Status:
COMPLETED
A Pilot Study of N-acetylcysteine in Patients With Sickle Cell Disease
Lead Sponsor:
Bloodworks
Collaborating Sponsors:
University of Washington
Conditions:
Sickle Cell Disease
Sickle Cell Anemia
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
Part 1: A pilot study in patients with homozygous S (HbSS) or hemoglobin S with beta zero thalassemia(HbS-βo thalassemia), with the aim of examining the effect of intravenous NAC treatment on plasma V...
Detailed Description
Two primary processes dominate the complications associated with sickle cell disease (SCD): vasoocclusion and hemolysis. The plasma and vessel wall adhesive protein von Willebrand factor (VWF) is thou...
Eligibility Criteria
Inclusion
- Age \>= 18 years of age
- Diagnosis of homozygous sickle cell (SS) or S-beta thalassemia with at least two episodes of vaso-occlusive crises (VOC) requiring narcotics in each of the past 2 years. For part 2 can include hemoglobin SC disease.
- For females of reproductive age, use of contraception and negative pregnancy test
Exclusion
- An additional hematologic diagnosis
- Hemoglobin (Hgb) \< 7gm/dL for part 1, \< 6 gm/dL for part 2.
- Asthma requiring medication
- Liver function tests \[alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin (BilliT) \> three times upper normal limit for Part 1.
- Chronic transfusion therapy, or transfusion within 2 months of enrollment. For part 2 anticipated need for simple or exchange transfusion during hospitalization.
- VOC requiring narcotic therapy within the prior week or requiring hospitalization with discharge \< 2 weeks prior to study enrollment for Part 1, for part 2 admission for VOC within 30 days.
- Pregnancy or nursing
- Receiving another investigational drug
- Known allergy to NAC
- Per subject's physician not medically stable enough to participate
- Taking nitroglycerin, carbamazepine, or phosphodiesterase 5 (PDE5) inhibitors
- Abnormal baseline coagulation tests (\> 1.5 times normal limits)
- Platelets \<150,000/microliter for Part 1.
- For part 2, already enrolled in study twice.
Key Trial Info
Start Date :
March 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 30 2020
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT01800526
Start Date
March 1 2013
End Date
June 30 2020
Last Update
July 9 2020
Active Locations (1)
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1
University of Washington
Seattle, Washington, United States, 98106