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)

About the study

This will be an open-label, dose escalating study with a starting dose of 2mg. Up to 4 additional cohorts will be enrolled at subsequently higher doses of 4mg, 8mg, 16mg, and 32mg. Each dose will be taken orally, once daily, for 8 weeks.

Study point of contact

Nirmish Shah, MD
[email protected]
Lindsey Muller
[email protected]


18 Years - 45 Years


Phase 1/Phase 2

Study type






participation requirements

Written, informed consent
Age 18 to 45 years of age, inclusive at screening
Confirmed SS or S-b0-thalassemia SCD
Sickle crisis rate of 2-10 within the past year with no crisis in the last 28 days
HbF <8.6% of total Hb at screening Regular compliance with comprehensive care and previous therapy -

participation restrictions

Experienced severe sepsis or septic shock within the previous 12 weeks
Febrile illness in the 1 week prior to baseline visit
Acute complications due to SCD (i.e., hospitalization, acute pain, or acute chest syndrome) in the 28 days prior to screening visit
Plans for hospitalization, surgery, or other major procedures during the duration of the study or between screening and baseline
ALT ≥2X the upper limit of normal or albumin <2.0 mg/dL or direct (conjugated) bilirubin ≥ 1.5 mg/dl* Serum creatinine >2.9 mg/dL and calculated creatinine clearance <30 mL/min# * Platelet count >800 x 109/L OR <150 x 109/L* Absolute neutrophil count <1.5 x 109/L* Currently pregnant or breastfeeding Female of active childbearing potential$ who is unwilling or unable to adhere to the contraception requirements specified in the protocol Male with female partner(s) of childbearing potential$ who is unwilling or unable to adhere to the contraception requirements specified in the protocol Altered mental status or recurrent seizures requiring anti-seizure medications Moribund or any concurrent disease (e.g., hepatic, renal, cardiac, metabolic) of such severity that death within 24 weeks is likely Concurrent diagnosis of malignancy including MDS, leukemia, or an abnormal karyotype Known Vitamin-B12, folate, or iron deficiency New York Heart Association (NYHA) class III/IV status Eastern Co-operative Oncology Group (ECOG) performance status ≥3 Participant is on chronic transfusion therapy (e.g., for history of TIA or stroke) and medically contraindicated to discontinue transfusions (unless multiple allo-antibodies prevent the patient from getting transfusions as scheduled) Blood transfusion in the 28 days prior to screening visit or between screening and baseline visits Known history of illicit drug or alcohol abuse within the past 12 months. Current treatment with Oxbryta or Adakveo (must be off therapy for 30 days for Oxbryta with no plans to restart and off therapy for 3 months for Adakveo with no plans to restart) Other experimental or investigational drug therapy in the past 28 days -

Last updated 2022-12-17