Patients with Post-polio syndrome MedDRA version: 17.0 Level: PT Classification code 10036239 Term: Post polio syndrome System Organ Class: 10029205 - Nervous system disorders MedDRA version: 17.0 Level: SOC Classification code 10029205 Term: Nervous system disorders System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: Flebogamma DIF 50 mg/ml - 200 ml
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin (IVIg)
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Sponsors
Instituto Grifols, S.A.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Clinical trial subject inclusion criteria: 1. Male or female aged 18 to 75 years. 2. Subjects who understand and voluntarily signed and dated the Clinical Trial Written Informed Consent Form for his/her clinical trial participation. 3. Subjects with a BMI less than 30 kg/m2. 4. Subjects who meet the clinical criteria for diagnosis of PPS as set by the March-of-Dimes. 5. Subjects who are ambulatory or are able to walk with a cane or other aids. 6. Subjects who have at least two newly weakened muscle groups, and one of them in a lower extremity, as defined by medical history and having a mMRC scale score of 3 or greater at the SV. 7. Female subjects of child-bearing potential must have a negative test for pregnancy (human chorionic gonadotropin [HCG]-based assay). 8. Female subjects 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. 9. Subjects 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 whole duration of the study. 10. Subjects who are able to walk a 2MWD of at least 50 meters at the Screening Visit (SV) and EV/IV1. 11. Subjects who are able to walk a consistent baseline 2MWD, that is, the difference in 2MWD between the SV and EV/IV1 is not more than 10%. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 70 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 140
Exclusion criteria
Exclusion criteria: Clinical trial subject exclusion criteria: 1. Subjects who have received immune globulin treatment given by intravenous, subcutaneous or intramuscular route within the last 3 years. 2. Subjects who are not ambulatory (wheelchair-bound individuals). 3. Subjects with poor venous access. 4. Subjects with intractable pain requiring narcotics or other psychotropic drugs. 5. Subjects with a history of anaphylactic reactions or severe reactions to any blood-derived product. 6. Subjects with a history of intolerance to any component of the investigational products, such as sorbitol. 7. Subjects who are receiving corticosteroids, except for those who are taking them for asthma. 8. Subjects with a documented diagnosis of hyperviscosity or hypercoagulable state or thrombotic complications to polyclonal IVIG therapy in the past. 9. Subjects with a history of recent (within the last year) myocardial infarction, stroke, or uncontrolled hypertension. 10. Subjects who suffer from congestive heart failure, embolism, or ECG changes indicative of unstable angina or atrial fibrillation. 11. Subjects with a history of chronic alcoholism or illicit drug abuse (addiction) in the preceding 12 months prior to the SV. 12. Subjects with active psychiatric illness that interferes with compliance or communication with health care personnel. 13. Subjects with depression with scores >30 as assessed by the CESD validated scale. 14. Females who are pregnant or are nursing an infant child. 15. 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. 16. Subjects currently receiving, or have received within 3 months prior to the Screening Visit, any investigational medicinal product or device. 17. Subjects who are unlikely to adhere 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. 18. Subjects with a known selective IgA deficiency and serum antibodies anti-IgA. 19. Subjects with renal impairment (i.e., serum creatinine exceeds more than 1.5 time the upper limit of normal [ULN] for the expected normal range for the testing laboratory). 20. Subjects with AST or ALT levels exceeding more than 2.5 times the ULN for the expected normal range for the testing laboratory. 21. Subjects with hemoglobin levels 50 mm/h or twice above normal. 22. Subjects with known seropositive to HCV, HIV-1 and/or HIV-2.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and efficacy of Flebogamma 5% DIF in patients with post-polio syndrome;Secondary Objective: To evaluate clinical effect of Flebogamma 5%DIF in PPS subjects by: - assessing pain , as measured by VAS of pain, compared to that of placebo - evaluating health-related quality of life (HRQoL), as measured by SF-36 PCS, compared to that of placebo - endurance, as measured by 6MWD, compared to that of placebo.;Primary end point(s): To test whether monthly infusions (every four weeks) of intravenous Flebogamma® 5% DIF in a 1 year treatment period in PPS subjects are superior to placebo by assessing physical performance, as measured by 2MWD (2-minutes walk distance) For Stage 1, to select the optimal dose of IVIG as compared to the placebo. For Stage 2, to establish superiority of the selected dose of IVIG as compared to placebo by combining both Stage 1 and Stage 2 data.;Timepoint(s) of evaluation of this end point: 1 year treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy endpoints: - Pain (Visual Analogue Scale [VAS] of pain) from baseline to the end of the treatment period. - HRQoL (Medical Outcomes Study 36-Item Short Form Health Survey [SF-36] Physical Component Summary [PCS]) from baseline to the end of the treatment period. - Endurance (Six Minutes Walk Distance [6MWD]) from baseline to the end of the treatment period. Safety endpoints will include Adverse Events (AEs), vital signs during infusions, physical assessments and blood tests for clinical safety.;Timepoint(s) of evaluation of this end point: One year treatment. And for the one year treatments and a half year without treatment (to assess the sustainability effect). | — |
Countries
Canada, Denmark, Germany, Italy, Netherlands, Poland, Romania, Spain, Sweden, United States
Contacts
Public ContactDepartment of Clinical Trials
Instituto Grifols, S.A.
Outcome results
None listed