Baseline characteristics of GUARDIAN study (NCT06713135) cohort

Topic:

Clinical Trials

Poster Number: 66 S

Author(s):

Nicolas Deconinck, Neuromuscular Reference Center, Zoya Alhaswani, Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK, Giovanni Baranello, MD, PhD, UCL Gt Ormond St Inst Child Health, London, UK, Ekaterina Gresko, physician-biochemist, PhD, Santhera Paramaceuticals, Saskia Houwen, Radboud University Medical Center, Amalia Children’s Hospital, Nijmegen, The Netherlands, Lenka Juříková, University Hospital Brno, Brno, Czechia, Aki Linden, MSc, Santhera Pharmaceuticals Schweiz AG, Raoul Rooman, MD, Santhera Pharmaceuticals Schweiz AG, Leanne Ward, MD FRCPC, University of Ottawa, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada

Background: Vamorolone is a dissociative corticosteroid, approved for DMD treatment, with comparable short-term efficacy to prednisone and a more favorable safety profile.

Objectives: Describe baseline (BL) characteristics of patients enrolled in long-term GUARDIAN study.

Methods: Forty-three patients who previously completed VISION-DMD (NCT03439670) or VBP15-002/003/LTE (NCT02760264/NCT02760277/NCT03038399) studies and were treated with vamorolone in various access programs according to routine clinical practice at sites in the EU, UK, New Zealand and Israel were enrolled in GUARDIAN. The study was designed to evaluate long-term effectiveness and safety of vamorolone for DMD. This analysis includes BL data from the first 40 patients enrolled as of August 15, 2025.

Results: At enrolment, mean age ±SD was 11±1.34 years, ages at first symptoms and at vamorolone initiation were 2.5±1.63 and 5.8±0.86 years, respectively. The mean (Min, Max) duration of vamorolone treatment at inclusion was 5.2 (4.0, 8.1) years, the mean ±SD daily dose 4.7±0.92 mg/kg/day. Mean ±SD height and BMI z-scores were -0.6±1.1 and 1.3±1.12. Among 17/40 patients with available data, median TTSTAND (Q1; Q3) was 7.3 s (5.1; 9.0), and 31/40 (77.5%) were ambulatory. Three of 37 (8.1%) patients with available spine X-rays had centrally confirmed vertebral fractures (VF), while 11/40 (27.5%) reported non-VF. Three of 39 (7.7%) individuals had cataracts (one pre-vamorolone), 5/40 (12.5%) had cardiac conditions, and 20/40 (50%) received cardiac medication. All patients ≥14 years old (N=3) reached Tanner stage ≥G2 (1 after pubertal induction with testosterone therapy). Glucose and lipid parameters were within normal ranges in the majority of patients. Morning cortisol levels were indicative of adrenal suppression; no symptomatic adrenal insufficiency was reported.

Conclusions: At GUARDIAN BL (after 4-8 years of vamorolone), patients showed normal growth, low VF and cataract prevalence, and non-VF prevalence in accordance with published literature. Non-ambulatory rates were comparable to other corticosteroids. Future GUARDIAN data will extend long-term effectiveness and safety evidence.