Efficacy, Safety Study and Benefit of Alkali Therapy in Sickle Cell Disease

NCT01894594 | Terminated Phase 1

• 1 other identifier: 09-12-13
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this study is to assess the effect of alkali administration on bicarbonate and potassium levels in patients with Sickle Cell Disease (SCD) and depressed serum bicarbonate levels. The study is a prospective non-blinded evaluation of tolerability and efficacy of alkali repletion with 4 weeks of observation and two sequential 4 week courses of escalating oral sodium bicarbonate treatment.

Conditions

Sickle Cell Anemia; Chronic Kidney Disease; Metabolic Acidosis

Keywords

Sickle cell disease; Chronic kidney disease; Bicarbonate therapy

Outcome Measures

Primary Outcomes (1)

• Serum bicarbonate level

  To assess the effect of alkali administration on bicarbonate and potassium levels in patients with SCD and depressed serum bicarbonate levels.

  12 weeks

Secondary Outcomes (1)

• Hemolysis markers

  12 weeks

Other Outcomes (1)

• Tubular effect

  12 weeks

Study Arms (1)

Sodium Bicarbonate

EXPERIMENTAL

Patients will be monitored at baseline bi-weekly intervals for 12 weeks, the first 4 weeks to establish a stable baseline, followed by 8 weeks of alkali therapy, as follows:

Drug: Sodium Bicarbonate

Interventions

Sodium Bicarbonate DRUG

Oral sodium bicarbonate tablets administered at a starting dose of 0.6 mEQ/Kg body weight and will be escalated once, at 4 weeks, to 0.9 mEQ/Kg body weight. Weekly schedule: 0-4 weeks: Serial Measurement at baseline, without therapy 4-8 weeks: \*\~0.6 mEq/kg of ideal body weight of Sodium Bicarbonate tablets divided into TID dosing 8-12 weeks: \*\~0.9 mEq/kg of ideal body weight of Sodium Bicarbonate tablets divided into TID dosing \* to the closest dose of 650 mg (7.74 mEq) Ideal Body Weight is defined by the following formulas (Devine Calculation): Ideal Body Weight (men) = 50 + 2.3 (Height (in) - 60) Ideal Body Weight (women) = 45.5 + 2.3 ( Height (in) - 60)

Also known as: Alkali Therapy

Sodium Bicarbonate

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Sickle cell disease patients with HbSS
• eGFR \<90 ml/min/1.73m2 (determined by abbreviated 4 variable modification MDRD equation) and/or measured urinary albumin to creatinine ratio \>30mg/g.
• Age ≥18 years

You may not qualify if:

• Previous chronic treatment with alkali (including sodium bicarbonate, calcium carbonate or baking soda)
• Bicarbonate level \>25 mEq/L
• Decompensated heart failure
• Uncontrolled systolic blood pressure \>140 mm/Hg (the cutoff for systolic hypertension in SCD is lower than in non-SCD)
• Moderate-to-severe lower extremity edema
• Projected progression to ESRD within 6 months
• Kidney transplantation
• Treatment with immunosuppressives within the last 3 months
• Vasoocclusive (VOC) within 1 week of study entry
• Active (open) leg ulcer
• Change in hydroxyurea dose within the last 3 months, unless a self-limited interruption of a stable dose
• Blood transfusion within 8 weeks, unless on chronic transfusions
• Pregnancy
• Inability to give informed consent

Sponsors & Collaborators

• University Hospitals Cleveland Medical Center: lead

Study Sites (1)

University hospitals Case Medical Center

Cleveland, Ohio, 44106, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Renal Insufficiency, Chronic; Acidosis

Interventions

Sodium Bicarbonate

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Acid-Base Imbalance; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Bicarbonates; Carbonates; Carbonic Acid; Carbon Compounds, Inorganic; Inorganic Chemicals; Sodium Compounds

Study Officials

• Jane Little, MD

  University Hospitals Cleveland Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Sickle Cell Disease Program

Study Record Dates

• First Submitted: July 3, 2013
• First Posted: July 10, 2013
• Study Start: June 1, 2013
• Primary Completion: March 1, 2019
• Study Completion: March 1, 2019
• Last Updated: March 20, 2019

Record last verified: 2018-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)