Clinical Trial Trends: Rare Metabolic

Metabolic diseases impact a person’s ability to process carbohydrates, sugar, protein, fat, or a combination thereof. In turn, development and growth can be impacted, organs affected, and the symptoms can be severe, even life-threatening. These metabolic issues usually have a genetic origin and are present in as many as 1 in 1,500 births (the incidence rate is closer to 1 in 2,500 in developed countries), but they are often quite rare individually.  

In either case, metabolic issues seem to occur in populations with certain markers and in places with high consanguinity. Some are present at birth, some manifest depending on environmental factors, and any could manifest alongside other issues. This combination of factors makes rare metabolic research even more challenging. 

In this article, we will look at some of the trends in rare metabolic research by leveraging data from Citeline. We will continue by looking at Precision’s experience in this therapeutic area. 

Understanding the Rare Metabolic Clinical Trial Landscape  

Metabolic disorder has been getting more attention in health media, but many metabolic conditions are much more rare. As such, there are many fewer clinical trials in this therapeutic area than many other indications. Acute Myelogenous Leukemia (AML), for example, is considered a rare disease and it has had almost 5,400 trials conducted. Rare Cardiovascular as a whole has had barely 6,000.  

Rare Metabolic Clinical Trials by Status 

Around 17% of rare metabolic trials are current in that their status is Open or Planned.  

Rare Metabolic Trials by Status 

Citeline Trialtrove^® – 18JUN2024

Rare Metabolic Trials by Start Date 

The number of current rare metabolic trials is increasing year over year. More than 250 trials in this indication were started in 2023. 

Planned & Ongoing Rare Metabolic Trials by Start Date 

Citeline Trialtrove^® – 18JUN2024 

Rare Metabolic Trials by Country  

Interest in rare metabolic research spans the globe. The countries with the most current sites involved with rare metabolic clinical trials are the United States and China. 

Planned & Ongoing Rare Metabolic Trials by Site Country 

Citeline Trialtrove^® – 18JUN2024 

Rare Metabolic Trials by Indication  

As mentioned, the rare metabolic therapeutic area includes a variety of indications. Here, we look at which indications have the most active clinical trials. 

Planned & Ongoing Rare Metabolic Trials by Indication 

Citeline Trialtrove^® – 18JUN2024 

Rare Metabolic Trials by Phase  

Current rare metabolic trials are seeing representation in both early and late phase research. 

Planned & Ongoing Rare Metabolic Trials by Phase 

 Citeline Trialtrove^® – 18JUN2024 

Precision's Expertise in Rare Metabolic Research  

Despite the infrequency of rare metabolic research, Precision has worked on 30 clinical trials in this space.  

Rare Metabolic Trial Starts  

Precision has started 6 rare metabolic trials since 2019, and we have several active trials in the space. 

 Precision for Medicine's Rare Metabolic Trials by Start Date 

Citeline Trialtrove^® – 18JUN2024 

Precision's Reach in Rare Metabolic Research  

Looking at our global footprint, Precision has conducted rare metabolic clinical trials in 48 countries. 

Precision for Medicine's Rare Metabolic Trials by Site Country 

Citeline Trialtrove^® – 18JUN2024
 

Trials by Phase  

While Precision has conducted comparatively more early phase rare metabolic trials than later phase, the difference is marginal. 

Precision for Medicine's Rare Metabolic Trials by Phase 

Citeline Trialtrove^® – 18JUN2024
 

Precision's Experience Indication 

Precision’s rare metabolic trials have encompassed patients with a wide variety of diagnoses.  

Precision for Medicine's Rare Metabolic Trials by Indication 

Citeline Trialtrove^® – 18JUN2024

Understanding the Precision Difference 

Thanks to genetic differences, scarcity of patient populations, and high amounts of comorbidity, finding candidates for participation in rare metabolic trials can be challenging so choosing a CRO who understands those populations is critical—both for recruitment and retention.

Precision understands the patient burden of rare metabolic issues and how best to mitigate those difficulties. Learn the difference Precision makes. We are your best partner for rare metabolic trials.