Clinical trial NCT06016634: Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
Official title: A Feasibility Study of Alendronate as Treatment for Osteonecrosis in Adults With Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Sickle Cell Disease
Sickle Cell Anemia
Osteonecrosis
Ischemic Necrosis
Avascular Necrosis
Keywords: sickle cell disease
sickle cell anemia
osteonecrosis of the femoral head
hip osteonecrosis
ischemic necrosis of the femur
avascular necrosis of the femur
Lead sponsor: University of California, Davis
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
name: Doris Duke Charitable Foundation
First posted: 2023-08-30
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Prospective cohort of 24 adults with sickle cell disease and hip osteonecrosis
Primary purpose: TREATMENT

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

Detailed description: 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:

* To determine the recruitment and retention rates of adults with SCD and osteonecrosis 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 osteonecrosis 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 2026.

Eligibility criteria: 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

Primary outcomes:
measure: Recruitment and retention rates
timeFrame: up to 28 weeks
measure: Incidence of Treatment-Emergent Adverse Events as assessed by participant report
timeFrame: up to 28 weeks
measure: Pain assessed by the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire
timeFrame: up to 28 weeks
Secondary outcomes:
measure: Serum CTX-1
timeFrame: baseline, 3 months, 6 months
measure: Serum P1NP
timeFrame: baseline, 3 months, 6 months
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis Comprehensive Cancer Center
state: California
Study officials:
affiliation: UC Davis School of Medicine
name: Oyebimpe O Adesina, MD, MS
role: PRINCIPAL_INVESTIGATOR
References: