Alendronate for Hip Osteonecrosis in Adults With Sickle Cell Disease

Sponsor

University of California, Davis (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT06016634

Collaborator

National Heart, Lung, and Blood Institute (NHLBI) (NIH), Doris Duke Charitable Foundation (Other)

Enrollment

Arm

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease Sickle Cell Anemia Osteonecrosis Ischemic Necrosis Avascular Necrosis	Drug: Alendronate Sodium	Phase 2

Detailed Description

The investigators hypothesize that adults with sickle cell disease (SCD) and osteonecrosis of the femoral head (ONFH) will tolerate oral alendronate 70 mg administered once a week for 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 2023- Dec 2024.

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 May 2025.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

24 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

Prospective cohort of 24 adults with sickle cell disease and hip osteonecrosisProspective cohort of 24 adults with sickle cell disease and hip osteonecrosis

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Oct 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2024

Anticipated Study Completion Date :

Jun 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Alendronate group Single-arm prospective cohort of 24 adult with SCD	Drug: Alendronate Sodium Administer oral alendronate 70 mg once a week x 24 weeks to all study participants Other Names: alendronate tablet oral alendronate Fosamax Binosto

Arm

Intervention/Treatment

Experimental: Alendronate group

Single-arm prospective cohort of 24 adult with SCD

Drug: Alendronate Sodium

Administer oral alendronate 70 mg once a week x 24 weeks to all study participants

Other Names:

alendronate tablet

oral alendronate

Fosamax

Binosto

Outcome Measures

Primary Outcome Measures

Recruitment and retention rates [up to 28 weeks]
Proportion of eligible patients who get recruited to the study; number of enrolled patients who complete 6-month study
Incidence of Treatment-Emergent Adverse Events as assessed by participant report [up to 28 weeks]
Collect all adverse events reported by patients and determine if related to study drug
Pain assessed by the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire [up to 28 weeks]
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.

Secondary Outcome Measures

Serum CTX-1 [baseline, 3 months, 6 months]
Measurement of C-terminal telopeptide of type I collagen (CTX) in serum as a biomarker for osteoclast activity (bone resorption)
Serum P1NP [baseline, 3 months, 6 months]
Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum as a biomarker for osteoblast activity (bone formation)

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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