Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis

• Status: Withdrawn
• Trial end date: 2015-10-01
• Target enrollment: 0
• Participant gender: All

Summary

Sickle Cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In Sickle Cell disease, as red blood cells circulate and oxygen is released, the deoxygenated abnormal Hemoglobin S can begin to polymerize and cause red cells to become sticky and elongated. These "sickled" red cells are less flexible and will obstruct small blood vessels and prevent normal red cells from circulating freely, which limits oxygen delivery to tissues and organs. This is known as a "sickling crisis" or "vaso-occlusive crisis" and is the leading cause of hospitalization in patients with Sickle Cell disease.

Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death. Current therapy is limited to hydration and symptomatic pain relief. The administration of MP4CO as an adjunct treatment to standard therapy may alleviate pain associated with a sickling crisis and potentially reduce the severity and duration of a crisis. This may shorten the time in hospital and potentially improve the quality of life for patients with sickle cell anemia.

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

Details

• Lead Sponsor: Sangart
• Treatments: Pharmaceutical Solutions

Criteria

Inclusion Criteria:

• Signed Informed Consent (and assent as required for minors)

• Diagnosis of SCD (known HbSS or HbSß0)

• Sixteen years of age or older

• Prior history of at least one VOC requiring hospitalization within the last 24 months

Exclusion Criteria:

• ≥ 5 VOCs within the preceding 6 months requiring Emergency Room (ER) visits or hospital admissions

• History of overt stroke or cerebral vascular accident within the previous 12 months

• Remained in the hospital for ≥2 weeks (14 days) for VOC management within the previous 6 months

• Known pulmonary hypertension based on an estimated tricuspid regurgitant jet velocity (TRJV) >2.90 m/s or definitive diagnosis by prior right heart catheterization

• Baseline SaO2 level by pulse oximetry <92% on room air

• Systemic hypertension (baseline systolic pressure ≥ 160 mmHg or diastolic pressure ≥ 90 mmHg)

• History of myocardial infarction, myocardial ischemia, or angina

• On a chronic red blood cell transfusion therapy program (simple or exchange)

• Renal dysfunction presenting with a GFR<60 mL/min/1.73m

• Any diagnosis of a concurrent chronic debilitating disease that may affect the completion of the study or results of the study as determined by the investigator

• Currently enrolled in any other investigational treatment study

• Significant substance abuse.

• Known to have HIV, active hepatitis B, or C infection, or active tuberculosis