Clinical Trials By Design

The development of innovative and transformational therapies depends upon contributions of many people, especially those who generously participate in clinical trials. We invite you to learn about our clinical trial opportunities below.


Friedreich Ataxia (DT-216)

A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients with Friedreich Ataxia

RECRUITMENT STATUS: Active, Recruiting

Access to Investigational Medicines

Design is pioneering the research and development of novel small-molecule therapeutic candidates, called GeneTAC™ molecules, initially targeting serious genetic disorders caused by nucleotide repeat expansions. In order to obtain regulatory approval for these novel treatments and ultimately make them available to the broader patient population, we must first conduct a series of clinical trials to test their safety and effectiveness. Participation in a clinical trial, if certain criteria are met, offers the opportunity to contribute to research that may benefit many other patients.

More information about clinical trials of Design Therapeutics investigational medicines can be found at

Expanded Access or Compassionate Use

Design Therapeutics has developed an expanded access policy with guidelines under which patients with serious or life-threatening illness who are not eligible for our clinical trials might receive an investigational medicine before regulatory approval.

Criteria Used for Considering Expanded Access Requests:

  • Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition
  • There is no comparable or satisfactory alternative therapy to treat the disease or condition
  • Patient enrollment in a clinical trial is not possible
  • Sufficient clinical evidence of safety and effectiveness in the indication has been established, the potential benefit justifies the potential risks, and the potential risks are not unreasonable within the context of the disease or condition
  • Providing the investigational drug will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication
  • Adequate supply of the investigational drug is available

Consistent with the above criteria, Design Therapeutics is currently not accepting expanded access requests because our investigational medicines are in nonclinical or early stage of development and therefore lack sufficient evidence of clinical safety and effectiveness to support pre-approval expanded access. We continually evaluate the benefit-risk profile of each of our investigational medicines based on evolving data as they become available. If the policy changes, this webpage will be updated.

Please feel free to contact us at with any questions.