Actual & potential clinical trials for non-transfusion therapies for hemolytic anemia

LewisHsu · #1 (**permalink**) 11-10-2008, 10:57 AM

Title:Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
Category: Potential Phase II clinical trial
Eligibility criteria:18 to 75 years of age; diagnosed with Thrombotic Microangiopathy based on presence of:
Thrombocytopenia, defined as a platelet count <100 x 109 per liter;
Microangiopathic hemolytic anemia, defined by negative findings on direct antiglobin test, and evidence of accelerated red blood cell (RBC) production and destruction); AND
Absence of a clinically apparent alternative explanation for thrombocytopenia and anemia, e.g., disseminated intravascular coagulation (DIC), eclampsia, HELLP syndrome, Evans syndrome;
Contact information:See ClinicalTrials.gov identifier: NCT00726544
Contact: James Gilbert, MD +1 617-621-7700 jgilbert@archemix.com
Date posted:
Description:The purpose of this ascending-dose research study is to determine whether the administration of ARC1779 Injection improves subject's health profile by protecting the brain, heart, and kidney from damage due to formation of small blood clots in blood vessels. It will also determine the safety of ARC1779 Injection, how ARC1779 Injection enters and leaves the blood and tissue over time, and its effect on laboratory tests related to blood clotting, heart and brain function, and other body systems.

Title :Evaluating the Safety and Effectiveness of Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease (CHAMPS)
Category:Clinical Trial
Eligibility criteria:Sickle cell Hemoglobin SC Disease
Contact information

see Home - ClinicalTrials.gov Identifier: NCT00532883)
Date posted:
Description:Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. Hemoglobin SCD (HbSC) is a form of SCD that is characterized by dense red blood cells. The purpose of this study is to evaluate the safety and effectiveness of hydroxyurea and magnesium pidolate, alone and combined, at reducing red blood cell density and the frequency of pain episodes in people with HbSC.
Study funding was cancelled by NIH for slow enrollment 10/27/08.

Title:Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises
Category:Phase II Clinical Trial
Eligibility criteria:Patients 10 years of age or older with sickle cell disease (known SS, S-beta-thalassemia or other blood problems causing sickle cell disease) may be eligible for this study. Patients whose disease is due to hemoglobin (Hgb) SC are excluded.
Contact information

see Home - ClinicalTrials.gov Identifier: NCT00094887)
Date posted:
Description:This study will examine whether nitric oxide (NO) gas can reduce the time it takes for pain to go away in patients who are in sickle cell crisis. NO is important in regulating blood vessel dilation, and consequently, blood flow. The gas is continuously produced by cells that line the blood vessels. It is also transported from the lungs by hemoglobin in red blood cells.

Participants are admitted to the hospital in a pain crisis. They are evaluated and then randomly assigned to receive one of two treatments: 1) standard treatment plus NO, or 2) standard treatment plus placebo. The placebo used in this study is nitrogen, a gas that makes up most of the air we breathe and is not known to help in sickle cell disease.

For the first 8 hours of the study, patients receive placebo or NO through a facemask. The mask may be taken off for 5 minutes every hour and for not more than 20 minutes to eat a meal. After the first 8 hours, the gas is delivered through a nasal cannula (small plastic tubing that rests under the nose) that may be taken off only while showering or using the restroom. Patients are questioned about the severity of their pain when they start the study and then every few hours while they are in the hospital. Their vital signs (temperature, breathing rate, and blood pressure) and medicines are checked. Patients will breathe the gas for a maximum of 3 days, but will stay hospitalized until the patient feels well enough to go home. Patients are followed up about 1 month after starting the study by a return visit to the hospital or by a phone call.

Title:Aspirin Prophylaxis in Sickle Cell Disease
Category:Phase I/II Clinical Trial
Eligibility criteria:Children ages 2 - 7.99 years with a diagnosis of Hb SS or Hb Sß0 thalassemia
Contact information

see Home - ClinicalTrials.gov Identifier: NCT00178464)
University of Rochester Medical Center Recruiting
Rochester, New York, United States, 14642
Contact: Norma B Lerner, MD, MPH 585-275-2981 Norma_Lerner@urmc.rochester.edu
Date posted:
Description:Neurologic complications secondary to cerebrovascular damage are prevalent in children with sickle cell disease. These patients experience both clinically overt cerebrovascular accidents and "silent infarctions" demonstrated by magnetic resonance imaging (MRI). They are also at risk for neurocognitive abnormalities.We hypothesize that daily, low-dose aspirin therapy will safely diminish the incidence and progression of cognitive deficits as well as the predisposition to overt and silent stroke in children with homozygous sickle cell disease (Hgb SS) or hemoglobin S Beta Zero Thalassemia (Hgb SB-0 Thal). In order to optimize the design of a future trial to test this hypothesis, we propose a pilot study to test the safety and tolerability of aspirin in young children with sickle cell. disease.

Title:Niacin to Improve Blood Flow in People With Sickle Cell Disease
Category:Phase II Clinical Trial
Eligibility criteria:Patients with sickle cell disease, between 18 and 65 years of age may be eligible for this study.
Contact information

see Home - ClinicalTrials.gov Identifier: NCT00508989)
Date posted:
Description:This study will determine whether niacin can improve blood flow in people with sickle cell disease, in which abnormal red blood cells interfere with blood flow to cause the disease symptoms. Niacin, a drug that has been used to increase HDL (good cholesterol) levels, improves blood flow in people without sickle cell disease. This study will see if it can do the same in people with the disease.

Title:Sildenafil Therapy for Pulmonary Hypertension and Sickle Cell Disease
Category:Phase II Clinical Trial
Eligibility criteria:Patients with sickle cell disease and pulmonary hypertension, 12 years of age and older.
Contact information

see Home - ClinicalTrials.gov Identifier: NCT00492531)
Date posted:
Description:This study will examine whether the drug sildenafil can lower blood pressure in the pulmonary artery (the blood vessel that leads from the heart to the lungs) in patients with sickle cell disease and pulmonary hypertension (high blood pressure in the lungs). It will see if this treatment can reduce disease complications, such as shortness of breath, pain crisis, pneumonia, and increase survival.

Patients 12 years of age and older with sickle cell disease and pulmonary hypertension may be eligible for this study. Participants are randomly assigned to receive sildenafil or placebo (sugar pill) for 16 weeks. Before starting treatment, patients have baseline studies, including a pregnancy test for females of childbearing age; a chest x-ray; pulmonary function tests to measure how much air the patient can breathe in and out; an echocardiogram (heart ultrasound); a 6-minute walk test to measure exercise capacity; a quality-of-life assessment and a pain inventory. Patients with moderate to severe pulmonary hypertension undergo heart catheterization to evaluate the severity of hypertension before beginning sildenafil therapy.

Title:Hypnosis to Manage Pain and Symptoms in Patients With Sickle Cell Disease
Category:Phase III Clinical Trial
Eligibility criteria:Sickle cell disease
Contact information

see Home - ClinicalTrials.gov Identifier: NCT00393250)
Date posted:
Description:This study will examine whether hypnosis can reduce the frequency and intensity of pain in patients with sickle cell disease. Patients 18 years of age and older with sickle cell disease and a history of pain associated with their disease may be eligible for this study.

Title:PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
Category:Potential Clinical Trial
Eligibility criteria:Sickle cell disease
Contact information

see Home - ClinicalTrials.gov Identifier: NCT00343954)
Date posted:
Description:To evaluate pharmacokinetics and pharmacodynamics responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.

Title: Tadalafil for Treatment of Priapism in Men With Sickle Cell Anemia
Category:Phase II Clinical Trial
Eligibility criteria:Men with sickle cell disease (18 years of age and older) with actively recurrent ischemic priapism
Contact information:see Home - ClinicalTrials.gov Identifier: NCT00538564
Contact Johns Hopkins Urology Institute: Irene N. Trueheart, BSN (443) 287-1011 itruehe1@jhmi.edu Arthur L Burnett, MD (410)955-1042 aburnett@jhmi.edu
Date posted:
Description:This research is being done to compare the effect of tadalafil with placebo (an inactive substance that looks like the study drug, but which should have no effect)on the frequency of recurrent priapism (prolonged erection, unassociated with sexual interest or desire) and the nature of sexual experiences in male patients with sickle cell disease. The study will also look at the effect of tadalafil on pulmonary hypertension (increased blood pressure in the blood vessels supplying the lungs).

Title:Stroke With Transfusions Changing to Hydroxyurea (SWiTCH)
Category:Phase III Clinical Trial
Eligibility criteria:Children aged 5-18 with stroke and s ickle cell disease
Contact information:see Home - ClinicalTrials.gov Identifier: NCT00122980
Date posted:
Description:This is a Phase III randomized clinical trial for children with SCA. The hypothesis is that hydroxyurea and phlebotomy can maintain an acceptable stroke recurrence rate and significantly reduce the hepatic iron burden. The primary aim is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload. Additional aims include comparisons of growth and development, frequency of non-stroke neurological and other sickle-related events, and quality of life. The use of hydroxyurea for secondary stroke prevention, coupled with removal of excess iron by phlebotomy, would represent a significant improvement in the management of individuals with SCA and stroke. If hydroxyurea is effective for the prevention of secondary stroke, it may also be beneficial for other children with SCA and cerebrovascular disease, including those at risk for primary stroke.

The trial includes approximately 130 children (5.0-18.9 years of age with 65 subjects per treatment arm) with SCA who have had symptomatic cerebral infarctions and have been treated with red cell transfusions for at least 18 months. After completing baseline screening studies, half the participants will be switched to a therapeutic program of hydroxyurea and phlebotomy. Half of the participants will remain on transfusion and chelation. The composite primary endpoint in this study is to compare two modalities of treatment for the prevention of secondary stroke and management of iron overload. The impetus for this trial is the fact that long-term transfusion and chelation therapy in children is difficult, is frequently unsuccessful, and is often complicated by severe symptomatic iron overload, particularly of the heart, lungs, and liver.

Thread Tools	Search this Thread
Show Printable Version Email this Page	Search this Thread: Advanced Search
Display Modes	Rate This Thread
Linear Mode Switch to Hybrid Mode Switch to Threaded Mode	Rate This Thread:

Similar Threads
Thread	Thread Starter	Forum	Replies	Last Post
sickle cell or other hemolytic anemia - consider looking for clinical trials	LewisHsu	Medical Articles and Abstracts	0	11-09-2008 11:00 PM
The Future of Clinical Trials Evaluating Blood Substitutes	hemostatguy	General Discussions	0	05-27-2008 10:23 PM
Oxygen carriers coursing through clinical trials	Jan B. Wade	News and Hot Topics such as Hepatitis C, SARS and AIDS	0	11-14-2005 10:01 PM
Phase III clinical trials with darbepoetin: implications for clinicians.	Nika	Medical Articles and Abstracts	0	04-01-2005 07:46 PM

Actual & potential clinical trials for non-transfusion therapies for hemolytic anemia

Welcome to the latest in bloodless medicine and blood transfusion alternatives.

We are a community of medical professionals and members of the public who are responding to the worldwide concern about the efficacy, cost and availability of donor blood.

The Following User Says Thank You to LewisHsu For This Useful Post:
Sharon Grant (11-19-2008)

Currently Active Users Viewing This Thread: 1 (0 members and 1 guests)