Current Trials

Current Clinical Trials

Explore active studies at Uncommon Cures

Browse current clinical trial opportunities and learn more about studies that may be available for patients and families. Some studies may include in-person visits, some may be remote, and some may use a hybrid model.

Looking for a study?

We know that many patients, parents, caregivers, and advocacy groups come here to learn what studies may currently be active and what disease areas we are working in.

Explore the opportunities below to learn more about each study, who it may be for, and what the next step may look like.

Why Participate in a Clinical Trial?

By participating in a clinical trial, you gain access to cutting-edge treatments that may not be available elsewhere. You also play a vital role in helping us advance research that can provide better treatment options for the rare disease community.

Select a study below to learn more about eligibility, participation, and next steps.

HARMONY Study for Patients With HCU

Ornithine Transcarbamylase (OTC) Deficiency Research

Phenylketonuria PKU Study For Patients Taking Palynziq

Friedreich's Ataxia Research

What participation may look like

Every clinical trial is different. Some studies may include visits to our facility, some may be remote, and some may use a hybrid approach that includes both in-person and virtual touchpoints.No matter how a study is structured, our team is here to help patients and families feel informed, supported, and guided along the way.

In-Person

Some studies may include visits at our facility for certain parts of participation.

Remote

Some studies may allow participation from home using virtual communication and remote coordination.

Hybrid

Some studies may include a mix of in-person visits, virtual support, local clinics, or home-based services.

What happens next?

If you see a study that may be relevant, the next step is to reach out through the study page or contact form.

Not every study is right for every family, but we want the process of learning more to feel clear and supportive.

Clinical Trial FAQ

Helpful answers to common questions about exploring studies, learning more about participation, and taking the next step.

What is a clinical trial?

A clinical trial is a research study that helps doctors and researchers learn more about a potential treatment, how it works, and whether it may be safe and effective.

Will every study require travel?

No. Some studies may include in-person visits, while others may be remote or use a hybrid approach. The study details will help explain what participation may involve.

How do I know if a study may be right for me or my family?

Every study has different requirements. A good next step is to review the study information and reach out to learn more about whether it may be a fit.

What happens after I submit interest?

Our team can help answer questions, explain the study in more detail, and guide you through what the next steps may look like.

Can I ask questions before deciding?

Yes. We want patients and families to feel informed and supported before making any decisions about participation.

What if I do not see the right study right now?

Study opportunities change over time. If you do not see the right fit today, you can still reach out so our team can help point you in the right direction.