LUMINA-1 Phase II Clinical Trial Results Published in medRxiv

Regeneron Pharmaceuticals shares safety and efficacy information from the clinical trial of garetosmab, a fully-human monoclonal antibody blocking activin A 

On January 12, 2023, Regeneron Pharmaceuticals published LUMINA-1 Phase II Trial Results in medRxiv. medRxiv publishes preprints which have not been certified by peer review. You can see a list of authors on this publication below. 

Maja Di Rocco, Eduardo Forleo-Neto, Robert Pignolo, Richard Keen, Philippe Orcel, Thomas Funck-Brentano, Christian Roux, Sami Kolta, Annalisa Madeo, Judith S Bubbear, Jacek Tabarkiewicz, Małgorzata Szczepanek, Javier Bachiller-Corral, Angela M Cheung, Kathryn M Dahir, Esmée Botman, Pieter G Raijmakers, Mona Al Mukaddam, Lianne Tile, Cynthia Portal-Celhay, Neena Sarkar, Peijie Hou, Bret Musser, Anita Boyapati, Kusha Mohammadi, Scott Mellis, Aris N Economides, Dinko Gonzalez Trotter, Gary Herman, Sarah J O'Meara, Richard DelGizzi, David M. Weinreich, George D Yancopolous, E. Marelise W Eekhoff, Frederick S Kaplan

What’s Included in the medRxiv Publication

The medRxiv publication includes a 386-word Abstract, a 52-page publication and 67 pages of Supplementary Material. There is a lot of information in the Supplementary Material, so we have created a table of contents below. 

Thank You

As we see the first published results of the garetosmab LUMINA-1 Phase II Trial which also include case summaries of the patients who passed away, we are grateful for every patient and family that participated in this important clinical trial. We also appreciate the work of the trial investigators and their commitment to FOP families. We are also grateful to Regeneron Pharmaceuticals for their commitment to the FOP community and the development of garetosmab.  

Questions about this publication? Contact us at [email protected]

Phase III Clinical Trial

If you would like more information about the OPTIMA Phase III Clinical Trial for garetosmab, visit ifopa.org/optima.  

Key Terms

Some key terms you'll find in the publication.

Period 1 – Adult patients with FOP were randomized to garetosmab or placebo for 28 weeks

Period 2 – Open-label treatment period where all adult patients received garetosmab for 28 weeks

Open-label Extension – Patients were allowed to stay on garetosmab in an open-label extension following the end of Period 2

Adverse Event (AE) and Treatment Emergent Adverse Event (TEAE)
Source: European Patients’ Academy on Therapeutic Innovation

In medicines development terminology, an adverse event is any undesirable event that occurs after a participant officially consents to take part in a trial
(and could occur before treatment begins).

An adverse event may or may not be associated with the medicine under investigation, but must be documented because it happened during the trial period.

Treatment emergent adverse events are undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the treatment.

A treatment emergent adverse event is an adverse event that occurs only once treatment has started.

HO-Total Lesion Activity (HO-TLA) – Heterotopic ossification total lesion activity (evaluated by 18F-sodium fluoride positron emission tomography ([18F-NaF PET])

HO Total Lesion Volume – Heterotopic ossification total lesion activity evaluated by computed tomography (CT) scan

Table of Contents for Supplementary Materials

Figure S1. Activin A activates signaling by FOP-mutant ACVR1 but inhibits signaling by wild type ACVR1 - Page 1

Figure S2. Schematic overview of the study design - Page 2

Figure S3. Trial profile - Page 3

Figure S4. Percent of patients with new lesions by quantitative imaging in Period_1 - Page 4

Figure S5. Effect of garetosmab on flare-up events - Page 5

Figure S6. Effect of garetosmab in Period_2 relative to Period_1 - Page 6 

Figure S7. Coagulation and platelet functional assays at baseline and post treatment in LUMINA-1 patients - Page 7

Figure S8. Changes in BMP9 levels are minor and not associated with episodes of epistaxis - Page 9

Table S1. Overview of protocol and statistical methodology amendments - Page 13

Table S2. Key inclusion/exclusion criteria - Page 21

Table S3. List of Key Primary and Secondary Outcomes for Period_1, Period_2 and open label extension (Per Protocol) - Page 22

Table S4. Baseline demographics and disease characteristics - Page 27

Table S5. Summary of primary, and secondary endpoints for Period_1 and Period_2 - Page 30

Table S6. Key prespecified secondary, exploratory, and post-hoc analyses for Period 1 - Page 36 

Table S7. TEAEs that occurred in ≥10% of the garetosmab 10 mg/kg Q4W group in the double-blind period of the study (Period_1) - Page 38

Table S8. Skin and soft tissue infection TEAEs - Page 41

Table S9. Serious adverse events that occurred in the double-blind period of the study (Period_1) - Page 43

Table S10. Bleeding TEAEs - Page 44

Table S11. TEAEs that occurred in ≥10% in Period_2 and the open-label extension period until end of study - Page 45

Table S12. Serious adverse events that occurred in the open-label period - Page 48

Table S13. Case summaries of patients who died - Page 50

Table S14. Exploratory analysis of percent change of total volume (cm3) of target lesions by CT at week 8 and week 28 versus baseline - Page 54

Study Sites and Investigators - Page 56

Supplementary section: Methods - Page 57

Supplementary section: Results - Page 65

Supplementary references - Page 67

 

 

 

 

 

 

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