AB1 for Sickle Cell Disease

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Sickle Cell DiseaseAB1 - Drug
Eligibility
18 - 45
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new drug by giving increasing doses to groups of patients to see what effects it has. The drug is taken orally once a day for 8 weeks.

Eligible Conditions
  • Sickle Cell Disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

2 Primary · 11 Secondary · Reporting Duration: From the time of consent up to 12 months

Baseline, week4, week8, week10
Change in AUC o-t as measured by blood test
Change in CL as measured by blood test
Change in Cmax as measured by blood test
Change in Vss as measured by blood test
Change in Vz as measured by blood test
Change in dose normalized AUC o-inf as measured by blood test
Change in t1/2 as measured by blood test
Change in tmax as measured by blood test
Month 12
Number of adverse events/serious adverse events as measured by patient report/medical records
Number of ≥Grade 2 study related adverse events as measured by patient report/medical record
Screening, baseline, week2,week4, week6, week8, week10, week12
Change in percent reticulocytes as measured by blood tests
Change in percentage of F-cells measured by flow cytometry
Change in percentage of total percentage of total hemoglobin (HB) as measured by HPLC

Trial Safety

Safety Progress

1 of 3

Trial Design

1 Treatment Group

AB1
1 of 1

Experimental Treatment

30 Total Participants · 1 Treatment Group

Primary Treatment: AB1 · No Placebo Group · Phase 1 & 2

AB1
Drug
Experimental Group · 1 Intervention: AB1 · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: from the time of consent up to 12 months

Who is running the clinical trial?

Nirmish ShahLead Sponsor
1 Previous Clinical Trials
16 Total Patients Enrolled
Nirmish Shah, MDPrincipal InvestigatorDuke University
5 Previous Clinical Trials
137 Total Patients Enrolled

Eligibility Criteria

Age 18 - 45 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a HbF level of less than 8.6% of total Hb at screening.
You have a confirmed diagnosis of SCD.
The sickle crisis rate is between 2 and 10 in the past year with no crisis in the last 28 days.