Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PID

NCT ID: NCT01175213

Last Updated: 2021-05-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 66 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-07-28
• Study Completion Date: 2013-08-06

Brief Summary

The original purpose of the study is to assess the long-term safety, tolerability, and practicability of the subcutaneous (SC) treatment with Immune Globulin Subcutaneous Solution (IGSC), 10% facilitated with recombinant human hyaluronidase (rHuPH20) in participants with Primary Immunodeficiency Diseases (PID) who have completed Baxter Clinical Study Protocol No. 160603.

Following a discussion with the FDA, all participants still active in the study stopped treatment with rHuPH20 to assure safety of the participants participating in the study and went into a safety follow-up.

During this safety follow-up period, participants underwent either intravenous (IV) or SC treatment with IGSC, 10%. The IV or SC administration route was at the discretion of the participant and the investigator.

Detailed Description

IGSC, 10% is the same product as IMMUNE GLOBULIN INTRAVENOUS (HUMAN), 10% (IGIV, 10%) quoted in study 160603.

IGSC, 10% is abbreviated to IGI, 10% [IMMUNE GLOBULIN INFUSION (HUMAN), 10%]

In the US the product is licensed (trade name GAMMAGARD LIQUID) for the intravenous (IV) and SC replacement therapy of antibody deficiency in patients with PID.

In the EU this product is licensed (trade name KIOVIG)

IGSC, 10% with rHuPH20 established name is Innume Glubulin Infusion 10% (Human) with Recombinant Human Hyualuronidase.

US trade name is HYQVIA EU trade name is HyQvia

Conditions

Primary Immunodeficiency Diseases (PID)

Study Design

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

Study Groups

SC/IGSC, 10% with rHuPH20 followed by SC of IGSC, 10% (safety)

Efficacy and safety of subcutaneous (SC) administration of Immune Globulin Subcutaneous Solution (IGSC), 10% after SC administration of recombinant human hyaluronidase (rHuPH20) followed by Safety of SC of IGSC, 10% only (safety follow-up)

Group Type: EXPERIMENTAL

SC treatment with IGSC, 10% with rHuPH20 followed by SC/IGSC, 10% only (safety)

Intervention Type: BIOLOGICAL

Participants are to continue on the same doses and treatment intervals of IGSC, 10% adjusted for body weight, and rHuPH20 that were used for the last infusions in Study epoch 2 of Study 160603 (NCT00814320).

After 3 infusions, participants are to change treatment to a 2-week interval, if agreed with participant and investigator.

During this safety follow-up period, participants are treated with IGSC, 10% via the SC route. Treatment occurred once every week. The dose was the weekly equivalent of the most recent IV dose (adjusted per body weight) and multiplied by 1.37.

SC/IGSC, 10% with rHuPH20 followed by IV of IGSC, 10% (safety

Efficacy and safety of subcutaneous (SC) administration of Immune Globulin Subcutaneous Solution (IGSC), 10% after SC administration of recombinant human hyaluronidase (rHuPH20) followed by Safety of intravenous (IV) administration of IGSC, 10% only (safety follow-up)

Group Type: EXPERIMENTAL

SC treatment with IGSC, 10% with rHuPH20 followed by IV/IGSC, 10% only (safety)

Intervention Type: BIOLOGICAL

Participants are to continue on the same doses and treatment intervals of IGSC, 10% adjusted for body weight, and rHuPH20 that were used for the last infusions in Study epoch 2 of Study 160603 (NCT00814320).

After 3 infusions, participants are to change treatment to a 2-week interval, if agreed with participant and investigator.

During this safety follow-up period, participants are treated with IGSC, 10% via the intravenous (IV) route. Treatment occurred once every 3-4 weeks. The weekly dose equivalent was 100% of the most recent IV dose.

IV treatment with IGSC, 10% only

Partial efficacy (trough levels of immunoglobulin G \[IgG\] only) and safety of intravenous (IV) administration of IGSC, 10% only. This was for participants enrolled in the study who had anti-rHuPH20 andibody titer from study160603

Group Type: EXPERIMENTAL

IV treatment with IGSC, 10%

Intervention Type: BIOLOGICAL

During this safety follow-up period, participants are treated with IGSC, 10% via the intravenous (IV) route.

Interventions

SC treatment with IGSC, 10% with rHuPH20 followed by SC/IGSC, 10% only (safety)

Participants are to continue on the same doses and treatment intervals of IGSC, 10% adjusted for body weight, and rHuPH20 that were used for the last infusions in Study epoch 2 of Study 160603 (NCT00814320).

After 3 infusions, participants are to change treatment to a 2-week interval, if agreed with participant and investigator.

During this safety follow-up period, participants are treated with IGSC, 10% via the SC route. Treatment occurred once every week. The dose was the weekly equivalent of the most recent IV dose (adjusted per body weight) and multiplied by 1.37.

Intervention Type: BIOLOGICAL

SC treatment with IGSC, 10% with rHuPH20 followed by IV/IGSC, 10% only (safety)

Participants are to continue on the same doses and treatment intervals of IGSC, 10% adjusted for body weight, and rHuPH20 that were used for the last infusions in Study epoch 2 of Study 160603 (NCT00814320).

After 3 infusions, participants are to change treatment to a 2-week interval, if agreed with participant and investigator.

During this safety follow-up period, participants are treated with IGSC, 10% via the intravenous (IV) route. Treatment occurred once every 3-4 weeks. The weekly dose equivalent was 100% of the most recent IV dose.

Intervention Type: BIOLOGICAL

IV treatment with IGSC, 10%

During this safety follow-up period, participants are treated with IGSC, 10% via the intravenous (IV) route.

Intervention Type: BIOLOGICAL

Other Intervention Names

HYQVIA (IGSC; 10% with rHuPH20 [US]); 10% with rHuPH20 [EU]); GAMMAGARD LIQUID (IGSC; 10% [US]); KIOVIG (IGSC; 10% [EU]); GAMMAGARD LIQUID (IGSC; 10% [US]); KIOVIG (IGSC; 10% [EU]); HyQvia (IGSC; 10% with rHuPH20 [EU]); 10% with rHuPH20 [US]); GAMMAGARD LIQUID (US); KIOVIG (EU)

Eligibility Criteria

Inclusion Criteria

• Participant has completed or is about to complete Baxter Clinical Study Protocol No. 160603. Participants who have discontinued rHuPH20 and reverted to intravenous or subcutaneous treatment due to an anti-rHuPH20 antibody also may enroll for long-term safety monitoring.
• Participant/caretaker has reviewed, signed and dated informed consent
• Participant is willing and able to comply with the requirements of the protocol

Exclusion Criteria

• Participant has a serious medical condition such that the participant's safety or medical care would be impacted by participation in Study 160902
• Participant is scheduled to participate in another non-Baxter clinical study involving an investigational product or investigational device during the course of this study
• If female of childbearing potential, participant is pregnant or has a negative pregnancy test and does not agree to employ adequate birth control measures for the duration of the study

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

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Cypress, California, United States

Irvine, California, United States

Los Angeles, California, United States

San Francisco, California, United States

Centennial, Colorado, United States

North Palm Beach, Florida, United States

Atlanta, Georgia, United States

Hinsdale, Illinois, United States

The Bronx, New York, United States

Dallas, Texas, United States

Galveston, Texas, United States

Countries

United States

References

Wasserman RL, Melamed I, Stein MR, Engl W, Sharkhawy M, Leibl H, Puck J, Rubinstein A, Kobrynski L, Gupta S, Grant AJ, Ratnayake A, Richmond WG, Church J, Yel L, Gelmont D. Long-Term Tolerability, Safety, and Efficacy of Recombinant Human Hyaluronidase-Facilitated Subcutaneous Infusion of Human Immunoglobulin for Primary Immunodeficiency. J Clin Immunol. 2016 Aug;36(6):571-82. doi: 10.1007/s10875-016-0298-x. Epub 2016 May 25.

Reference Type: RESULT

PMID: 27220317 (View on PubMed)

Wasserman RL, Gupta S, Stein M, Rabbat CJ, Engl W, Leibl H, Yel L. Infection rates and tolerability of three different immunoglobulin administration modalities in patients with primary immunodeficiency diseases. Immunotherapy. 2022 Mar;14(4):215-224. doi: 10.2217/imt-2021-0256. Epub 2021 Dec 21.

Reference Type: DERIVED

PMID: 34931880 (View on PubMed)

Other Identifiers

160902

Identifier Type: -

Identifier Source: org_study_id