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

NCT ID: NCT02549170

Last Updated: 2023-05-24

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

138 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-12-15

Study Completion Date

2022-02-23

Brief Summary

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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

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Conditions

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Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Epoch 1: HYQVIA/HyQvia

Participants received HYQVIA/HyQvia (rHuPH20) 80 U/g IG, following by SC injection of immunoglobulin (IGI) 10% at the same monthly equivalent IG dose as the individual participant's pre-randomized IG dose when administered every 2, 3, or 4 weeks for 30.28 weeks or until relapse.

Group Type EXPERIMENTAL

HYQVIA

Intervention Type BIOLOGICAL

Participants will receive HYQVIA/HyQvia SC which contains both Immune Globulin Infusion 10% (Human) (IGI, 10%) and recombinant human hyaluronidase (rHuPH20).

Epoch 1: Placebo with rHuPH20

Participants received rHuPH20 80 U/I0 mL placebo solution, followed by SC placebo infusion at matching infusion volume as the participant's pre-randomization monthly equivalent IG dose when administered every 2, 3, or 4 weeks for 31.54 weeks or until relapse.

Group Type PLACEBO_COMPARATOR

0.25% albumin placebo solution with rHuPH20

Intervention Type BIOLOGICAL

Participants will receive placebo solution (0.25% human albumin in Lactated Ringer's solution) and rHuPH20.

Epoch 2: E1: Placebo Relapse - E2: GGL/KIOVIG

Participants who were enrolled to receive placebo with rHuPH20 and achieved chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) relapse during Epoch 1 received the induction dose of GGL/KIOVIG 2 g/kg bi-weekly (BW), followed by IV infusion at the same monthly equivalent dose as the participant's pre-randomization IGIV dosing regimen when administered every 3 weeks for 25.63 weeks or until relapse.

Group Type EXPERIMENTAL

IGIV GAMMAGARD LIQUID/KIOVIG

Intervention Type BIOLOGICAL

Participants will receive GAMMAGARD LIQUID/KIOVIG

Epoch 2: E1: HYQVIA Relapse - E2: GGL/KIOVIG

Participants who were enrolled to receive HYQVIA/HyQvia (rHuPH20) and achieved CIDP relapse during Epoch 1 received the induction dose of GGL/KIOVIG 2 g/kg BW, followed by IV infusion at the same monthly equivalent dose as the participant's pre-randomization IGIV dosing regimen when administered every 3 weeks for 28.67 weeks or until relapse.

Group Type EXPERIMENTAL

IGIV GAMMAGARD LIQUID/KIOVIG

Intervention Type BIOLOGICAL

Participants will receive GAMMAGARD LIQUID/KIOVIG

Epoch 2: E1: Placebo Relapse - E2: GAMMUNEX-C

Participants who were enrolled to receive placebo with rHuPH20 and achieved CIDP relapse during Epoch 1 received the induction dose of GAMMUNEX-C 2 g/kg BW, followed by IV infusion at the same monthly equivalent dose as the participant's pre-randomization IGIV dosing regimen when administered every 3 weeks for 24.33 weeks or until relapse.

Group Type EXPERIMENTAL

IGIV GAMUNEX®-C

Intervention Type BIOLOGICAL

Participants will receive GAMUNEX®-C

Interventions

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HYQVIA

Participants will receive HYQVIA/HyQvia SC which contains both Immune Globulin Infusion 10% (Human) (IGI, 10%) and recombinant human hyaluronidase (rHuPH20).

Intervention Type BIOLOGICAL

0.25% albumin placebo solution with rHuPH20

Participants will receive placebo solution (0.25% human albumin in Lactated Ringer's solution) and rHuPH20.

Intervention Type BIOLOGICAL

IGIV GAMMAGARD LIQUID/KIOVIG

Participants will receive GAMMAGARD LIQUID/KIOVIG

Intervention Type BIOLOGICAL

IGIV GAMUNEX®-C

Participants will receive GAMUNEX®-C

Intervention Type BIOLOGICAL

Other Intervention Names

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10%) with recombinant human hyaluronidase (rHuPH20) Immune Globulin Infusion 10% (Human) (IGI IGI 10% with rHuPH20 Immune Globulin Infusion (Human) Intravenous immunoglobulin G 10% (GAMMAGARD LIQUID/KIOVIG) GAMMAGARD LIQUID Immune Globulin Infusion (Human) Intravenous immunoglobulin G Approved IGIV product for US sites

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Takeda Development Center Americas, Inc.

INDUSTRY

Sponsor Role collaborator

Baxalta now part of Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Shire

Locations

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Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

Arizona Neuromuscular Research Center

Phoenix, Arizona, United States

Site Status

HonorHealth Neurology

Scottsdale, Arizona, United States

Site Status

University of California-Irvine

Orange, California, United States

Site Status

Forbes Norris Mda/als Ctr

San Francisco, California, United States

Site Status

Regents of the University of colorado

Aurora, Colorado, United States

Site Status

Immunoe Research Centers

Centennial, Colorado, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, United States

Site Status

William Beaumont Hospital

Royal Oak, Michigan, United States

Site Status

Neurology Center of Las Vegas

Las Vegas, Nevada, United States

Site Status

Rutgers New Jersey Medical School

Newark, New Jersey, United States

Site Status

Hospital for Special Surgery

New York, New York, United States

Site Status

Wake Forest University

Winston-Salem, North Carolina, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Austin Neuromuscular Center

Austin, Texas, United States

Site Status

The Methodist Hospital Research Institute

Houston, Texas, United States

Site Status

University Texas Physicians CAR

Houston, Texas, United States

Site Status

Hospital Italiano

Ciudad Autonoma Buenos Aires, Buenos Aires, Argentina

Site Status

Hospital Britanico de Buenos Aires

Ciudad Autonoma Buenos Aires, , Argentina

Site Status

Complejo Medico de la Policia Federal Argentina Churruca Visca

Ciudad Autonoma Buenos Aires, , Argentina

Site Status

Instituto de Investigaciones Neurologicas Raul Carrea, FLENI

Ciudad Autonoma Buenos Aires, , Argentina

Site Status

Instituto de Neurologia de Curitiba - Hospital Ecoville

Curitiba, Paraná, Brazil

Site Status

HUAP - UFF - Hospital Universitario Antonio Pedro - Universidade Federal Fluminense

Niterói, Rio Do Janeiro, Brazil

Site Status

Hospital das Clínicas da Faculdade de Medicina da UNICAMP

Campinas, São Paulo, Brazil

Site Status

Hospital das Clínicas FMRP-USP

Ribeirão Preto, São Paulo, Brazil

Site Status

Hospital Sao Paulo

São Paulo, São Paulo, Brazil

Site Status

University of Alberta Hospital

Edmonton, Alberta, Canada

Site Status

LHSC - University Hospital

London, Ontario, Canada

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Institucion Prestadora de Servicios de Salud de la Universidad de Antioquia "IPS UNIVERSITARIA"

Medellín, , Colombia

Site Status

Clinical Hospital Centre Rijeka

Rijeka, , Croatia

Site Status

Clinical Hospital Centar Zagreb

Zagreb, , Croatia

Site Status

University Hospital Centre "Sestre Milosrdnice"

Zagreb, , Croatia

Site Status

Fakultni nemocnice Brno

Brno, , Czechia

Site Status

Fakultni nemocnice Ostrava

Ostrava - Poruba, , Czechia

Site Status

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status

Århus Universitetshospital

Aarhus, , Denmark

Site Status

CHU de Nice Hôpital Pasteur 2

Nice, Alpes Maritimes, France

Site Status

Hôpital de la Timone

Marseille, Bouches-du-Rhône, France

Site Status

Groupe Hospitalier Pellegrin - Hôpital Pellegrin

Bordeaux, Gironde, France

Site Status

Hopital Neurologique Pierre Wertheimer

Bron, Rhone, France

Site Status

Universitaetsmedizin Goettingen

Göttingen, Lower Saxony, Germany

Site Status

Universitaetsklinikum Essen

Essen, North Rhine-Westphalia, Germany

Site Status

Universitaetsklinikum Leipzig AoeR

Leipzig, Saxony, Germany

Site Status

University Hospital of Patra

Pátrai, , Greece

Site Status

Chaim Sheba Medical Center

Ramat Gan, , Israel

Site Status

IRCCS Ospedale Casa Sollievo della Sofferenza

San Giovanni Rotondo, Foggia, Italy

Site Status

Istituto Clinico Humanitas

Rozzano, Milano, Italy

Site Status

Azienda Ospedaliera Universitaria Policlinico Tor Vergata

Rome, Roma, Italy

Site Status

Azienda Ospedaliero Universitaria San Martino

Genova, , Italy

Site Status

Azienda Ospedaliera Universitaria Policlinico G. Martino

Messina, , Italy

Site Status

Casa di Cura del Policlinico

Milan, , Italy

Site Status

Fondazione Istituto Neurologico Casimiro Mondino

Pavia, , Italy

Site Status

Azienda Ospedaliero Universitaria Pisana

Pisa, , Italy

Site Status

Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza

Roma, , Italy

Site Status

Azienda Ospedaliera Città della Salute e della Scienza di Torino

Torino, , Italy

Site Status

Azienda Ospedaliero-Universitaria Santa Maria della Misericordia

Udine, , Italy

Site Status

Instituto Nacional de Ciencias Médicas y Nutricion Dr. Salvador Zubiran

Mexico City, Mexico City, Mexico

Site Status

Oslo Universitetssykehus HF, Ullevål

Oslo, , Norway

Site Status

COPERNICUS Podmiot Leczniczy Sp. z o. o.,

Gdansk, , Poland

Site Status

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Site Status

Uniwersytecki Szpital Kliniczny nr 1 im. Norberta Barlickiego

Lodz, , Poland

Site Status

Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie

Lublin, , Poland

Site Status

Clinical Center of Serbia

Belgrade, , Serbia

Site Status

Military Medical Academy

Belgrade, , Serbia

Site Status

Clinical Center Nis

Niš, , Serbia

Site Status

Univerzitna nemocnica Bratislava Nemocnica ak. L. Derera, II. Neurologicka klinika

Bratislava, , Slovakia

Site Status

Fakultna nemocnica Nitra

Nitra, , Slovakia

Site Status

Hospital Universitari de Bellvitge

L'Hospitalet de Llobregat, Barcelona, Spain

Site Status

Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

Site Status

Hospital Universitari Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Hallands sjukhus

Halmstad, , Sweden

Site Status

Pamukkale Uni. Med. Fac.

Denizli, , Turkey (Türkiye)

Site Status

Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty

Istanbul, , Turkey (Türkiye)

Site Status

Dokuz Eylul University Faculty of Medicine

Izmir, , Turkey (Türkiye)

Site Status

Selcuk Universitesi Selcuklu Tip Fakultesi Hastanesi

Konya, , Turkey (Türkiye)

Site Status

Celal Bayar University Medical Faculty

Manisa, , Turkey (Türkiye)

Site Status

Southmead Hospital

Bristol, Avon, United Kingdom

Site Status

King's College Hospital

London, Greater London, United Kingdom

Site Status

The Walton Centre

Liverpool, Merseyside, United Kingdom

Site Status

Countries

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United States Argentina Brazil Canada Colombia Croatia Czechia Denmark France Germany Greece Israel Italy Mexico Norway Poland Serbia Slovakia Spain Sweden Turkey (Türkiye) United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://clinicaltrials.takeda.com/study-detail/5f6b5fc54db2bf003ab45bd5

To obtain more information on the study, click here/on this link

Other Identifiers

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2014-005496-87

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

161403

Identifier Type: -

Identifier Source: org_study_id

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