Chronic inflammatory demyelinating polyneuropathy (CIDP) and treatment with subcutaneous immunoglobulin (IgPro20)

• Register: EUCTR
• Last refreshed on: 17 October 2016
• Main ID: EUCTR2011-003448-28-DE
• Date of registration: 31/01/2012
• Prospective Registration: Yes
• Primary sponsor: CSL Behring GmbH
• Public title: Chronic inflammatory demyelinating polyneuropathy (CIDP) and treatment with subcutaneous immunoglobulin (IgPro20)
• Scientific title: Randomized, multicenter, double-blind, placebo-controlled, parallel-group phase III study to investigate the efficacy, safety, and tolerability of 2 different doses of IgPro20 (subcutaneous immunoglobulin) for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) – the PATH study - PATH
• Date of first enrolment: 20/04/2012
• Target sample size: 350
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-003448-28
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Austria, Belgium, Canada, Czech Republic, Estonia, Finland, France, Germany, Israel, Italy, Japan, Lithuania, Netherlands, Poland, Spain, United Kingdom, United States

Contacts

• Name: Trial Disclosure Manager
• Affiliation: CSL Behring GmbH

• Name: Trial Disclosure Manager
• Affiliation: CSL Behring GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
1. Definite or probable CIDP according to the EFNS/PNS criteria 2010.
2. Repeated treatment with IVIG (= 4 doses) within the last 9 months prior to enrollment.
3. An IVIG treatment during the last 8 weeks prior to enrollment.
4. Age =18 years.
5. Male or female.
6. Written informed consent for study participation obtained before undergoing any studyspecific
procedures.
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 310
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

Exclusion criteria:
1. Any polyneuropathy of other causes
2. Any other disease (mainly neurological or chronic orthopedic) that has caused neurological symptoms or may interfere with treatment or outcome assessments
3. Severe diseases and conditions that are likely to interfere with evaluation of the study product or satisfactory conduct of the study
4. History of thrombotic episodes within the 2 years prior to enrolment
5. Known allergic or other severe reactions to blood products including intolerability to previous IVIG


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
MedDRA version: 19.0 Level: PT Classification code 10061811 Term: Demyelinating polyneuropathy System Organ Class: 10029205 - Nervous system disorders

Intervention(s)

Trade Name: Hizentra®
Product Name: Hizentra®
Product Code: IgPro20
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Human normal immunoglobulin (SCIg)
CAS Number: 0
Current Sponsor code: IgPro10-SOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Main Objective: To determine the efficacy of 2 different doses of IgPro20 (0.2 g/kg bw and/or 0.4 g/kg bw) in the maintenance treatment of CIDP in comparison to placebo.

Timepoint(s) of evaluation of this end point: During SC treatment period - 24 weeks

Secondary Objective: - To investigate the efficacy of IgPro20 with additional clinical outcome measures in comparison to placebo.
- To investigate the safety and tolerability of IgPro20 in comparison to placebo.
- To investigate health-related quality of life (HRQL) following treatment with IgPro20.
- To investigate the safety and efficacy of IgPro10 re-stabilization therapy.
- To investigate the safety and efficacy of IgPro10 rescue therapy.

Primary end point(s): Efficacy:
Percentage (%) of subjects who have a CIDP relapse during SC treatment or are withdrawn from the study during SC treatment for any reason.

Secondary Outcome(s)

Secondary end point(s): Efficacy:
• Changes in means at SC Treatment Period completion visit compared to baseline between groups in:
- INCAT score.
- Mean grip strength (dominant/non-dominant hand).
- MRC sum score.
- R-ODS.
• Difference in “time to CIDP relapse or withdrawal due to any other reason” using a Kaplan-Meier estimation comparing both IgPro20 groups with placebo as well as the 2 IgPro20 groups pairwise.
• Time to improvement on IgPro10 re-stabilization therapy (INCAT, R-ODS, Grip strength).
• Changes in means before and at the end of IgPro10 re-stabilization or rescue therapy in
- Mean grip strength (dominant/non-dominant hand).
- MRC sum score.
- R-ODS.
- INCAT disability score.
• Time to improvement after CIDP relapse in the SC Treatment Period and IgPro10 rescue therapy, defined as a decrease in INCAT score back to or below baseline.
Safety:
• For IgPro20: Rate of AEs per infusion, number and % of subjects with AEs during the SC Treatment Period
• For IgPro10: rate of AEs per infusion, number and % of subjects with AEs during IVIG Restabilization Period and during IVIG rescue therapy.

Timepoint(s) of evaluation of this end point: During study - up to 52 weeks

Secondary ID(s)

IgPro20_3003

Source(s) of Monetary Support

CSL Behring GmbH