Status:
TERMINATED
AB1 in Adult Patients with Sickle Cell Disease (SCD)
Lead Sponsor:
Nirmish Shah
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
18-45 years
Phase:
PHASE1
PHASE2
Brief Summary
This will be an open-label, dose escalating study with a starting dose of 2mg. Up to 6 additional cohorts will be enrolled at subsequently higher doses of 4mg, 8mg, 10mg, 12mg, 16mg, and 32mg. In each...
Detailed Description
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. * Cohort 1 - Two (...
Eligibility Criteria
Inclusion
- 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 -
Exclusion
- 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 -
Key Trial Info
Start Date :
December 5 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2024
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT05261711
Start Date
December 5 2022
End Date
October 1 2024
Last Update
December 4 2024
Active Locations (3)
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1
Augusta University Medical Center
Augusta, Georgia, United States, 30912
2
Duke University Medical Center
Durham, North Carolina, United States, 27710
3
East Carolina University
Greenville, North Carolina, United States, 27834