Evaluation of Safety, Tolerability, PK and PD of AB1 in Adult Sickle Cell Disease Patients - Clinical Trial

What is the Purpose of this Study?

We are doing this study to find out if an investigational drug called AB1 (the study drug) is a safe and effective option for people with sickle cell disease (SCD). We also want to see what dose of the study drug appears to work best.

What is the Condition Being Studied?

Sickle Cell Disease (SCD)

Who Can Participate in the Study?

Adults ages 18-45 who:

- Are diagnosed with SCD

- Have not managed a sickle cell crisis for at least 28 days when beginning the study

- Have fetal hemoglobin (HbF) that measures at less than 15% at the beginning of the study

For more information about who can join this study, please contact the study team at lindsey.muller@duke.edu.

Age Group

What is Involved?

If you choose to join this study, you will take the study drug once each day for 8 weeks. The study drug is a pill that you take by mouth. The dose of the study drug that you get will depend on when you join the study. These are the dose levels of the study drug that will bet tested:

- 2 mg

- 4 mg

- 8 mg

- 10 mg

- 12 mg

- 16 mg

- 32 mg

Procedures duing the study visits will include:

- Medical history and concomitant medications

- Physical examination and vital signs

- Blood will be collected for various laboratory studies

- Adverse events and crisis frequency/occurrence

Study Details

Full Title
A Phase 1/2 Open-Label, Dose Escalating Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AB1 in Adult Patients with Sickle Cell Disease (SCD)
Principal Investigator
Protocol Number
IRB: PRO00110179
NCT: NCT05261711
Phase I/II
Enrollment Status
Open for Enrollment