Phase III Efficacy, Safety, and Tolerability Study of HYQVIA/HyQvia and GAMMAGARD LIQUID/KIOVIG in CIDP

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Brief Title

A Study of HyQvia and Gammagard Liquid (Kiovig) in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Official Title

A Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Immune Globulin Infusion 10% (Human) With Recombinant Human Hyaluronidase (HYQVIA/HyQvia) and Immune Globulin Infusion (Human), 10% (GAMMAGARD LIQUID/KIOVIG) for the Treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Brief Summary

      The aim of this study is to learn more about the following treatment options in adults with
      CIDP:

        -  Subcutaneous self-infusion with HyQvia.

        -  Intravenous infusion with Gammagard/Kiovig. Gammagard and Kiovig are the brand names for
           the same immunoglobulin compound.

      The study is in two parts. In Part 1, participants receive either HyQvia or a placebo
      subcutaneously. In Part 2 (only for participants who have a CIPD relapse during Part 1),
      participants will receive Gammagard Liquid/Kiovig intravenously. US participants will receive
      Gamunex-C.

      The first SC infusion will be given in the study clinic. The remaining SC infusions may be
      given in the study clinic or the participant's home. This will be decided by the study doctor
      and whether the participant or their caregiver can do the self-infusion.
    

Detailed Description

      Phase III Efficacy, Safety, and Tolerability Study of HYQVIA/HyQvia and GAMMAGARD
      LIQUID/KIOVIG in CIDP
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Epoch 1: Relapse Rate on Every 2 Weeks Dosing Regimen (Q2W) at Pre-SC Baseline Week 1

Secondary Outcome

 Epoch 1: Percentage of Participants Who Experience a Worsening of Functional Disability

Condition

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Intervention

HYQVIA

Study Arms / Comparison Groups

 Epoch 1: HYQVIA/HyQvia
Description:  Participants will receive HYQVIA/HyQvia at a dose of 80 Unit per gram (U/g) SC immunoglobulin (IgG) which will be same as the participants pre-randomization monthly equivalent IgG dose (or at matching infusion volume for participants in the placebo group) when administered at a dosing frequency of every 2, 3, or 4 weeks for 6 months or until relapse.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

138

Start Date

December 15, 2015

Completion Date

February 23, 2022

Primary Completion Date

February 23, 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Males or females of age greater than or equal to (>=)18 years old at the time of
             screening.

          2. Participant has a documented diagnosis of definite or probable Chronic inflammatory
             demyelinating polyradiculoneuropathy (CIDP) (focal atypical CIDP and pure sensory
             atypical CIDP will be excluded), as confirmed by a neurologist
             specializing/experienced in neuromuscular diseases to be consistent with the European
             Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) 2010 criteria
             (European Federation of Neurological Societies, 2010). Fulfillment of
             electrodiagnostic criteria must be confirmed by an independent qualified/experienced
             central reader.

          3. Participant has responded to IgG treatment in the past (partial or complete resolution
             of neurological symptoms and deficits), and must currently be on stable doses of IGIV
             treatment within the dose range equivalent to a cumulative monthly dose of 0.4 to 2.4
             gram per kilogram (g/kg) BW (inclusive) administered intravenously for at least 12
             weeks prior to screening. The dosing interval of IGIV treatment must be between 2 and
             6 weeks (inclusive). Variations in the dosing interval of up to ± 7 days or monthly
             dose amount of up to ± 20% between participant's pre-study Immunoglobulin G (IgG)
             infusions are within acceptable limits.

          4. INCAT disability score between 0 and 7 (inclusive). Participants with INCAT scores of
             0, 1 (whether from upper or lower extremities), or 2 (if at least 1 point is from an
             upper extremity) at screening and/or baseline will be required to have a history of
             significant disability as defined by an INCAT disability score of 2 (must be
             exclusively from the lower extremities) or greater documented in the medical record.
             Participants will be eligible if one of the below eligibility criteria are met:

             Screening and Baseline INCAT disability score of between 3 and 7 inclusive.

               1. Screening and/or Baseline INCAT disability score of 2 (both points are from lower
                  extremities).

               2. Screening and/or Baseline INCAT disability score of 2 (both points are not from
                  lower extremities) AND has at least a score of 2 or greater documented in the
                  medical record prior to screening. If a score was greater than 2 documented in
                  the medical record prior to screening at least 2 points must be from lower
                  extremities.

               3. Screening and/or Baseline INCAT disability score of 0 or 1 AND has at least a
                  score of 2 or greater (both from lower extremities) documented in the medical
                  record prior to screening, at least 2 points must be from lower extremities.

          5. If female of childbearing potential, the participant must have a negative pregnancy
             test at screening and agree to employ a highly effective contraceptive measure
             throughout the course of the study and for at least 30 days after the last
             administration of investigational product (IP).

          6. Participant is willing and able to sign an Informed Consent Form (ICF).

          7. Participant is willing and able to comply with the requirements of the protocol.

        Exclusion Criteria:

          1. Participants with Focal atypical CIDP or pure sensory atypical CIDP.

          2. Any neuropathy of other causes, including:

               1. Hereditary demyelinating neuropathies, such as hereditary sensory and motor
                  neuropathy (HSMN) (Charcot-Marie-Tooth [CMT] disease), and hereditary sensory and
                  autonomic neuropathies (HSANs).

               2. Neuropathies secondary to infections, disorders, or systemic diseases such as
                  Borrelia burgdorferi infection (Lyme disease), diphtheria, systemic lupus
                  erythematosus, POEMS (polyneuropathy, organomegaly, endocrinopathy, M-protein,
                  and skin changes) syndrome, osteosclerotic myeloma, diabetic and non-diabetic
                  lumbosacral radiculoplexus neuropathy, lymphoma, and amyloidosis.

               3. Multifocal acquired demyelinating sensory and motor neuropathy (MADSAM).

               4. Multifocal motor neuropathy (MMN).

               5. Drug-, biologic-, chemotherapy-, or toxin-induced peripheral neuropathy.

          3. Immunoglobulin M (IgM) paraproteinemia, including IgM monoclonal gammopathy with high
             titer antibodies to myelin-associated glycoprotein.

          4. Prominent sphincter disturbance.

          5. Central demyelinating disorders (eg, multiple sclerosis).

          6. Any chronic or debilitating disease, or central nervous disorder that causes
             neurological symptoms or may interfere with assessment of CIDP or outcome measures
             (eg, arthritis, stroke, Parkinson's disease, and diabetic peripheral neuropathy)
             (Participants with clinically diagnosed diabetes mellitus who do not have diabetic
             peripheral neuropathy, who have adequate glycemic control with Hemoglobin A1C; also
             known as glycosylated or glycated hemoglobin (HbA1C) of less than (<) 7.5% at
             screening, and who agree to maintain adequate glycemic control during the study are
             allowed).

          7. Congestive heart failure (New York Heart Association [NYHA] Class III/IV), unstable
             angina, unstable cardiac arrhythmias, or uncontrolled hypertension (ie, diastolic
             blood pressure greater than (>) 100 millimeter of mercury (mmHg) and/or systolic blood
             pressure >160 mmHg).

          8. History of deep vein thrombosis or thromboembolic events (eg, cerebrovascular
             accident, pulmonary embolism) in the past 12 months.

          9. Condition(s) which could alter protein catabolism and/or IgG utilization (eg,
             protein-losing enteropathies, nephrotic syndrome).

         10. Known history of chronic kidney disease, or glomerular filtration rate (GFR) of <60
             milliliter per minute per 1.73 square meter (mL/min/1.73m^2) estimated based on
             CKD-EPI equation (Levey et al., 2009) at the time of screening.

         11. Participant with active malignancy requiring chemotherapy and/or radiotherapy, or
             history of malignancy with less than 2 years of complete remission prior to screening.
             Exceptions are: adequately treated basal cell or squamous cell carcinoma of the skin,
             carcinoma in situ of the cervix, and stable prostate cancer not requiring treatment.

         12. Clinically significant anemia or hemoglobin (Hgb) level of less than (<) 10.0 grams
             per deciliter (g/dL) at screening.

         13. Hypersensitivity or adverse reactions (AR's) (eg, urticaria, breathing difficulty,
             severe hypotension, or anaphylaxis) to human blood products such as human IgG,
             albumin, or other blood components.

         14. Known allergy to hyaluronidase of human (including recombinant human hyaluronidase) or
             animal origin (such as bee or wasp venom).

         15. Known history of or immunoglobulin A (IgA) deficiency (<8 milligram per deciliter
             [mg/dL]) at screening.

         16. Abnormal laboratory values at screening:

               1. Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) > 2.5*
                  upper limit of normal (ULN)

               2. Platelet count <100,000 cells per microliter (cells/mcL).

               3. Absolute neutrophil count (ANC) <1000 cells/mcL.

         17. Ongoing/active infection with hepatitis A virus (HAV), hepatitis B virus (HBV),
             hepatitis C virus (HCV), or human immunodeficiency virus (HIV) Type 1/2 infection.

         18. The participant has received or is currently receiving treatment with
             immunomodulatory/ immunosuppressive agents within 6 months prior to screening.

         19. Participant has received or is currently receiving treatment with corticosteroids dose
             within 8 weeks prior to screening, regardless of indication.

         20. Participant has undergone plasma exchange (PE) within 3 months prior to screening.

         21. The participant has any disorder or condition that in the investigator's judgment may
             impede the participant's participation in the study, pose increased risk to the
             participant, or confound the results of the study.

         22. The participant is nursing or intends to begin nursing during the course of the study.

         23. Participant has participated in another clinical study involving an IP or
             investigational device within 30 days prior to enrollment, or is scheduled to
             participate in another clinical study (with the exception of the HYQVIA/HyQvia
             extension study in CIDP) involving an IP or investigational device during the course
             of this study.

         24. The participant is a family member or employee of the investigator.

         25. Participants with acquired or inherited thrombophilic disorders. These will include
             the specific types of acquired or inherited thrombophilic disorders that could put
             participants at risk of develop thrombotic events. Examples include:

             a. Hereditary Thrombophilias i. Factor V Leiden mutation. ii. Prothrombin 20210A
             mutation. iii. Protein C deficiency. iv. Protein S deficiency. v. Antithrombin
             deficiency. b. Acquired thrombophilias i. Antiphospholipid antibody syndrome. ii.
             Activated protein C Resistance acquired. iii. Homocystinemia.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Study Director, , 

Location Countries

Argentina

Location Countries

Argentina

Administrative Informations


NCT ID

NCT02549170

Organization ID

161403

Secondary IDs

2014-005496-87

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Collaborators

 Takeda Development Center Americas, Inc.

Study Sponsor

Study Director, Study Director, Takeda


Verification Date

February 2022