A Phase 2 Study of FP-045 in Children and Young Adults With Fanconi Anemia


Full Title

A Multinational, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of FP-045 in Patients with Fanconi Anemia


Researchers want to find the best dose of FP-045 to treat low blood cell counts in people with Fanconi anemia. The people in this study have Fanconi anemia with blood counts that are mildly or moderately low.

Fanconi anemia causes high levels of substances called aldehydes in the body, which can damage blood cells. FP-045 increases the activity of aldehyde dehydrogenase, an enzyme that helps the body break down aldehydes. By protecting blood cells from aldehydes, FP-045 may help improve your blood counts. FP-045 is taken orally (by mouth).

Who Can Join

To join this study, there are a few conditions. You or your child must:

  • Have Fanconi anemia with mildly or moderately low blood counts.
  • Have not had a bone marrow transplant.
  • Be age 3-25.


For more information and to see if you or your child can join this study, please call 833-MSK-KIDS.




Phase II (phase 2)

Disease Status

Newly Diagnosed



ClinicalTrials.gov ID