Phase 3
N=132
A Study of HyQvia and Gammagard Liquid (Kiovig) in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Bottom Line
View on ClinicalTrials.gov: NCT02549170 ↗Enrolled (actual)
132
Serious AEs
4.6%
Results posted
May 2023
Primary outcome: Primary: Epoch 1: Relapse Rate — 31.4; 9.7 percentage of participants — p==0.0045
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- HYQVIA (Biological); 0.25% albumin placebo solution with rHuPH20 (Biological); IGIV GAMMAGARD LIQUID/KIOVIG (Biological); IGIV GAMUNEX®-C (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- Feb 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Epoch 1: Relapse Rate |
31.4; 9.7 | =0.0045 sig |
| PRIMARY Epoch 2: Responder Rate |
100 | — |
| SECONDARY Epoch 1: Percentage of Participants Who Experience a Worsening of Functional Disability |
54.4; 37.5 | =0.0896 |
| SECONDARY Time to Relapse |
NA; NA | =0.002 sig |
| SECONDARY Epoch 1: Change From Pre-Subcutaneous (SC) Treatment Baseline in Rasch-built Overall Disability Scale (R-ODS) |
-6.1; -0.9 | =0.030 sig |
| SECONDARY Epoch 1: Number of Participants Experiencing Any Treatment-Emergent Serious and/or Non-serious Adverse Events (SAEs and/or AEs), Regardless of Causality |
40; 49; 5; 2 | — |
| SECONDARY Epoch 1: Number of Participants Experiencing Causally Related Serious and/or Non-Serious Adverse Events (SAEs and/or AEs) |
19; 38; 5; 1 | — |
| SECONDARY Epoch 1: Number of Participants With Serious and/or Non-serious Adverse Reactions (ARs) Plus Suspected ARs |
27; 41; 5; 1 | — |
| SECONDARY Epoch 1: Number of Treatment-emergent Serious and/or Non-serious Adverse Events (SAEs and/or AEs) Associated With Infusions Regardless of Causality |
144; 340; 5; 2 | — |
| SECONDARY Epoch 1: Number of Causally Related Serious and/or Non-serious Adverse Events (SAEs and/or AEs) Associated With Infusions |
53; 223; 5; 1 | — |
| SECONDARY Epoch 1: Number of Adverse Events (AEs) Temporally Associated With Infusions |
61; 251 | — |
| SECONDARY Epoch 1: Number of Serious and/or Non-serious Adverse Reactions (ARs) Plus Suspected ARs Associated With Infusions |
76; 261; 5; 1 | — |
| SECONDARY Epoch 1: Number of Treatment-emergent Systemic Adverse Events (AEs) Associated With Infusions |
149; 342 | — |
| SECONDARY Epoch 1: Number of Treatment-emergent Local Infusion Site Reactions Associated With Infusions |
20; 141 | — |
| SECONDARY Epoch 1: Number of Infusions in Participants for Which the Infusion Rate Was Reduced And/Or the Infusion Was Interrupted or Stopped Due to Intolerability And/Or Adverse Events (AEs) |
1; 3 | — |
| SECONDARY Epoch 1: Rates of Systemic and Local Adverse Events (AEs), Regardless of Causality, Expressed as Number of Events Per Infusion |
0.20; 0.34; 0.03; 0.24 | — |
| SECONDARY Epoch 1: Rates of Systemic and Local Adverse Events (AEs), Regardless of Causality, Expressed as Number of Events Per Participant |
1.84; 3.24; 0.29; 2.27 | — |
| SECONDARY Epoch 1: Rates of Systemic and Local Adverse Events (AEs), Regardless of Causality, Expressed as Number of Events Per 1000 Participant-year |
4689.22; 7341.52; 727.01; 5150.02 | — |
| SECONDARY Epoch 1: Rates of Causally Related Systemic and Local Adverse Events (AEs), Expressed As Number of Events Per Infusion |
0.06; 0.17; 0.02; 0.21 | — |
| SECONDARY Epoch 1: Rates of Causally Related Systemic and Local Adverse Events (AEs), Expressed As Number of Events Per Participant |
0.54; 1.6; 0.21; 2 | — |
| SECONDARY Epoch 1: Rates of Causally Related Systemic and Local Adverse Events (AEs), Expressed as Number of Events Per Participant-year |
1381.32; 3615.98; 545.26; 4529.1 | — |
| SECONDARY Epoch 1: Rates of Systemic and Local Adverse Reactions (ARs) Plus Suspected ARs, Expressed as Number of Events Per Infusion |
0.09; 0.20; 0.03; 0.24 | — |
| SECONDARY Epoch 1: Rates of Systemic and Local Adverse Reactions (ARs) Plus Suspected ARs, Expressed as Number of Events Per Participant |
0.80; 1.94; 0.29; 2.27 | — |
| SECONDARY Epoch 1: Rates of Systemic and Local Adverse Reactions (ARs) Plus Suspected ARs, Expressed as Number of Events Per 1000 Participant-Year |
2035.63; 4383.00; 727.01; 5150.02 | — |
| SECONDARY Epoch 1: Number of Participants Who Develop Binding and/or Neutralizing Antibodies to Recombinant Human Hyaluronidase (rHuPH20) |
7; 1 | — |
| SECONDARY Epoch 2: Number of Participants Experiencing Any Treatment-emergent Serious and/or Non-serious Adverse Events (SAEs and/or AEs), Regardless of Causality |
11; 3; 1; 0; 0; 0 | — |
| SECONDARY Epoch 2: Number of Participants Experiencing Causally Related Serious and/or Non-serious Adverse Events (SAEs and/or AEs) |
8; 3; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Number of Participants With Serious and/or Non-serious Adverse Reactions (ARs) Plus Suspected ARs |
10; 3; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Number of Treatment-emergent Serious and/or Non-serious Adverse Events (SAEs and/or AEs) Associated With Infusions Regardless of Causality |
0; 0; 0; 35; 25; 1 | — |
| SECONDARY Epoch 2: Number of Causally Related Serious and/or Non-serious Adverse Events (SAEs and/or AEs) Associated With Infusions |
23; 0; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Number of Adverse Events (AEs) Temporally Associated With Infusions |
25; 11; 0 | — |
| SECONDARY Epoch 2: Number of Serious and/or Non-serious Adverse Reactions (ARs) Plus Suspected ARs Associated With Infusions |
28; 11; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Number of Treatment-emergent Systemic Adverse Events (AEs) Associated With Infusions |
35; 25; 1 | — |
| SECONDARY Epoch 2: Number of Treatment-emergent Local Infusion Site Reactions Associated With Infusions |
0; 0; 0 | — |
| SECONDARY Epoch 2: Number of Infusions for Which the Infusion Rate Was Reduced and/or the Infusion Was Interrupted or Stopped Due to Intolerability and/or Adverse Events (AEs) |
1; 1 | — |
| SECONDARY Epoch 2: Rates of Systemic and Local Adverse Events (AEs), Regardless of Causality, Expressed as Number of Events Per Infusion |
0.11; 0.41; 0.06; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Systemic and Local Adverse Events (AEs), Regardless of Causality, Expressed as Number of Events Per Participant |
2.19; 6.25; 1.00; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Systemic and Local Adverse Events (AEs), Regardless of Causality, Expressed as Number of Events Per 1000 Participant-year |
4645.26; 12806.80; 2135.96; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Causally Related Systemic and Local Adverse Events (AEs), Expressed As Number of Events Per Infusion |
0.07; 0.13; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Causally Related Systemic and Local Adverse Events (AEs), Expressed as Number of Events Per Participant |
1.44; 2; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Causally Related Systemic and Local Adverse Events (AEs), Expressed as Number of Events Per Participant-Year |
3052.6; 4098.18; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Systemic and Local Adverse Reactions (ARs) Plus Suspected Ars, Expressed as Number of Events Per Infusion |
0.09; 0.18; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Systemic and Local Adverse Reactions (ARs) Plus Suspected ARs, Expressed as Number of Events Per Participant |
1.75; 2.75; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Rates of Systemic and Local Adverse Reactions (ARs) Plus Suspected ARs, Expressed as Number of Events Per 1000 Participant-Year |
3716.21; 5634.99; 0; 0; 0; 0 | — |
| SECONDARY Epoch 2: Percentage of Participants With Clinically Meaningful Improvement in Functional Ability |
100; 100 | — |
Summary
The aim of this study is to learn more about the following treatment options in adults with CIDP:
* Subcutaneous self-infusion with HyQvia.
* Intravenous infusion with Gammagard/Kiovig. Gammagard and Kiovig are the brand names for the same immunoglobulin compound.
The study is in two parts. In Part 1, participants receive either HyQvia or a placebo subcutaneously. In Part 2 (only for participants who have a CIPD relapse during Part 1), participants will receive Gammagard Liquid/Kiovig intravenously. US participants will receive Gamunex-C.
The first SC infusion will be given in the study clinic. The remaining SC infusions may be given in the study clinic or the participant's home. This will be decided by the study doctor and whether the participant or their caregiver can do the self-infusion.
Eligibility Criteria
Inclusion Criteria
- Males or females of age greater than or equal to (>=)18 years old at the time of screening.
- Participant has a documented diagnosis of definite or probable Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) (focal atypical CIDP and pure sensory atypical CIDP will be excluded), as confirmed by a neurologist specializing/experienced in neuromuscular diseases to be consistent with the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) 2010 criteria (European Federation of Neurological Societies, 2010). Fulfillment of electrodiagnostic criteria must be confirmed by an independent qualified/experienced central reader.
- Participant has responded to IgG treatment in the past (partial or complete resolution of neurological symptoms and deficits), and must currently be on stable doses of IGIV treatment within the dose range equivalent to a cumulative monthly dose of 0.4 to 2.4 gram per kilogram (g/kg) BW (inclusive) administered intravenously for at least 12 weeks prior to screening. The dosing interval of IGIV treatment must be between 2 and 6 weeks (inclusive). Variations in the dosing interval of up to ± 7 days or monthly dose amount of up to ± 20% between participant's pre-study Immunoglobulin G (IgG) infusions are within acceptable limits.
- INCAT disability score between 0 and 7 (inclusive). Participants with INCAT scores of 0, 1 (whether from upper or lower extremities), or 2 (if at least 1 point is from an upper extremity) at screening and/or baseline will be required to have a history of significant disability as defined by an INCAT disability score of 2 (must be exclusively from the lower extremities) or greater documented in the medical record. Participants will be eligible if one of the below eligibility criteria are met:
Screening and Baseline INCAT disability score of between 3 and 7 inclusive.
- Screening and/or Baseline INCAT disability score of 2 (both points are from lower extremities).
- Screening and/or Baseline INCAT disability score of 2 (both points are not from lower extremities) AND has at least a score of 2 or greater documented in the medical record prior to screening. If a score was greater than 2 documented in the medical record prior to screening at least 2 points must be from lower extremities.
- Screening and/or Baseline INCAT disability score of 0 or 1 AND has at least a score of 2 or greater (both from lower extremities) documented in the medical record prior to screening, at least 2 points must be from lower extremities.
- If female of childbearing potential, the participant must have a negative pregnancy test at screening and agree to employ a highly effective contraceptive measure throughout the course of the study and for at least 30 days after the last administration of investigational product (IP).
- Participant is willing and able to sign an Informed Consent Form (ICF).
- Participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria
- Participants with Focal atypical CIDP or pure sensory atypical CIDP.
- Any neuropathy of other causes, including:
- Hereditary demyelinating neuropathies, such as hereditary sensory and motor neuropathy (HSMN) (Charcot-Marie-Tooth [CMT] disease), and hereditary sensory and autonomic neuropathies (HSANs).
- Neuropathies secondary to infections, disorders, or systemic diseases such as Borrelia burgdorferi infection (Lyme disease), diphtheria, systemic lupus erythematosus, POEMS (polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes) syndrome, osteosclerotic myeloma, diabetic and non-diabetic lumbosacral radiculoplexus neuropathy, lymphoma, and amyloidosis.
- Multifocal acquired demyelinating sensory and motor neuropathy (MADSAM).
- Multifocal motor neuropathy (MMN).
- Drug-, biologic-, chemotherapy-, or toxin-induced peripheral neuropathy.
- Immunoglobulin M (IgM) paraproteinemia, including IgM monoclonal gammopathy wi
Data sourced from ClinicalTrials.gov (NCT02549170). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.