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Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (The PATH Study): study protocol for a randomized controlled trial.

ABSTRACT: Subcutaneous administration of Ig (SCIg) has gained popularity as an alternative route of administration but has never been rigorously examined in chronic inflammatory demyelinating polyneuropathy (CIDP).The primary objective of the PATH study (Polyneuropathy and Treatment with Hizentra) is to determine the efficacy of two different doses of SCIg IgPro20 (0.2 g/kg bw or 0.4 g/kg bw) in a 24-week maintenance treatment of CIDP in comparison to placebo. The primary efficacy endpoint will be the proportion of patients who show CIDP relapse (1-point deterioration on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score) or are withdrawn within 24 weeks after randomization for any reason. IVIg-dependent adult patients with definite or probable CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society who fulfil the inclusion and exclusion criteria will be eligible. Based on sample-size calculation and relapse assumptions in the three arms, a sample size of 58 is needed per arm (overall sample size will be 350, of which 174 will be randomized). All eligible patients will progress through three study periods: an IgG dependency period (?12 weeks) to select those who are Ig dependent; an IVIg restabilization period (10 or 13 weeks), which will be performed using the 10 % IgPro10 product; and an SC treatment period (24 weeks, followed by a 1-week completion visit after last follow-up). Patients showing IVIg restabilization will be randomized to demonstrate the efficacy of SCIg IgPro20 maintenance treatment over placebo. After completing the study, subjects are eligible to enter a long-term, open-label, extension study of 1 year or return to their previous treatment. In case of CIDP relapse during the 24-week SC treatment period, IgPro10 rescue medication will be offered. Safety, tolerability, and patients' preference of Ig administration route will be examined.The PATH trial, which started in March 2012, is expected to finish at the end of 2016. The results will increase knowledge about the efficacy, safety, and tolerability of SCIg in maintenance management of CIDP patients.ClinicalTrials.gov, NCT01545076 . Registered on 1 March 2012.

SUBMITTER: van Schaik IN

PROVIDER: S-EPMC4960813 | biostudies-literature | 2016 Jul

REPOSITORIES: biostudies-literature

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Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (The PATH Study): study protocol for a randomized controlled trial.

van Schaik Ivo N IN van Geloven Nan N Bril Vera V Hartung Hans-Peter HP Lewis Richard A RA Sobue Gen G Lawo John-Philip JP Mielke Orell O Cornblath David R DR Merkies Ingemar S J IS

Trials 20160725 1

<h4>Background</h4>Subcutaneous administration of Ig (SCIg) has gained popularity as an alternative route of administration but has never been rigorously examined in chronic inflammatory demyelinating polyneuropathy (CIDP).<h4>Methods/design</h4>The primary objective of the PATH study (Polyneuropathy and Treatment with Hizentra) is to determine the efficacy of two different doses of SCIg IgPro20 (0.2 g/kg bw or 0.4 g/kg bw) in a 24-week maintenance treatment of CIDP in comparison to placebo. The ...[more]

PMID: 27455854

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Project description:BackgroundChronic inflammatory demyelinating polyneuropathy (CIDP) is a rare, progressive autoimmune disease causing peripheral nervous system dysfunction. Guidelines recommend immunoglobulin (IG) therapy as an immunomodulatory agent in CIDP. Drawbacks and unmet needs with intravenous immunoglobulin (IVIG) include adverse effects and wear-off effects, along with the burden of administration based on site of care. Subcutaneous administration of Hizentra, a subcutaneous immunoglobulin (SCIG) reduces patient burden by allowing self-administration outside the hospital setting and has fewer adverse events (AEs).ObjectiveWe aimed to compare the expected cost of treatment and the budget impact of Hizentra compared with IVIG for maintenance treatment of CIDP in the United States.MethodsA decision tree model was developed to estimate the expected budget impact of maintenance treatment with Hizentra for US stakeholders. The model adopts primarily a US integrated delivery network perspective and, secondarily, a commercial perspective over a 1-year time horizon. Pharmacy costs were based on a payment mix of average sales price (73%), wholesale acquisition cost (2%), and average wholesale price (25%). Costs in the model reflect 2022 US dollars. In accordance with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) guidelines and recommendations for budget impact modeling, no discounting was performed. The PATH clinical study of Hizentra maintenance in CIDP was used to determine clinical inputs for relapse rates at initial assessment (24 weeks) and at 52 weeks for Hizentra. The ICE clinical study of Gamunex maintenance in CIDP was the basis of relapse rates for Gamunex (and other IVIGs). Literature-based estimates were obtained for infusion costs by site of care, costs of IVIG infusion-related complications, and significant IVIG AE rates. Hizentra AE rates from the US Hizentra prescribing information were assessed but were not included in the model as the AEs in CIDP were mild, easily treated, and self-limited. Sensitivity analyses and scenario analyses were conducted to evaluate variations from the base case.ResultsThe model showed that a Hizentra starting dose of 0.2 g/kg is expected to result in annual cost savings of US$32,447 per patient compared with IVIG. For a hypothetical 25-million-member plan, the budget impact of a 10% market share shift from IVIG to Hizentra is expected to result in savings of US$2,296,235.ConclusionThis analysis projects that Hizentra is likely associated with favorable economic benefit compared with IVIG in managing CIDP.

Dataset Information

Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (The PATH Study): study protocol for a randomized controlled trial.

Publications

Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (The PATH Study): study protocol for a randomized controlled trial.

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