Study of the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma® 5% Dual Inactivation and Filtration (DIF) in Participants With Post-polio Syndrome

NCT02176863 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: IG11042013-004503-39
• Study Type: interventional
• Target: 191
• Locations: 9 countries
• Sites: 21

Brief Summary

This was a multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial with adaptive dose selection in participants with post-polio syndrome (PPS). The main purpose of this study was 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 Two-Minute Walk Distance (2MWD) test. The study consisted 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).

Conditions

Post-polio Syndrome

Keywords

FORCE; Post-polio syndrome; Flebogamma®; Immune Globulin Intravenous; IVIG

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Physical Performance Assessed by Two-Minute Walk Distance (2MWD) Test

  The 2MWD was used to assess physical performance by measuring the distance that a participant can quickly walk at a self-preferred speed on an indoor flat, hard surface 30 m (100-ft) hallway in a period of 2 minutes. A positive change from baseline indicates improvement (i.e. a patient can walk farther). Increase in distance walked (in meters) indicates improvement.

  Baseline to Week 52

Secondary Outcomes (3)

• Change From Baseline in Pain Using Visual Analogue Scale (VAS) of Pain

  Baseline to Week 52

• Change From Baseline in Health-Related Quality of Life (HRQoL) Assessed by Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36) Physical Component Summary (PCS)

  Baseline to Week 52

• Change From Baseline in Endurance Assessed by Six-Minute Walk Distance (6MWD) Test

  Baseline to Week 52

Study Arms (5)

Stage 1 Arm 1: 2 g/kg Flebogamma® 5% DIF

EXPERIMENTAL

Participants received Flebogamma® 5% DIF 2 g/kg of body weight administered via intravenous (IV) infusion over 2 consecutive days (Flebogamma® 5% DIF 1 g/kg infused on Day 1, followed by Flebogamma® 5% DIF 1 g/kg infused on Day 2) every 4 weeks for 52 weeks.

Biological: Flebogamma® 5% DIF

Stage 1 Arm 2: 1 g/kg Flebogamma® 5% DIF

EXPERIMENTAL

Participants received Flebogamma® 5% DIF 1 g/kg of body weight administered via IV infusion on Day 1, followed by 20 mL/kg of matching placebo administered on a separate day, for a total dosing period of 2 consecutive days, every 4 weeks for 52 weeks. The order of 1 g/kg Flebogamma® 5% DIF or matching placebo was randomly determined for each participant and was the same for the participant for all infusion visits during the treatment period.

Biological: Flebogamma® 5% DIF; Drug: Placebo

Stage 1 Arm 3: Placebo

PLACEBO COMPARATOR

Participants received matching placebo at a total dose of 40 mL/kg of body weight administered via IV infusion over 2 consecutive days. On Day 1, a dose of 20 mL/kg of matching placebo was given, followed by the second dose of 20 mL/kg of matching placebo on Day 2, administered every 4 weeks for 52 weeks.

Drug: Placebo

Stage 2 Arm 1: 1 g/kg Flebogamma® 5% DIF

EXPERIMENTAL

Participants received Flebogamma® 5% DIF 1 g/kg of body weight administered via IV infusion on Day 1, followed by 20 mL/kg of matching placebo administered on a separate day, for a total dosing period of 2 consecutive days, every 4 weeks for 52 weeks. The order of 1 g/kg of Flebogamma® 5% DIF or matching placebo was randomly determined for each participant and was the same for the participant for all infusion visits during the treatment period.

Biological: Flebogamma® 5% DIF; Drug: Placebo

Stage 2 Arm 2: Placebo

PLACEBO COMPARATOR

Participants received matching placebo at a total dose of 40 mL/kg of body weight administered via IV infusion over 2 consecutive days. On Day 1, a dose of 20 mL/kg of matching placebo was given, followed by the second dose of 20 mL/kg of matching placebo on Day 2, administered every 4 weeks for 52 weeks.

Drug: Placebo

Interventions

Flebogamma® 5% DIF BIOLOGICAL

Human plasma-derived immunoglobulin

Also known as: immune globulin intravenous (human)

Stage 1 Arm 1: 2 g/kg Flebogamma® 5% DIF; Stage 1 Arm 2: 1 g/kg Flebogamma® 5% DIF; Stage 2 Arm 1: 1 g/kg Flebogamma® 5% DIF

Placebo DRUG

Matching placebo

Stage 1 Arm 2: 1 g/kg Flebogamma® 5% DIF; Stage 1 Arm 3: Placebo; Stage 2 Arm 1: 1 g/kg Flebogamma® 5% DIF; Stage 2 Arm 2: Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants with Body Mass Index less than 35 kg/m\^2.
• Participants who meet the clinical criteria for diagnosis of PPS as set by March-of-Dimes.
• Participants who are ambulatory or are able to walk with a cane or other aids or use a wheelchair (but they are not wheelchair-bound).
• Participants 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 a Medical Research Council (MRC) scale score greater than 3 at the Manual Muscle Testing (MMT) performed by the independent assessor at the Screening Visit (SV).
• Female of child-bearing potential must have a negative test for pregnancy (Human chorionic gonadotropin (HCG)-based assay).
• Female of child-bearing potential and their sexual partners have agreed to practice contraception using a method of proven reliability (i.e., hormonal methods; barrier methods; intrauterine devices methods) to prevent a pregnancy during the course of the clinical trial.
• Participants must be willing to comply with all aspects of the clinical trial protocol, including blood sampling and long-term storage of extra samples for the entire duration of the study.
• Participants who are able to walk a 2MWD of at least 50 meters at the SV and Enrollment Visit/Infusion Visit 1 (EV/IV1)
• Participants 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%.

You may not qualify if:

• Participants who have received human normal immune globulin treatment given by intravenous, subcutaneous, or intramuscular route within the last 3 years.
• Participants who are not ambulatory (wheelchair-bound individuals).
• Participants with poor venous access.
• Participants with intractable pain requiring narcotics or other psychotropic drugs.
• Participants with a history of anaphylactic reactions or severe reactions to any blood-derived product.
• Participants with a history of intolerance to any component of the investigational products, such as sorbitol.
• Participants receiving corticosteroids, except for those who are taking inhaled corticosteroids for asthma.
• Participants with a documented diagnosis of hyper viscosity or hypercoagulable state or thrombotic complications to polyclonal intravenous immunoglobulin (IVIG) therapy in the past.
• Participants with a history of recent (within the last year) myocardial infarction, stroke, or uncontrolled hypertension.
• Participants who suffer from congestive heart failure, embolism, or electrocardiogram changes indicative of unstable angina or atrial fibrillation.
• Participants with a history of chronic alcoholism or illicit drug abuse (addiction) in the preceding 12 months prior to the SV.
• Participants with active psychiatric illness that interferes with compliance or communication with health care personnel.
• Participants with depression with scores \>30 as assessed by the Center for Epidemiologic Studies Depression (CESD) validated scale.
• Females who are pregnant or are nursing an infant child.
• Participants 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.

Sponsors & Collaborators

• Instituto Grifols, S.A.: lead

Study Sites (21)

Washington University

St Louis, Missouri, 63110, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226-3596, United States

Location

Montreal Neurological Institute Clinical Research Unit, McGill University

Montreal, Quebec, H3A 2B4, Canada

Location

Thomayerova nemocnice, Klinicko-farmakologická jednotka

Prague, 4, Czechia

Location

Aarhus Universitets Hospital-Neurologisk Forskning

Aarhus N, 8200, Denmark

Location

Rigshospitalet

København Ø, 2100, Denmark

Location

Charité Campus Mitte

Berlin, 10117, Germany

Location

Hannover Medical School

Hanover, 30625, Germany

Location

Universitätsklinikum Jena

Jena, 07747, Germany

Location

Klinik für konservative Orthopädie und des Poliozentrums

Koblenz, 56073, Germany

Location

Westfälische Wilhelms-Universität Münster

Münster, 48149, Germany

Location

Azienda Ospedaliera Universitaria Integrata Verona (Ospedale Borgo Roma)

Verona, 37124, Italy

Location

Academisch Medisch Centrum Amsterdam UMC, Locatie AMC

Amsterdam, 1105 AZ, Netherlands

Location

MedTrials

Krakow, 31-436, Poland

Location

Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie

Lublin, 20-954, Poland

Location

Clinical Research Center Sp. z o.o.

Poznan, 60-848, Poland

Location

Samodzielny Publiczny Centralny Szpital Kliniczny

Warsaw, 02-097, Poland

Location

Institut Guttman

Badalona, 08916, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

MeSH Terms

Conditions

Postpoliomyelitis Syndrome

Interventions

gamma-Globulins; Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Poliomyelitis; Myelitis; Central Nervous System Infections; Infections; Enterovirus Infections; Picornaviridae Infections; RNA Virus Infections; Virus Diseases; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Neurodegenerative Diseases; Neuroinflammatory Diseases; Neuromuscular Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Immunoglobulin G; Immunoglobulin Isotypes; Antibodies

Results Point of Contact

• Title: Sandra Camprubi, PhD/Senior Director, Clinical Operations
• Organization: Instituto Grifols, S.A.

sandra.camprubi@grifols.com

+34 67 0923160

Study Officials

• Marinos Dalakas, MD

  Coordinating Investigator

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2014
• First Posted: June 27, 2014
• Study Start: September 23, 2014
• Primary Completion: November 24, 2022
• Study Completion: November 24, 2022
• Last Updated: December 5, 2025
• Results First Posted: December 5, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (1); US (4); DE (5); PL (4); CA (1); NL (1); ES (2); IT (1); DK (2)