E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic inflammatory demyelinating polyradiculoneuropathy |
|
E.1.1.1 | Medical condition in easily understood language |
Chronic Inflammatory Demyelinating Polyneuropathy is a rare disorder
of the peripheral nerves (nerves outside the central nervous system)
caused by damage to the covering of the nerves called myelin |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10057645 |
E.1.2 | Term | Chronic inflammatory demyelinating polyradiculoneuropathy |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the long-term safety, tolerability, and immunogenicity of HYQVIA/HyQvia. |
|
E.2.2 | Secondary objectives of the trial |
To assess the long-term effect of HYQVIA/HyQvia on clinical outcome measures, including prevention of relapse, change in functional ability, hand grip strength, and muscle strength.
To assess the long-term effect of HYQVIA/HyQvia on quality of life, health utility, health resource utilization (HRU), treatment satisfaction, treatment preference, and subject global impression of change.
To explore further the pharmacokinetics of HYQVIA/HyQvia in CIDP subjects. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Has completed Epoch 1 of Study 161403 or Study 161601 without CIDP worsening.
If female of childbearing potential, the subject must have a negative pregnancy test at baseline and agree to employ adequate birth control measures (e.g. birth control pills/patches,intrauterine device (IUD), or diaphragm or condom [for male partner] with spermicidal jelly or foam) throughout the course of the study. |
|
E.4 | Principal exclusion criteria |
Subject has a serious medical condition such that in the opinion of the investigator the subject’s safety or medical care would be impacted by participation in this Extension Study.
New medical condition that developed during participation in Study 161403 or Study 161601 that in the judgment of the investigator could increase risk to the subject or interfere with the evaluation of investigational medicinal product and/or conduct of the study.
Subject is scheduled to participate in another, non-Baxalta clinical study involving an IP or investigational device during the course of this study.
The subject is nursing or intends to begin nursing during the course of the study.
Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment, or is scheduled to participate in another clinical study (with the exception of Study 161403 or 161601) involving an IP or investigational device during the course of this study.
The subject is a family member or employee of the investigator. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety/Tolerability
Number (percentage) of subjects experiencing any treatment-emergent serious and/or non-serious adverse events (SAEs and/or AEs, respectively), regardless of causality
Number (percentage) of subjects experiencing causally related SAEs and/or AEs
Number (percentage) of subjects with serious and/or non-serious adverse reactions (ARs) plus suspected ARs
Rate of AEs that may be a result of immune-mediated response to either immunoglobulin,rHuPH20, or other factors as listed in Table 12-1, expressed as number of events per infusion and per subject-year
Number (percentage) of infusions associated with treatment-emergent SAEs and/or AEs, regardless of causality
Number (percentage) of infusions associated with causally related SAEs and/or AEs
Number (percentage) of infusions temporally associated with AEs (defined as AEs
occurring during or within 72 hours after completion of an infusion)
Number (percentage) of infusions associated with serious and/or non-serious ARs plus suspected ARs
Number (percentage) of infusions associated with 1 or more systemic AEs
Number (percentage) of infusions associated with 1 or more local infusion site reactions
Number and proportion of infusions for which the infusion rate was reduced and/or the infusion was interrupted or stopped due to intolerability and/or AEs
Rates of systemic and local AEs, regardless of causality, expressed as number of events per infusion, per subject, and per subject-year
Rates of causally related systemic and local AEs, expressed as number of events per infusion, per subject, and per subject-year
Rates of systemic and local ARs plus suspected ARs, expressed as number of events per infusion, per subject, and per subject-year
Number of subjects with AE(s) that led to discontinuation from study
Number and rate per infusion of moderate or severe AEs that may be a result of immune mediated response to either immunoglobulin, rHuPH20 or other factors
Immunogenicity
Incidence of binding antibodies to rHuPH20
Incidence of neutralizing antibodies to rHuPH20
Number of subjects with a decline of anti-rHuPH20 antibody titers to the antibody titer level at baseline in study 161403 or 161601 and/or to <160 at the study completion or early discontinuation
For subjects who have >10,000 titer of binding antibodies to rHuPH20:
neutralizing antibodies and cross reactivity with Hyal-1,2 and 4 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Outcome measures
Pharmacokinetics—Serum IgG levels
Efficacy measures
Proportion subjects who relapse
EuroQoL
HRU
Treatment satisfaction
Treatment preference
Administration outcomes
Number of sites per infusion
Maximum volume per infusion site
Time to administer the study product (immunoglobulin/rHuPH20)
Monthly infusion time
Number of subjects/caregivers unable to continue with self/home infusion and reason(s) for this failure.
Maximum infusion rates
Optional photo recording of infusion procedures
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Pharmacokinetics
Timepoints: Week 1, 2, 13, 26, 39, 52, 65, 78, 91, 104
Efficacy measures
Timepoints: Week 13, 26, 39, 52, 65, 78, 91, 104
EuroQoL
Timepoints: Baseline, week 26, 52, 78
HRU
Timepoints: Baseline, Week 13, 26, 39, 52, 65,78, 91, 104
Treatment satisfaction
Timepoints: Baseline, week 26, 52, 78
Treatment preference
Timepoints: Baseline, week 26, 52, 78
Administration outcomes
Timepoints: Every 2, 3, or 4 weeks (every infusion)
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|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | Yes |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 40 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
Colombia |
Czech Republic |
Denmark |
France |
Germany |
Greece |
Israel |
Italy |
Mexico |
Norway |
Serbia |
Slovakia |
Spain |
Sweden |
Switzerland |
Turkey |
United Kingdom |
|
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
Each subject will have the opportunity to receive HyQvia for a maximum of 2.5 years OR up to 3 years if mandated by regulations |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 5 |