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| ID | Type | Description | Link |
|---|---|---|---|
| 5K23HL148310 | U.S. NIH Grant/Contract | View source | |
| 2020095 | Other Grant/Funding Number | Doris Duke Charitable Foundation |
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| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
| Doris Duke Charitable Foundation | OTHER |
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A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
The investigators hypothesize that adults with sickle cell disease (SCD) and osteonecrosis of the femoral (hip) and/or humeral (shoulder) heads will tolerate oral alendronate 70 mg administered once a week x 24 weeks (6 months). In addition to collecting safety and tolerability data on alendronate in study participants, the investigators will also measure the preliminary efficacy of alendronate using changes in the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact scores from baseline to 3-months and 6-months after alendronate initiation. Serum and urine specimen will also be collected to assess changes in bone biomarkers at baseline, 3-months, and 6-months.
The investigators plan to recruit 24 adults with SCD from the University of California Davis Medical Center to this single-arm, open label, interventional study. The investigators anticipate enrolling 1-2 participants per month between Sep 2024- Dec 2025.
The study endpoints are summarized below:
The investigators' goal is to complete primary data analysis by May 2026.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Alendronate group | Experimental | Single-arm prospective cohort of 30 adult with SCD |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alendronate Sodium | Drug | Administer oral alendronate 70 mg once a week x 24 weeks to all study participants |
|
| Measure | Description | Time Frame |
|---|---|---|
| Recruitment and retention rates | Proportion of eligible patients who get recruited to the study; number of enrolled patients who complete 6-month study | up to 28 weeks |
| Incidence of Treatment-Emergent Adverse Events as assessed by participant report | Collect all adverse events reported by patients and determine if related to study drug | up to 28 weeks |
| Pain assessed by the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire | The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire is a patient-reported outcome measure of pain level in the past 7 days. The ASCQ-Me pain scale ranges from 0-100, with a standardized mean of 50 and a standard deviation of 10, where lower scores signify worse disease impact. | up to 28 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Serum CTX-1 | Measurement of C-terminal telopeptide of type I collagen (CTX) in serum as a biomarker for osteoclast activity (bone resorption) | baseline, 3 months, 6 months |
| Serum P1NP | Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum as a biomarker for osteoblast activity (bone formation) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Leyla Y Teos, PhD | Contact | (916) 460-2749 | lyteos@ucdavis.edu |
| Name | Affiliation | Role |
|---|---|---|
| Oyebimpe O Adesina, MD, MS | UC Davis School of Medicine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis Comprehensive Cancer Center | Recruiting | Sacramento | California | 95817 | United States |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D010020 | Osteonecrosis |
| D007873 | Legg-Calve-Perthes Disease |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D019386 | Alendronate |
| ID | Term |
|---|---|
| D004164 | Diphosphonates |
| D063065 | Organophosphonates |
| D009943 | Organophosphorus Compounds |
| D009930 | Organic Chemicals |
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Prospective cohort of 30 adults with sickle cell disease and hip osteonecrosis
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| baseline, 3 months, 6 months |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D009336 | Necrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D005271 | Femur Head Necrosis |