Alendronate for Osteonecrosis in Adults With Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:

A Feasibility Study of Alendronate as Treatment for Osteonecrosis in Adults With Sickle Cell Disease

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06016634
• Other study ID #: 1693339
• Secondary ID: 5K23HL1483102020
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: March 2024
• Est. completion date: July 2025

Study information

• Verified date: September 2023
• Source: University of California, Davis
• Contact: Leyla Y Teos, PhD
• Phone: (916) 460-2749
• Email: lyteos[@]ucdavis.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis

Description:

The investigators hypothesize that adults with sickle cell disease (SCD) and osteonecrosis in the hip or shoulder joint(s) will tolerate oral alendronate 70 mg administered once a week for 24 weeks (6 months). In addition to collecting safety and tolerability data of alendronate in study participants, the investigators will measure how alendronate therapy impacts quality of life measures using the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) questionnaires from baseline to 3-months and 6-months in study participants. 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 Mar 2024- Feb 2025. The study endpoints are summarized below: - To determine the recruitment and retention rates of adults with SCD-related ONFH enrolled in this prospective, single-arm, open label alendronate interventional study - To measure the safety, tolerability, and preliminary efficacy of oral alendronate in adults with SCD-related ONFH over a 6-month treatment duration - To measure changes in bone biomarkers in the serum and urine of study participants not receiving chronic red blood cell transfusions at 3 time points: baseline, 3-months, and 6-months after initiation of alendronate The investigators' goal is to complete primary data analysis by July 2025.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 24
• Est. completion date: July 2025
• Est. primary completion date: February 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

Inclusion Criteria:

• Age 18-80 years with SCD (any genotype, confirmed by hemoglobin electrophoresis or high performance liquid chromatography)
• Ability to provide written informed consent
• Ability to lay on a dual-energy X-ray absorptiometry (DXA) scanner
• Negative urine pregnancy test for anyone of childbearing potential at study entry

Exclusion Criteria:

• Pregnant women
• Adults unable to consent
• Individuals who are not yet adults (infants, children, teenagers)
• Prisoners
• Hospitalizations (for any cause) within 2 weeks of study entry

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Avascular Necrosis
• Necrosis
• Osteonecrosis
• Sickle Cell Anemia
• Sickle Cell Disease

Intervention

Drug:
• Alendronate Sodium
Administer oral alendronate 70 mg once a week x 24 weeks to all study participants

Locations

Country	Name	City	State
n/a

Sponsors (3)

Lead Sponsor	Collaborator
University of California, Davis	Doris Duke Charitable Foundation, National Heart, Lung, and Blood Institute (NHLBI)

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Secondary	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
Secondary	Serum P1NP	Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum as a biomarker for osteoblast activity (bone formation)	baseline, 3 months, 6 months