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Phase 2 N=51 Randomized Single-blind Treatment

Hypertension Intervention to Reduce Osteonecrosis in Children With Acute Lymphoblastic Leukemia/Lymphoma

Hypertension · Osteonecrosis · Osteonecrosis Due to Drug

Enrolled (actual)
51
Serious AEs
88.0%
Results posted
Jul 2024
Primary outcome: Primary: Extensive Radiographic Osteonecrosis — 20; 21; 1; 2 Participants — p=0.7139

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Intensive Antihypertensive Therapy (Drug); Conventional Antihypertensive Therapy (Drug); Symptom Survey (Other); Semi-structured interview (Other)
Age
Pediatric, Adult, Older Adult · 10+ yrs
Sex
All
Sponsor
St. Jude Children's Research Hospital
Primary completion
Sep 2023

Outcome Measures

OutcomeResultp-value
PRIMARY
Extensive Radiographic Osteonecrosis
20; 21; 1; 2 0.7139
SECONDARY
Rate of Clinically Significant Osteonecrosis
SECONDARY
Rate of Clinically Significant Osteonecrosis vs. Historical Control
SECONDARY
Blood Pressure Control on Trial
114.00; 117.00; 70.00; 74.00
SECONDARY
Biomarkers of Vascular Dysfunction - eNO Synthetase (pg/mL)
187.78; 267.03; 193.28; 266.53
SECONDARY
Biomarker of Vascular Dysfunction - Von Willebrand Factor (%)
195.00; 191.00; 211.00; 164.00
SECONDARY
Biomarker of Vascular Dysfunction - TNF-alpha (pg/mL)
0.87; 0.79; 1.56; 1.44
SECONDARY
Biomarker of Vascular Dysfunction - D-dimer (µg/mL)
0.85; 0.60; 0.44; 0.29
SECONDARY
Biomarker of Vascular Dysfunction - PAI-1 (AU/mL)
17.85; 15.80; 38.00; 41.10
SECONDARY
Biomarker of Vascular Dysfunction - E-selectin (ng/mL)
23.53; 23.24; 65.16; 53.68
SECONDARY
Biomarker of Vascular Dysfunction - ICAM-1 (ng/mL)
328.79; 314.22; 629.13; 639.25
SECONDARY
Biomarker of Vascular Dysfunction - Arterial Elasticity (ml/mmHg)
11.60; 10.70; 12.30; 10.40
SECONDARY
Biomarker of Vascular Dysfunction - Pulse Wave Velocity (m/Sec)
5.00; 5.35; 5.00; 5.30
SECONDARY
Magnetic Resonance Imaging (MRI) of Right and Left Hip and Knee
1; 2; 1; 2; 1; 1

Summary

This is a randomized unblinded Phase II clinical trial evaluating the impact of intensive antihypertensive control (targeted to the 50-75th percentile for age, sex, and height) compared to conventional antihypertensive control (targeted to the 90-95th percentile for age, sex, and height) on the incidence of radiographically extensive osteonecrosis in children and young adults receiving treatment for newly diagnosed acute lymphoblastic leukemia/lymphoma (ALL). Primary Objective * Compare the frequency of radiographically extensive osteonecrosis in patients receiving intensive compared to conventional antihypertensive therapy. Secondary Objectives * Evaluate the efficacy of intensive antihypertensive control compared to conventional antihypertensive control in the prevention of clinically significant (CTCAE Grade 2 or higher) and radiologically extensive osteonecrosis, overall and stratified by joints. * Compare the frequency of clinically significant and radiographically extensive osteonecrosis in patients receiving antihypertensive therapy and historical controls. * Compare blood pressures achieved in intensive and conventional arms using both pressures obtained as part of routine patient care and ambulatory blood pressure monitoring. * Compare levels of vascular dysfunction as measured physiologically, radiographically, and in blood samples in patients receiving intensive compared to standard antihypertensive therapy. Exploratory Objectives * Identify predictive patterns of blood biomarkers which identify patients at high- risk of developing clinically significant osteonecrosis. * Identify MRI findings during late induction which correlate with osteonecrosis lesions seen during reinduction. * Identify patterns of diurnal blood pressure variation as measured by ambulatory blood pressure monitoring associated with the later development of osteonecrosis. * Compare induction blood pressure control and intervention arm to echocardiographic changes at reinduction II. * Evaluate patient-reported, health-related quality of life in patients during induction and after 1.5 years of therapy when many experience the symptoms of osteonecrosis.

Eligibility Criteria

Inclusion Criteria

  • Patient is being treated for newly diagnosed acute lymphoblastic leukemia or lymphoma (ALL) on the TOT17 protocol. Patients do not need to be hypertensive to enroll.
  • Patient is 10 years of age or older at the time of enrollment on TOT17.
  • Patient has completed ≤ 4 days of protocol therapy (patients are eligible on Day 4 of TOT17 therapy).

Exclusion Criteria

  • Moderate-severe renal dysfunction (glomerular filtration rate <45 ml/min/1.73m2).
  • Down's syndrome (germline Trisomy 21) or other syndrome resulting in growth delay or alterations in stature.
  • Chronic inability to ambulate. Patients with limitations in movement due to acute complications of leukemia/lymphoma are not excluded.
  • Permanent contraindication to MRI evaluation.
  • Participants who are pregnant or lactating. Males or females of reproductive potential must agree to use effective contraception for the duration of study participation.
  • Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04401267). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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