Study of the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma 5% DIF in Patients With Post-polio Syndrome

  • STATUS
    Recruiting
  • End date
    Dec 29, 2022
  • participants needed
    210
  • sponsor
    Instituto Grifols, S.A.
Updated on 29 January 2021

Summary

This is a multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial with adaptive dose selection in subjects with post polio syndrome (PPS).

The main purpose of this study is to select a dose of Flebogamma 5% DIF and confirm the efficacy of the selected Flebogamma 5% DIF dose by assessing physical performance, as measured by 2 Minutes Walk Distance (2MWD) test.

The study will consist of 2 stages, with each stage consisting of a screening period (up to 4 weeks), a treatment period (52 weeks), and a follow-up period (24 weeks).

Description

This is a phase II/III multicenter, prospective, randomized, placebo-controlled, double-blind, parallel-group clinical trial with an adaptive design (flexible group sequential design with adaptive dose selection) in subjects with PPS.

This study will consist of two stages. The first stage (Stage 1) is for dose selection, and the second stage (Stage 2) is to establish the superiority (efficacy confirmation) of Flebogamma 5% DIF and for overall safety analysis. Stage 1 is a 3-arm evaluation of 2 dose levels of Flebogamma 5% DIF. Flebogamma 5% DIF 2 g/kg of body weight (IVIG 2 g/kg arm), Flebogamma 5% DIF 1 g/kg of body weight plus the equivalent volume of Normal Saline Solution (20 mL/kg of body weight) (IVIG 1 g/kg arm), or a total dose of 40 mL/kg of body weight Normal Saline Solution (equivalent volume of the 2 g/kg of body weight Flebogamma 5% DIF infusions) (placebo arm) will be administered over 2 consecutive days every 4 weeks during a 52-week treatment period. At the end of Stage 1, an interim analysis will be conducted and 1 of the 2 Flebogamma 5% DIF doses will be selected based on predefined criteria to be used for Stage 2.

Stage 2 will consist of 2 treatment arms, the selected dose of Flebogamma 5% DIF from Stage 1 and Normal Saline Solution (40 mL/kg of body weight). Study drug will be administered over 2 consecutive days every 4 weeks during a 52-week treatment period. During Stage 2, the selected dose of Flebogamma 5% DIF and Normal Saline Solution will be administered in the same manner as in Stage 1, including administering the total dose for both treatment arms at a volume equivalent to that for the IVIG 2 g/kg arm, regardless of the selected dose.

Primary efficacy endpoint will be:

  • Physical performance (2MWD) from baseline to the end of the treatment period (at End of Treatment Visit -Week 52).

Details
Condition Postpolio syndrome, post-polio syndrome, post polio syndrome
Treatment Placebo, Flebogamma 5% DIF
Clinical Study IdentifierNCT02176863
SponsorInstituto Grifols, S.A.
Last Modified on29 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Subjects with Body Mass Index less than 35 kg/m^2
Subjects meet March-of-Dimes clinical criteria for diagnosis of PPS
Subjects who are ambulatory or are able to walk with a cane or other aids or use a wheelchair (but they are not wheelchair-bound)
Subjects who have at least 2 newly weakened muscle groups due to PPS (as defined by medical history), with at least 1 of them in a lower extremity, and having an Medical Research Council (MRC) scale score greater than 3 at the MMT performed by the independent assessor at the Screening Visit (SV)
Female of child-bearing potential must have a negative test for pregnancy
Female of child-bearing potential and their sexual partners have agreed to practice contraception using a method of proven reliability
Subjects who are able to walk a 2MWD of at least 50 meters at the SV and Enrollment Visit/Infusion Visit 1 (EV/IV1)
Subjects who are able to walk a consistent baseline 2 MWD, that is, the difference in 2MWD between the SV and EV/IV1 is not more than 10%

Exclusion Criteria

Subjects have received human normal immune globulin treatment given by intravenous, subcutaneous, or intramuscular route within the last 3 years
Subjects who are not ambulatory (wheelchair-bound individuals)
Subjects with poor venous access
Subjects with intractable pain requiring narcotics or other psychotropic drugs
Subjects with a history of anaphylactic reactions or severe reactions to any blood-derived product
Subjects with a history of intolerance to any component of the investigational products, such as sorbitol
Subjects receiving corticosteroids, except for those who are taking inhaled corticosteroids for asthma
Subjects with a documented diagnosis of hyperviscosity or hypercoagulable state or thrombotic complications to polyclonal intravenous immunoglobulin (IVIG) therapy in the past
Subjects with a history of recent (within the last year) myocardial infarction, stroke, or uncontrolled hypertension
Subjects who suffer from congestive heart failure, embolism, or electrocardiogram changes indicative of unstable angina or atrial fibrillation
Subjects with a history of chronic alcoholism or illicit drug abuse (addiction) in the preceding 12 months prior to the SV
Subjects with active psychiatric illness that interferes with compliance or communication with health care personnel
Subjects with depression with scores >30 as assessed by the Center for Epidemiologic Studies Depression validated scale
Females who are pregnant or are nursing an infant child
Subjects with any medical condition which makes clinical trial participation unadvisable or which is likely to interfere with the evaluation of the study treatment and/or the satisfactory conduct of the clinical trial according to the Investigator's judgment
Subjects currently receiving, or have received within 3 months prior to the SV, any investigational medicinal product or device
Subjects who are unlikely to adhere to the protocol requirements, or are likely to be uncooperative, or unable to provide a storage serum/plasma sample prior to the first investigational drug infusion
Subjects with known selective IgA deficiency and serum antibodies anti-IgA
Subjects with renal impairment (i.e., serum creatinine exceeds more than 1.5 time the upper limit of normal [ULN])
Subjects with aspartate aminotransferase or alanine aminotransferase levels exceeding more than 2.5 times the ULN
Subjects with hemoglobin levels <10 g/dL, platelets levels <100,000/mm^3, white blood cells count <3.0 k/L, and erythrocyte sedimentation rate >50 mm/h or twice above normal
Subjects with known seropositive to Hepatitis C virus, Human immunodeficiency virus-1 and/or Human immunodeficiency virus-2
Subjects with a history of intolerance to fructose
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