventions

IgG Next Generation (BT595)

Intervention Type: BIOLOGICAL

Other Intervention Names

Immune Globulin Intravenous (Human), 10% Liquid

Eligibility Criteria

Inclusion Criteria

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.

4. Established replacement therapy with any immunoglobulin for intravenous administration (IVIg) reference preparation during the previous 6 months, including documentation of IgG trough levels.

5. 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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Syneos Health

OTHER

Sponsor Role: collaborator

Biotest

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gergely Krivan, MD

Role: PRINCIPAL_INVESTIGATOR

Egyesitett Szent Istvan es Szent Laszlo Korhaz, Budapest, Hungary

Locations

Investigational site # 0104

Birmingham, Alabama, United States

Investigational site # 0116

Los Angeles, California, United States

Investigational site # 0103

Centennial, Colorado, United States

Investigational site # 0114

Thornton, Colorado, United States

Investigational site # 0111

Chicago, Illinois, United States

Investigational site # 0106

South Bend, Indiana, United States

Investigational site # 0105

Toledo, Ohio, United States

Investigational site #0115

Memphis, Tennessee, United States

Investigational Site # 0102

Dallas, Texas, United States

Investigational site # 4902

Frankfurt am Main, , Germany

Investigational site # 4904

Freiburg im Breisgau, , Germany

Investigational site #4905

Leipzig, , Germany

Investigational site # 3602

Budapest, , Hungary

Investigational Site # 3605

Miskolc, , Hungary

Investigational site #3603

Nyíregyháza, , Hungary

Investigational site # 0702

Moscow, , Russia

Investigational site # 0704

Yekaterinburg, , Russia

Investigational site # 3403

Barcelona, , Spain

Investigational site # 3405

Madrid, , Spain

Countries

United States; Germany; Hungary; Russia; Spain

References

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.

Reference Type: RESULT

PMID: 35944615 (View on PubMed)

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.

Reference Type: RESULT

PMID: 36383294 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2015-003652-52

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

991

Identifier Type: -

Identifier Source: org_study_id