NCT00001197

Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia

Completed Phase 2 Last updated 16 December 2019

What this trial tests

Phase 2 trial testing Hydroxyurea in Sickle Cell Anemia in 41 participants. Completed in 18 May 2015.

Timeline

7 February 1984

Primary endpoint
21 December 2003

18 May 2015

Quick facts

Lead sponsor	National Heart, Lung, and Blood Institute (NHLBI)
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Primary purpose	treatment
Enrollment	41
Start date	7 February 1984
Primary completion	21 December 2003
Estimated completion	18 May 2015
Sites	1 location across United States

Drugs / interventions tested

• Hydroxyurea — full drug profile →

Conditions studied

• Sickle Cell Anemia — all drugs for Sickle Cell Anemia →

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

18 and older, any sex, with Sickle Cell Anemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

A total of fifty severely affected patients with homozygous sickle cell disease or other sickling disorders (e.g. B negative or B positive Thalassemia/Sickle) who are greater than 18 years of age will be eligible for treatment. Such patients must be able to tolerate an extensive period without blood transfusion and have relatively well preserved renal and hepatic function (creatinine less than 1.5 mg/dl and normal liver function test with exception of a mild elevation in transaminase). Evidence of severe sickle cell anemia will include recurrent pain crisis, chronic bone oain, evidence of aseptic necrosis with symptoms, and intractable leg ulcer, etc.

On admission to the study, each patient will receive a complete history and physical examination. These data and standard laboratory evaluation, including a test for pregnancy if appropriate, will be adequate to ascertain whether any of the criteria for exclusion are present. Each patient must accept responsibility for for using an effective means of contraception. Patients who are found to be HIV positive will be excluded from the study....

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Hydroxyurea (hydroxycarbamide) for sickle cell disease.
   Rankine-Mullings AE, Nevitt SJ. · · 2022 · cited 42× · PMID 36047926 · DOI 10.1002/14651858.cd002202.pub3
2. Pharmacotherapy in sickle cell disease--state of the art and future prospects.
   Hankins J, Aygun B. · · 2009 · cited 18× · PMID 19222472 · DOI 10.1111/j.1365-2141.2009.07602.x