Study to evaluate the safety and efficacy of Flebogamma 5% DIF in patients with Post-Polio Syndorme

Phase 1

• Conditions: Patients with Post-polio syndrome; MedDRA version: 18.1Level: SOCClassification code 10029205Term: Nervous system disordersSystem Organ Class: 10029205 - Nervous system disorders; MedDRA version: 18.1Level: PTClassification code 10036239Term: Post polio syndromeSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2013-004503-39-RO
• Lead Sponsor: Instituto Grifols, S.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-11-17
• Last Update Posted: 8 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 210

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 body mass index (BMI) less than 35 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 MRC scale score greater than
three 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 entire 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

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 inhaled corticosteroids 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 <10 g/dL, platelets levels
<100,000/mm3, white blood cells count <3.0 k/µL and ESR
>50 mm/h or twice above normal.
22. Subjects with known seropositive to HCV, HIV-1 and/or
HIV-2.
23. Subjects with a history of intolerance to fructose.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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:<br>- assessing pain , as measured by VAS of pain, compared to that of placebo<br>- evaluating health-related quality of life (HRQoL), as measured by SF-36 PCS, compared to that of placebo<br>- endurance, as measured by 6MWD, compared to that of placebo.;Primary end point(s): To test whether monthly infusions (every four weeks) of<br>intravenous Flebogamma® 5% DIF in a 1 year treatment period<br>in PPS subjects are superior to placebo by assessing physical<br>performance, as measured by 2MWD (2-minutes walk distance)<br>For Stage 1, to select the optimal dose of IVIG as compared to<br>the placebo.<br>For Stage 2, to establish superiority of the selected dose of IVIG<br>as compared to placebo by combining both Stage 1 and Stage 2<br>data.;Timepoint(s) of evaluation of this end point: 1 year treatment

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Efficacy endpoints:<br>- Pain (Visual Analogue Scale [VAS] of pain) from baseline<br>to the end of the treatment period.<br>- HRQoL (Medical Outcomes Study 36-Item Short Form<br>Health Survey [SF-36] Physical Component Summary<br>[PCS]) from baseline to the end of the treatment period.<br>- Endurance (Six Minutes Walk Distance [6MWD]) from<br>baseline to the end of the treatment period.<br><br>Safety endpoints will include Adverse Events (AEs), vital signs during infusions,<br>physical assessments and blood tests for clinical safety.;Timepoint(s) of evaluation of this end point: One year treatment.<br>And for the one year treatments and a half year without treatment (to assess the sustainability effect).