Supporting At-Home Treatment for a Phase 4 Immunodeficiency Trial

Study Snapshot

• Study: Phase 4 Primary Immunodeficiency (PID) trial evaluating Hizentra® (IgPro20)
• 64 in-home visits delivered
• 5 patients supported
• 3 research sites supported
• 11-month study duration
• 0 missed patient visits
• 11 days from Last Patient Last Visit to database lock
• Flexible home-based care delivered through Decentralized Clinical Services
• Country: United States

Study Overview

Primary immunodeficiency (PID) is a group of rare disorders that weaken the immune system, leaving patients vulnerable to recurrent and severe infections. Immunoglobulin replacement therapy plays a vital role in reducing infection risk and helping patients maintain their quality of life.

This Phase 4 clinical trial evaluated whether patients newly diagnosed with PID could begin treatment with Hizentra® (IgPro20) immediately following diagnosis, without first receiving intravenous immunoglobulin (IVIG), while safely self-administering treatment at home. To support this objective, mdgroup delivered Decentralized Clinical Services, enabling patients to complete study visits in their own homes while maintaining protocol compliance and high-quality clinical data collection.

The Challenge

The study required mdgroup to support patients across multiple research sites in the United States with an intensive schedule of home visits, patient education, sample collection and investigational product infusion support.

Several operational challenges had to be overcome to ensure successful study delivery:

• An intensive visit schedule requiring multiple visits to be completed on exact protocol days, with little or no scheduling flexibility.
• Healthcare professionals needed specialist training on the investigational infusion pump to support self-administration and respond to site queries regarding dosing.
• The first infusion had to take place at the research site before patients transitioned to home-based care, requiring careful coordination between research sites and healthcare professionals.
• One patient living with severe multiple sclerosis was unable to self-administer treatment, requiring mdgroup healthcare professionals to administer the investigational product while maintaining protocol compliance.
• Another patient needed to travel out of state during the study, requiring seamless continuity of care to prevent missed visits and protocol deviations.

The Solution

From the outset, mdgroup worked as an extension of the sponsor and site teams to ensure patients could safely receive treatment in their own homes while maintaining the highest standards of clinical quality.

Members of the mdgroup project team attended the Investigator Meeting in Philadelphia, where they received hands-on training with the investigational infusion pump while building strong relationships with site staff. This knowledge was incorporated into comprehensive study-specific training for every healthcare professional, with each clinician provided their own training pump to develop confidence before supporting patientts.

Working collaboratively across Clinical Management and the resourcing team, healthcare professionals were identified in advance for each participating site. This proactive planning ensured that visits with fixed protocol dates could be delivered without delay and enabled patients to transition smoothly from site-based treatment to home-based care.

When one patient was unable to self-administer treatment due to a severe comorbidity, mdgroup collaborated closely with the sponsor and research site to expand the service scope, providing trained healthcare professionals to administer the investigational product in the patient’s home.

Later in the study, when the same patient needed to travel out of state, mdgroup sourced a qualified local healthcare professional to continue treatment without interruption. This flexible approach ensured there were no missed visits, protected study integrity and enabled the patient to remain on the trial despite significant clinical and logistical challenges.

Throughout the study, mdgroup’s healthcare professionals became a trusted extension of each research site, maintaining continuous communication with investigators while supporting patients through every stage of treatment and ensuring safe, compliant home-based care.

The Results

Over an 11-month period, mdgroup successfully delivered 64 in-home visits across three research sites and five patients, providing complex home-based clinical support while maintaining protocol compliance throughout the study.

Key outcomes included:

• Successful delivery of one of mdgroup’s first Phase 4 studies demonstrating the value of Decentralized Clinical Services in supporting home-based treatment.
• Zero missed patient visits despite an intensive visit schedule and complex patient requirements.
• Flexible support that enabled patients to receive treatment at home while maintaining continuity of care during travel when required.
• Rapid adaptation of services to support a patient who was unable to self-administer treatment, ensuring they could continue participating in the study.
• High-quality home data collection and real-time quality control that contributed to a clean dataset, enabling database lock just 11 days after the last patient completed their final visit.

The Impact

By combining specialist clinical expertise with flexible, patient-centred service delivery, mdgroup reduced the burden of trial participation while maintaining the highest standards of protocol compliance and data quality.

Patients were able to receive treatment in the comfort of their own homes, creating a real-world treatment experience that aligned with the objectives of the study while reducing the need for unnecessary site visits. For the sponsor and research sites, mdgroup became a trusted extension of the clinical team, delivering operational flexibility, high-quality data collection and an exceptional patient experience that helped keep the study on track from first patient to database lock.

Client Testimonial

“It has been a pleasure working with the mdgroup team. Thank you for your continued support, responsiveness and flexibility in helping us overcome challenges throughout the study. Your commitment to delivering high-quality home healthcare services made a meaningful difference and contributed significantly to our success. I hope we have the opportunity to collaborate again in the future.”

– Study Execution Lead, Clinical Operations

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