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Immune Globulin Intravenous (IGIV) For Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Phase 3
Completed
Conditions
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Interventions
Drug: Albumin (Human) 25%, United States Pharmacopeia (USP)
Drug: Immune Globulin IV (Human), 10% Caprylate/Chromatography Purified
Registration Number
NCT00220740
Lead Sponsor
Grifols Therapeutics LLC
Brief Summary

The intent of this study is to demonstrate the efficacy and safety of Immune Globulin Intravenous (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in newly or previously diagnosed CIDP subjects. Eight courses of treatment with either placebo or IGIV-C will occur every 3 weeks. Neurological function will be measured by Inflammatory Neuropathy Cause and Treatment (INCAT) scores. Patients who deteriorate or show no improvement between day 16 and month 6 will receive the alternate study drug for an additional 6 months.

Detailed Description

110 subjects, 55 per treatment group, with newly or previously diagnosed CIDP defined by INCAT neurophysiological diagnostic criteria will be enrolled into the trial. Patients will not be replaced if they discontinue prematurely.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
117
Inclusion Criteria
  • Documented diagnosis of CIDP must be made by a neurologist specializing/experienced in neuromuscular diseases based on: a) Progressive or relapsing motor and sensory dysfunction of more than one limb resulting from neuropathy over the 2 months prior to the date informed consent is obtained, and b) Cerebrospinal fluid (CSF) less than 50 white cells/µl since CIDP diagnosis (CSF testing studies are NOT mandatory)
  • Fulfillment of INCAT neurophysiological criteria for focal demyelinating polyradiculoneuropathy
  • Overall INCAT score between 2-9 and significant disability in upper or lower limb function in at least 2 limbs. (An INCAT score of 2 must be exclusively from leg disability to qualify.)
Exclusion Criteria
  • Treatment with IGIV or plasma within 3 months prior to entry
  • Steroids (Prednisolone or equivalent) > 10 mg/day or equivalent (i.e., > 20 mg every 2 days) during the last 3 months prior to entry
  • Treatment with immunomodulatory/immunosuppressive agents (azathioprin, tacrolimus,cyclosporin, Muromonab-CD3 (OKT3), any interferon), previous lymphoid irradiation or prior treatment with cyclophosphamide, methotrexate, mitoxantrone or any other immunosuppressant drug within the past 6 months prior to entry
  • Concomitant use of supplements containing any amount of fish oil within 30 days prior to entry
  • Respiratory impairment requiring mechanical ventilation
  • Myelopathy or evidence of central demyelination or persisting neurological deficits from stroke, central nervous system (CNS) trauma or peripheral neuropathies of other cause which include diabetes mellitus (defined as a history of type 1 or type 2 diabetes with fasting plasma glucose ≥ 7.0 mmol/L), uremic, toxic and familial neuropathies
  • Pure motor syndrome fulfilling criteria for multifocal motor neuropathy with conduction block. Lower motor neuron disorder with motor weakness in an upper limb, without sensory deficit and with proximal conduction block (50% decrease in amplitude/area with proximal distal stimulation ) in motor nerves and normal sensory nerve conduction studies.
  • Clinical or known evidence of associated systemic diseases that might cause neuropathy, including but not limited to connective tissue disease, HIV infection, hepatitis, Lyme disease, cancer (with the exception of benign skin cancer), Castleman's disease and systemic lupus erythematosus, diabetes mellitus (defined as a history of type 1 or type 2 diabetes with fasting plasma glucose ≥ 7.0 mmol/L), a malignant plasma cell dysplasia, immunoglobulin M (IgM) paraproteinemia, and amiodarone therapy.
  • History of anaphylaxis or severe systemic response to immunoglobulin or with a blood product.
  • Cardiac insufficiency (NYHA III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease, or history of congestive heart failure, severe hypertension (diastolic pressure >120 mmHg or systolic >170 mmHg).
  • Females who are pregnant, breast feeding, or if of childbearing potential, unwilling to practice adequate contraception throughout the study.
  • Known hyperviscosity.
  • History of renal insufficiency or serum creatinine levels > 221 µmol/L (2.5 mg/dL).
  • Known selective immunoglobulin A (IgA) deficiency.
  • Other investigational drugs received within the 30 days prior to entry
  • Conditions whose symptoms and effects could alter protein catabolism and/or immunoglobulin G (IgG) utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
  • Known hypercoagulable state.
  • Mentally challenged adult subjects who cannot give independent informed consent.
  • Subjects with uncompensated hypothyroidism (abnormally high thyroid-stimulating hormone (TSH) and abnormally low T4) or vitamin B12 deficiency (abnormally low) within the last 3 months prior to entry.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Group 2Albumin (Human) 25%, United States Pharmacopeia (USP)-
Group 1Immune Globulin IV (Human), 10% Caprylate/Chromatography PurifiedIGIV-C
Primary Outcome Measures
NameTimeMethod
Comparison of the Responder Rates Between Two Treatment Groups in the Efficacy Period6 months

The primary efficacy objective was the comparison of IGIV-C and Placebo group Responder rates. An Efficacy Period Responder was defined as a subject with ≥ 1 point improvement in the adjusted Inflammatory Neuropathy Case And Treatment (INCAT) score, with the improvement maintained through the end of Week 24 in the Efficacy Period.

Measurements are reported in INCAT scale of 0-5 in both lower and upper extremities, for a total score of 0 to 10.

INCAT scores for arm disability: 0 = no upper limb problems; 5 = inability to use either arm for any purposeful movement.

INCAT scores for leg disability: 0= walking not affected; 5 = restricted to wheelchair, unable to stand and walk a few steps with help

Secondary Outcome Measures
NameTimeMethod
Mean Change in the Amplitude (Millivolts) in the Most Severely Affected Motor Nerve During the Efficacy Period6 months

Mean changes in amplitude \[mV\] measured at most proximal site in the most severely affected motor nerve from baseline to endpoint during the Efficacy Period (Intent to treat population)

Mean Change in Grip Strength During the Efficacy Period6 months
Time to Relapse for Subjects Who Were IGIV-C Responders or IGIV-C Rescue Successes, During the Randomized Withdrawal Period6 months

Trial Locations

Locations (32)

Saint Louis University Medical Center

🇺🇸

St. Louis, Missouri, United States

Fakultní nemocnice Ostrava

🇨🇿

Ostrava-Poruba, Czech Republic

Centre of Clinical Neurology, Neurology Department

🇵🇱

Cracow, Poland

Neurologická klinika Pardubice

🇨🇿

Pardubice, Czech Republic

Medical Acedemy, Clinical Hospital, Neurology Department

🇵🇱

Lubin, Poland

Fakultní nemocnice Motol

🇨🇿

Praha 5, Czech Republic

Columbia University

🇺🇸

New York, New York, United States

Yale University School of Medicine

🇺🇸

New Haven, Connecticut, United States

Wake Forest University-School of Medicine

🇺🇸

Winston-Salem, North Carolina, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

University of Texas-Southwestern Medical Center at Dallas

🇺🇸

Dallas, Texas, United States

Hospital Frances

🇦🇷

Buenos Aires, Argentina

Hospital Ramos Mejia

🇦🇷

Buenos Aires, Argentina

Fundacion para la Lucha contra Las Enfermedades Neurologicas de la Infacia (FLENI)

🇦🇷

Buenos Aires, Argentina

Instituto de Neurociencias Buenos Aires (INEBA)

🇦🇷

Capital Federal, Argentina

Vancouver Hospital and Health Sciences Center

🇨🇦

Vancouver, British Columbia, Canada

Fakultní nemocnice Brno

🇨🇿

Brno, Czech Republic

Jüdisches Krankenhaus

🇩🇪

Berlin, Germany

Tel Aviv Sourasky Medical Center

🇮🇱

Tel Aviv, Israel

Chaim Sheba Medical Center

🇮🇱

Tel Hashomer, Israel

Univesita delgi Studi di Genova, Dipartimento di Scienze, Neurologiche e della Visione

🇮🇹

Genova, Italy

Dipartimento di Neuroscienze, Sezione di Neurologia, AO Chieti

🇮🇹

Chieti, Italy

Assaf Harofe Medical Center

🇮🇱

Zrifin, Israel

Antiguo Hospital Civil de Guadalajara

🇲🇽

Guadalajara, Jalisco, Mexico

Hospital San Raffaele

🇮🇹

Milano, Italy

Hospital Angel Leano, Neurology Department

🇲🇽

Guadalajara, Jalisco, Mexico

County Specialist Hospital, Neurology Department

🇵🇱

Gdansk, Poland

Hospital Central San Luis Potosi, Neurology Department

🇲🇽

San Luis Potosi, Mexico

Barlicki Hospital

🇵🇱

Lodz, Poland

Central Clinical Hospital, Medical Academy Warsaw

🇵🇱

Warsaw, Poland

County Hospital

🇵🇱

Zgierz, Poland

University Hospital, University of Belgrade

🇷🇸

Belgrade, Serbia

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