Clinical Trial Trends: Rare CNS

In rare disease research, central nervous system (CNS) issues are very common, making up some 50% of all rare diseases on record. However, the field is extremely heterogeneous. Take autoimmune demyelinating diseases, for example. While they do all impact organ-specific immunity, that’s really the limit of their commonality. Each rare autoimmune demyelinating issue has a unique pathology with its own functional consequences. For example, Multiple Sclerosis (MS) is fundamentally different from rare MS variants and requires a different approach to diagnosis and disease management. 

In this article, we will examine the rare CNS research landscape, exploring the clinical trial pipeline across phases and mechanisms using research from Citeline. We will conclude by outlining Precision for Medicine’s past and current experience in the space. 

Understanding the rare CNS clinical trial landscape  

The complexity and diversity of rare CNS cases worldwide drive an ongoing quest for more effective treatments. Here, we explore the dynamic and evolving trial landscape of rare CNS research, highlighting key trends and statistics from Citeline’s comprehensive database. 

Rare CNS Clinical Trials by Status 

With 5,750 registered trials for rare CNS, approximately 24% (1,364) are in the planned or ongoing stages, indicating a significant commitment to advancing treatment. 

Trials by Status 

Citeline Trialtrove® – 30JUN2024 

Rare CNS Trials by Start Date 

Reviewing the start dates for rare CNS trials reveals a clear trend of escalating research activities year-over-year. 

Planned & Ongoing Rare CNS Trials by Start Date 

 Citeline Trialtrove® – 30JUN2024 

Rare CNS Trials by Country  

The United States leads with 485 planned and ongoing rare CNS trials, followed by China and Canada respectively, showcasing a global effort in research. 

Planned & Ongoing Rare CNS Trials by Site Country 

Citeline Trialtrove® – 30JUN2024 

Rare CNS Trials by Indication  

The distribution of rare CNS trials across indications is varied, but Amyotrophic Lateral Sclerosis and Muscular Dystrophy are particular areas of focus right now. 

Top Planned & Ongoing Rare CNS Trials by Indication 

Citeline Trialtrove® – 30JUN2024 

Rare CNS Trials by Phase

Most planned and ongoing rare CNS studies are well-distributed across phases. 

Planned & Ongoing Rare CNS Trials by Phase  

Citeline Trialtrove® – 30JUN2024 

Precision's expertise in rare CNS research  

Precision for Medicine has established a profound footprint in rare CNS research, orchestrating 27 trials that explore groundbreaking therapies. 

Rare CNS Trial Starts  

Amidst the challenges of rare CNS research, Precision for Medicine has successfully initiated 13 trials since 2019, striving to transform the clinical landscape. 

Precision for Medicine's Rare CNS Trials by Start Date 

Citeline Trialtrove® – 30JUN2024 

Precision's Reach in Rare CNS Research  

Precision for Medicine’s rare CNS clinical trial experience extends globally, with trials conducted in 18 countries, including key sites in the United States, Australia, and Europe. 

Precision for Medicine's Rare CNS Trials by Site Country 

Citeline Trialtrove® – 30JUN2024 

Trials by Phase

Our strategic emphasis on development and innovation is evident, with 15 early phase rare CNS trials, prioritizing rapid progression through these critical Phase 1, 1-2, and 2 research. 

Precision for Medicine's Rare CNS Trials by Phase 

Citeline Trialtrove® – 30JUN2024 

Precision's Experience by Indication 

Precision for Medicine’s trials in rare CNS cover various indications across a wide range of pathologies. 

 Precision’s Rare CNS Trials by Indication 

Citeline Trialtrove® – 30JUN2024 

Understanding the Precision difference

Overall, the rare CNS clinical trials environment reflects extensive global participation and a diverse phase distribution. Precision for Medicine is a proud partner to sponsors working in rare CNS research. Our expertise, capabilities and and experience make us a reliable partner to patients, caregivers, sponsors and sites. 

Learn about our unique approach to rare disease research.