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

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ClinicalTrials.gov Identifier: NCT01894594
Recruitment Status : Recruiting
First Posted : July 10, 2013
Last Update Posted : February 6, 2018
Sponsor:
Information provided by (Responsible Party):
Jane Little, University Hospitals Cleveland Medical Center

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Chronic Kidney Disease Metabolic Acidosis Drug: Sodium Bicarbonate Phase 1

Detailed Description:

Primary Objective:

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

Secondary Objectives:

To assess the effect of alkali administration on improvement in hemolysis and on sequelae of impaired kidney function, ie, LDH, Hgb, reticulocyte count, red cell half-life, and muscle strength, Vitamin D levels, and markers of bone turnover, respectively.

To assess the influence of alkali administrations on markers of kidney tubule inflammation.

To evaluate intraparenchymal iron in patients with SCD and renal dysfunction. Safety and adverse events of alkali patients with sickle cell disease will be monitored. This research will supplement current knowledge about management of the clinically important subset of people with SCD who have renal insufficiency and acid-base perturbation.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Alkali Therapy in Subjects With Sickle Cell Disease (SCD) - Evaluation of Efficacy, Safety, and Beneficial Effects
Study Start Date : June 2013
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Sodium Bicarbonate
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

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)

Other Name: Alkali Therapy




Primary Outcome Measures :
  1. Serum bicarbonate level [ Time Frame: 12 weeks ]
    To assess the effect of alkali administration on bicarbonate and potassium levels in patients with SCD and depressed serum bicarbonate levels.


Secondary Outcome Measures :
  1. Hemolysis markers [ Time Frame: 12 weeks ]
    To assess the effect of alkali administration on improvement in hemolysis and on sequelae of impaired kidney function, ie, LDH, Hgb, reticulocyte count, red cell half-life, and muscle strength, Vitamin D levels, and markers of bone turnover, respectively.


Other Outcome Measures:
  1. Tubular effect [ Time Frame: 12 weeks ]
    To assess the influence of alkali administrations on markers of kidney tubule inflammation.



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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01894594


Contacts
Contact: Jane Little, MD 216-844-5868

Locations
United States, Ohio
University hospitals Case Medical Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: Jane Little, MD    216-844-5868      
Principal Investigator: Jane Little, MD         
Sponsors and Collaborators
University Hospitals Cleveland Medical Center
Investigators
Principal Investigator: Jane Little, MD University Hospitals Cleveland Medical Center

Responsible Party: Jane Little, Director, Sickle Cell Disease Program, University Hospitals Cleveland Medical Center
ClinicalTrials.gov Identifier: NCT01894594     History of Changes
Other Study ID Numbers: 09-12-13
First Posted: July 10, 2013    Key Record Dates
Last Update Posted: February 6, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Jane Little, University Hospitals Cleveland Medical Center:
Sickle cell disease
Chronic kidney disease
Bicarbonate therapy

Additional relevant MeSH terms:
Kidney Diseases
Renal Insufficiency, Chronic
Anemia, Sickle Cell
Acidosis
Urologic Diseases
Renal Insufficiency
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Acid-Base Imbalance
Metabolic Diseases