A Pilot Study of N-acetylcysteine in Patients With Sickle Cell Disease

• Status: Completed
• Trial end date: 2020-06-30
• Target enrollment: 0
• Participant gender: All

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 VWF parameters and measures of redox and RBC function.

Part 2: A pilot study in patients with sickle cell disease admitted to the hospital in vaso-occlusive crisis to determine the effects of NAC infusions on plasma VWF parameters and measures of redox and RBC function, and on measures of pain and hospital length of stay.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bloodworks; Bloodworks (Puget Sound Blood Center)
• Collaborator: University of Washington
• Treatments: Acetylcysteine; N-monoacetylcystine

Criteria

Inclusion Criteria:

1. Age >= 18 years of age

2. 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.

3. For females of reproductive age, use of contraception and negative pregnancy test

Exclusion Criteria:

1. An additional hematologic diagnosis

2. Hemoglobin (Hgb) < 7gm/dL for part 1, < 6 gm/dL for part 2.

3. Asthma requiring medication

4. Liver function tests [alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin (BilliT) > three times upper normal limit for Part 1.

5. Chronic transfusion therapy, or transfusion within 2 months of enrollment. For part 2 anticipated need for simple or exchange transfusion during hospitalization.

6. 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.

7. Pregnancy or nursing

8. Receiving another investigational drug

9. Known allergy to NAC

10. Per subject's physician not medically stable enough to participate

11. Taking nitroglycerin, carbamazepine, or phosphodiesterase 5 (PDE5) inhibitors

12. Abnormal baseline coagulation tests (> 1.5 times normal limits)

13. Platelets <150,000/microliter for Part 1.

14. For part 2, already enrolled in study twice.