Phase 1 Study of Zoledronic Acid in Sickle Cell Disease

Study Overview
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Brief Summary
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The long-term goal of this study is to learn if Zoledronic Acid can prevent or reduce pain in sickle cell disease. The goal of this study is to learn about the safety of Zoledronic Acid in persons with sickle cell disease who experience chronic pain requiring medical treatment or use of narcotics.

Official Title
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Phase 1 Study of Zoledronic Acid in Sickle Cell Disease

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: Placebo
* Drug: Zoledronic Acid


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 years and older Male or female with sickle cell disease Pain related to sickle cell disease Patient history of health services utilization for acute SCD-related pain Patient history of use of narcotic analgesics for pain control within the past 6 months Able to tolerate hydration with 500 mL D51/2 NS prior to Zoledronic Acid or placebo Exclusion Criteria: Calculated creatinine clearance less than 60 mL/min Current active dental problems Recent (within 6 weeks) or planned dental or jaw surgery (e.g., extraction,implants) History of cirrhosis or chronic symptomatic liver disease; acute liver disease History of aspirin-induced asthma History of allergy to zoledronic acid or similar chemical-entities Pregnant or nursing No prior bisphosphonate use Receipt of an investigational drug within 30 days

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: 2<br>Patients will receive a single dose of Zoledronic Acid or placebo. The chances that a subject will receive placebo are 1 out of 3. | Drug: Placebo<br>* Patients will receive a single dose of placebo administered by vein over about 15 minutes. Placebo is mostly water without Zoledronic Acid.<br>|
| Experimental: 1<br>Patients will receive a single dose of Zoledronic Acid or placebo. The chances that a subject will receive Zolendronic Acid are 2 out of 3. | Drug: Zoledronic Acid<br>* Patients will receive a single dose of Zolendronic Acid. Zoledronic Acid is administered by vein over about 15 minutes.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The long-term goal of this project is to learn if Zoledronic Acid can prevent or reduce pain in sickle cell disease. |  | Within 2 weeks of study drug administration and then every month for approximately 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The specific purposes of this study are to learn if Zoledronic Acid has serious side effects for subjects with sickle cell disease and to study the effects of Zoledronic acid in subjects with sickle cell disease. |  | Two weeks after study drug administration and then every month for approximately 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Sickle cell disease