Delivering Global Access to Life-Changing Gene Therapy for Children with Ultra-Rare Disease

Study Overview

In 2019, mdgroup partnered with Rocket Pharmaceuticals to support a Phase I/II clinical study investigating a gene therapy for pediatric patients with severe Leukocyte Adhesion Deficiency-I (LAD-I), an ultra-rare and life-threatening immunodeficiency.

The study was led by Rocket Pharmaceuticals in collaboration with global investigators and clinical sites, with mdgroup providing operational and patient support services. Participants were dispersed globally and required highly specialized care, demanding a flexible, patient-first approach to ensure access, continuity, and long-term engagement.

In 2024, this partnership expanded into a long-term follow-up (LTFU) study, introducing a hybrid model of travel, payments, and in-home clinical services. mdgroup will continue supporting patients and sites for up to 15 years, ensuring vital long-term data is captured while minimizing burden on families.

Results at a glance:

• Global patient support across 5+ countries
• Hybrid trial delivery across site and home
• 0 missed visits across treatment and follow-up
• Up to 9 months’ relocation support per family
• Up to 15 years of ongoing patient support
• FDA approval of gene therapy

The Challenge

LAD-I is a rare genetic condition that severely compromises the immune system, leaving young patients vulnerable to recurrent, life-threatening infections and frequent hospitalizations.

Delivering this study presented significant operational and human challenges. Patients and their families were required to travel long distances—often internationally—and, in many cases, relocate for extended periods during treatment and recovery.

At the same time, the study required precise coordination across multiple regions, with several participants enrolled simultaneously, including siblings within the same family. These complexities were further heightened during the COVID-19 pandemic, where safety, reassurance, and continuity of care became even more critical.

Ensuring that no visits were missed—across both the initial study and continuing into the 15-year follow-up period—was essential to the success of the trial.
Participants travelled both internationally and domestically across regions including North America, Europe, and the Middle East, with individualized coordination to support access to treatment and follow-up care.

The Solution

mdgroup delivered a fully integrated, patient-first support model designed to remove barriers to participation and ensure a seamless experience for both patients and study teams.

Global travel and relocation logistics were carefully managed end-to-end, including international and domestic travel, visa document support, and the coordination of long-term accommodation for families relocating for up to nine months. Financial support was also provided to ease the burden of daily living, enabling families to focus entirely on treatment and recovery.

Recognizing the vulnerability of this patient population, particularly during the pandemic, additional measures were introduced to create safe and supportive living environments throughout their stay.

As the study progressed into its long-term follow-up phase, mdgroup evolved its approach to incorporate a hybrid delivery model. Patients continue to receive travel and financial support for site visits, while also benefiting from in-home clinical care delivered by trained healthcare professionals.

This includes the collection of lab samples, vital signs, and clinical assessments directly in the home, with careful coordination to ensure data is accurately captured across multiple laboratories and sites. This model reduces the burden on families while maintaining the integrity and consistency of clinical data collection.

What Made This Unique

• Delivering complex global logistics for an ultra-rare pediatric population, including multiple participants within the same family
• Supporting extended relocations for young patients and caregivers during critical treatment periods
• Maintaining continuity of care and patient support throughout the COVID-19 pandemic
• Implementing a hybrid model combining travel, payments, and in-home clinical services over a 15-year follow-up period

The Outcome

In March 2026, Rocket Pharmaceuticals announced the FDA approval of KRESLADI™, marking an important milestone for both this global study and the treatment of LAD-I.

mdgroup’s support ensured that patients were able to access treatment regardless of location, attend every required visit, and remain engaged throughout both the initial study and its long-term follow-up. Through this, critical data points were collected, contributing to the body of evidence supporting the regulatory submission.

KRESLADI™ was approved under the FDA’s accelerated approval pathway based on CD11a biomarker expression. Continued approval may be contingent upon verification of clinical benefit in long-term follow-up.

The Impact

For the Client

By alleviating logistical and geographic barriers, mdgroup supported Rocket Pharmaceuticals to deliver a complex global study. The seamless integration of travel, financial support, and in-home care ensured high levels of patient engagement and retention, while supporting the delivery of an important treatment and consistent collection of critical long-term data.

For Sites & Healthcare Professionals

Study teams benefited from a significant reduction in operational burden, with mdgroup managing complex logistics and delivering in-home clinical services. This allowed sites to focus on clinical delivery, while maintaining confidence in the quality and consistency of data collected both on-site and remotely.

For Patients & Families

For patients living with LAD-I, access to treatment may represent an important therapeutic option. Through comprehensive support, families were able to focus on care rather than logistics during extended periods away from home.

Today, patients who once faced severe, life-threatening infections have resume aspects of daily life based on follow-up observations—attending school, playing sports, and living full, active lives. For these families, this study has been life-changing.

Individual patient outcomes may vary, and long-term clinical benefit continues to be evaluated.

A father with three children in the study had this to say:

“mdgroup was fantastic to work with from start to finish. They handled all the planning for us, including travel and housing, which allowed us to focus on what mattered most: our kids.

As we’ve transitioned into the long-term follow-up phase, we’ve switched to in-home visits with both the nurse and doctor. This has been incredibly convenient for our busy family, especially with three kids involved in multiple sports, and it creates a much more comfortable experience for them overall.”

Looking Ahead

mdgroup will continue to support this study for the next 15 years, delivering a flexible, hybrid model that keeps patients engaged, reduces burden, and ensures the successful collection of long-term clinical data.

Client Feedback

“I have had the opportunity to work closely with mdgroup since 2019, when we treated the first Rocket patient in Europe. Over the years, our collaboration has spanned multiple studies, and their support has been consistently exceptional.

The mdgroup team, and particularly Brittany, have always been highly responsive, proactive, and solution-oriented. Their ability to quickly address challenges and provide effective support has been instrumental in maintaining momentum across complex study activities.

They have also provided invaluable support in managing international patients, which is especially critical in the context of rare diseases. Their experience and dedication have made a meaningful difference for patients, their families, and the study team.

Having partnered with them for several years, I truly value their reliability, professionalism, and collaborative approach. mdgroup has been a valued operational partner throughout the study.”

– Annelise von Bergen, MSc, Global Study Operations Lead – LVV programs

“I’ve had the pleasure of working with mdgroup since joining Rocket nearly four years ago, and they have been an integral partner throughout that time. From study start-up through each patient visit, their team has consistently made a meaningful impact on our programs.

Their ability to collect critical biospecimens in patients’ homes has been especially valuable, enabling us to continue generating important data across the duration of our studies. Their expertise in executing decentralized trials globally has made complex collections in rare disease populations not only feasible, but in many cases seamless.

We are truly grateful for their partnership and for the rigor, flexibility, and patient-centric approach they bring—helping make study participation as smooth as possible for our patients.”

– Nicholas Broccoli, MPH, Senior Manager, Biospecimen Operations

 

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