An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Santhera Pharmaceuticals (Schweiz) AG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

5 Dec 2024 → ongoing

Decision date (initial)

2024-10-04

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Santhera Pharmaceuticals (Switzerland) Ltd.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the safety of long-term treatment with vamorolone in boys with DMD regarding vertebral fractures

Secondary objectives 3

1. To evaluate the safety of long-term treatment with vamorolone in boys with DMD on non-vertebral fractures, cataracts, and delayed puberty.
2. To evaluate the overall safety of long-term treatment with vamorolone in boys with DMD
3. To evaluate the long-term treatment effect of vamorolone on ambulatory and non-ambulatory function

Conditions and MedDRA coding

Duchenne Muscular Dystrophy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Subject and/or subject’s parent(s) or legal guardian has provided written informed consent
2. Subject has previously completed either the VBP15-LTE or VBP15-004 study, and transitioned through the CUP, NPP or EAP
3. Subject is on vamorolone on the day of enrolment
4. Subject and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements

Exclusion criteria 3

1. Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments
2. Vamorolone treatment discontinued for ≥6 months within the year prior to enrolment for a non-safety reason, or vamorolone treatment previously discontinued at any time for a safety reason
3. Severe hepatic impairment

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of vertebral fractures per 1000 person-years based on X- ray central reading

Secondary endpoints 13

1. Time to first vertebral fractures (cumulative incidence)
2. Number of non-vertebral fractures per 1000 person-years based on investigator reporting
3. Time to first non-vertebral fractures (cumulative incidence)
4. Number of cataracts per 1000 person-years based on ophthalmologist assessment
5. Number of subjects not reaching Tanner stage 2 by 15 years of age
6. Frequency of adverse events (AEs) and serious adverse events (SAEs)
7. Change from baseline in body weight, height and body mass index (BMI)
8. Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol
9. Change from baseline in Time to Stand Test (TTSTAND) velocity
10. 6-Minute Walk Test (6MWT) distance
11. Change from baseline in 6MWT distance
12. NorthStar Ambulatory Assessment (NSAA) scores
13. Age at ambulatory and non-ambulatory milestones

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Santhera Pharmaceuticals (Schweiz) AG

Sponsor organisation

Santhera Pharmaceuticals (Schweiz) AG

Address

Hohenrainstrasse 24

City

Pratteln

Postcode

4133

Country

Switzerland

Scientific contact point

Organisation

Santhera Pharmaceuticals (Schweiz) AG

Contact name

Santhera General Inquiries

Public contact point

Organisation

Santhera Pharmaceuticals (Schweiz) AG

Contact name

Santhera General Inquiries

Locations

6 EU/EEA countries · 11 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 96 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

14 submissions — initial application plus substantial / non-substantial modifications