Fibrodysplasia Ossificans ProgressivaPhase 2LUMINA-1NCT03188666
A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
A Phase 2 study testing REGN2477 in people with Fibrodysplasia Ossificans Progressiva, run by Regeneron Pharmaceuticals at 11 sites in 8 countries.
Trial summary
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Fibrodysplasia Ossificans Progressiva
- What is being tested?
- REGN2477
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 11 sitesin 8 countries
- Study dates
- 26 Feb 2018to 16 Sep 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Men and women 18 to 60 years of age at screening.
- Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive heterotopic ossification (HO)).
- Confirmation of FOP diagnosis with documentation of any ACVR1 mutation.
- FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, and other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of heterotopic ossifications (increase in site or number of HO lesions) with/without being associated with flare-up episodes.
- Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study.
Who cannot join
- Significant concomitant illness or history of significant illness such as, but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study.
- Previous history or diagnosis of cancer.
- Use of bisphosphonate within 1 year of screening.
- Concurrent participation in another interventional clinical study, or a non-interventional study with radiographic measures or invasive procedures (e.g. collection of blood or tissue samples). Participation in the FOP Connection Registry or other studies in which participants complete study questionnaires are allowed.
- Treatment with another investigational drug, denosumab, imatinib or isotretinoin in the last 30 days or within 5 half-lives of the investigational drug, whichever is longer.
- Pregnant or breastfeeding women.
- Male and women of childbearing potential participants who are unwilling to practice highly effective contraception.
- Note: Other protocol defined Inclusion/Exclusion criteria apply
Trial locations and contacts
11 sites in 8 countries.
11 sites in 8 countries
United States3 sites
Vanderbilt University
Nashville, Tennessee, 37240
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
France2 sites
Hopital Cochin
Paris, 75014
Hopital Lariboisiere,Hospitalier Universitaire Nord
Paris, 751010
Netherlands1 site
VU University Medical Center
Amsterdam, North Holland, 1081 HV
United Kingdom1 site
Royal National Orthopaedic Hospital, Brockley Hill
Stanmore, Middlesex, HA7 4LP
About this study
Official title: A Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3).
Primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).
Read more
Primary efficacy objective of the study is to assess the effect of REGN2477 versus placebo on the change from baseline in heterotopic ossification (HO) in patients with FOP, as determined by 18-NaF uptake in HO lesions by positron emission tomography (PET) and in total volume of HO lesions by computed tomography (CT).
Key Secondary objectives are:
- To compare the effect of REGN2477 versus placebo on pain due to FOP, as measured by the area under the curve (AUC) for pain based on daily pain numeric rating scale (NRS) scores
- To assess the effect of REGN2477 versus placebo on the change from baseline in HO, as determined by the number of new HO lesions identified by 18F-NaF PET or by CT
- To assess the effect of REGN2477 versus placebo on the change from baseline in 18F-NaF standardized uptake value maximum (SUVmax) of individual active HO site(s) by PET
- To assess the effect of REGN2477, between week 28 and week 56, on the number, activity, and volume of HO lesions identified by 18F-NaF PET or by CT in patients who switch from placebo to REGN2477 at week 28 versus the same patients between baseline and week 28
- To assess the effect of REGN2477 versus placebo on the change from baseline in biochemical markers of bone formation
- To characterize the concentrations of total activin A at baseline and over time following the first dose of study drug
- To characterize the concentration-time profile (pharmacokinetics [PK]) of REGN2477 in patients with FOP
- To assess the immunogenicity of REGN2477
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Being tested
- REGN2477, Matching placebo
- Medical subject headings
- Myositis Ossificans
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
REGN2477Drug
Pharmaceutical form: liquid product for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment periods 1 and 2.
Matching placeboDrug
Pharmaceutical form: Liquid product for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment period 1 only.
| Study group | Role | What they receive |
|---|---|---|
| REGN2477 | Experimental | Not described Receives: Drug: REGN2477 |
| Placebo | Experimental | Not described Receives: Drug: Matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
Treatment-emergent adverse events (TEAEs) are adverse events not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period. A serious TEAE was defined as any untoward medical occurrence that resulted in any of following outcomes not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. Number of participants with TEAEs and Serious TEAEs are reported.
Measured: Up to Week 28
Period 1: Number of Participants With TEAEs by Severity
Severity of TEAEs were graded as follows: Mild: Does not interfere in a significant manner with the participant's normal functioning level. It may be an annoyance. Prescription drugs are not ordinarily needed for relief of symptoms but may be given because of personality of the participants. Moderate: Produces some impairment of functioning but is not hazardous to health. It was uncomfortable or an embarrassment. Treatment for symptom may be needed. Severe: Produces significant impairment of functioning or incapacitation and was a definite hazard to the participant's health. Treatment for symptom may be given and/or participants hospitalized. Number of participants with TEAEs by severity is reported.
Measured: Up to Week 28
Period 1: Time-Weighted Average (Standardized Area Under the Curve [AUC]) of the Percent Change From Baseline in Total Lesion Activity by Fluorine-18-labeled Sodium Fluoride (18^F-NaF) Positron Emission Tomography (PET) at Week 28 (AHO)
18^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (standardized area under the curve [AUC]) of the percent change from baseline in total lesion activity by 18^F-NaF PET up to Week 28 in AHO analysis set is reported.
Measured: Baseline and Week 28
Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by Computed Tomography (CT) at Week 28 (AHO)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions as assessed by CT during Period 1 at Week 28 is reported.
Measured: Week 28
Period 2: Number of New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. HO detectable by CT that developed after baseline are referred to as "new HO lesions." Number of new HO lesions as assessed by CT at Week 56 relative to Week 28 scan is reported.
Measured: Week 28, Week 56
Period 1: Time-weighted Average (Standardized AUC) of the Percent Change From Baseline in Total Lesion Activity Assessed by 18^F-NaF PET at Week 28 (AHOC)
18^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (Standardized AUC) of the percent change from baseline in total lesion activity as assessed by 18^F-NaF PET in Active HO Classic ACVR1 Mutation (AHOC) analysis set up to Week 28 is reported.
Measured: Week 28
Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by CT at Week 28 (AHOC)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions was assessed by CT at Week 28 in AHOC analysis set is reported.
Measured: Week 28
Secondary outcomes (36)
Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to Fibrodysplasia Ossificans Progressiva (FOP) Assessed by Daily Numeric Rating Scale (NRS) at Week 28 (AHO)
The pain NRS is a patient reported outcome (PRO) used by participants to rate their pain associated with FOP. Participants were asked to rate their pain on a scale that ranges from "0" (no pain) to "10" (worst possible pain), where the highest score indicated worst outcome. Time-weighted average (Standardized AUC) of the change from baseline in daily pain due to FOP assessed by daily NRS at Week 28 in AHO analysis set is reported.
Measured: Week 28
Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to FOP, Assessed by Daily NRS at Week 28 (AHOC)
The pain NRS is a PRO used by participants to rate their pain associated with FOP. Participants were asked to rate their pain on a scale that ranges from "0" (no pain) to "10" (worst possible pain), where the highest score indicated worst outcome. Time-Weighted average (standardized AUC) of the change from baseline in daily pain due to FOP assessed by daily NRS at Week 28 in AHOC analysis set is reported.
Measured: Week 28
Period 1: Percent Change From Baseline in 18^F-NaF SUVmax of Individual Active HO Site(s) Assessed by 18^F-NaF PET at Week 8 (AHOC)
Standardized uptake value max (SUVmax) was a measurement of the maximum radiopharmaceutical uptake within the volume of interest. Relative accuracy of a particular radiotracer in a particular tissue is determined by expressing the absolute accuracy (obtained in the primary outcome measure) in terms of percent difference between SUVmax values obtained from PET/CT. Percent Change in 18^F-NaF SUVmax of Individual Active HO Site(s) assessed by 18^F-NaF PET in AHOC analysis set is reported.
Measured: Week 8
Period 1: Percent Change From Baseline in 18^F-NaF SUVmax of Individual Active HO Site(s) as Assessed by 18^F-NaFPET at Week 8 (AHO)
Percent change in 18^F-NaF SUVmax of individual active HO site(s) as assessed by 18^F-NaF PET at Week 8 in AHO analysis set is reported.
Measured: Week 8
Period 1: Change From Baseline in Number of HO Lesions as Assessed by 18^F-NaF PET at Week 28 (AHOC)
Change from baseline in number of HO lesions was assessed by 18^F-NaF PET at Week 28 in AHOC analysis set is reported.
Measured: Week 28
Period 1: Change From Baseline in Number of HO Lesions as Assessed by 18^F-NaF PET at Week 28 (AHO)
Change from baseline in number of HO lesions was assessed by 18^F-NaF PET in AHO analysis set is reported.
Measured: Week 28
Period 1: Change From Baseline in Number of HO Lesions Detectable by CT at Week 28 (AHOC)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Change from baseline in number of HO lesions was detectable by CT using AHOC analysis set is reported.
Measured: Week 28
Period 1: Change From Baseline in Number of HO Lesions Detectable by CT at Week 28 (AHO)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Change from baseline in number of HO lesions detectable by CT at Week 28 in AHO analysis set is reported.
Measured: Week 28
Period 2: Number of New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Number of new HO lesions as assessed by 18^F-NaF PET at Week 56 Relative to Week 28 Scan is reported.
Measured: Week 28, Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percentage of participants with new HO lesions as assessed by CT at week 56 relative to week 28 scan is reported.
Measured: Week 28, Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
18^F-NaF PET is used to assess lesion and disease activity. Percentage of participants with new HO lesions as assessed by 18^F-NaF PET at week 56 relative to week 28 scan is reported.
Measured: Week 28, Week 56
Period 2: Number of New HO Lesions as Assessed by CT Only at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT Only: Computed Tomography (CT) assessment without reference to positron-emission tomography (PET). Number of new HO lesions as assessed by CT only at week 56 relative to week 28 scan is reported.
Measured: Week 28, Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by CT Only at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT Only: Computed Tomography (CT) assessment without reference to Positron-Emission Tomography (PET). Percentage of participants with new HO lesions as assessed by CT only at week 56 relative to week 28 scan is reported.
Measured: Week 28, Week 56
Period 2 vs. Period 1: Change From Week 28 in Number of Active HO Lesions as Assessed by 18^F-NaF PET to Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
18^F-NaF PET is used to assess lesion and disease activity. Difference of Change from Week 28 to Week 56 as assessed by 18^F-NaF PET versus from Baseline to Week 28
Measured: Week 28, Week 56
Period 2 vs. Period 1: Change From Week 28 in Number of Active HO Lesions as Assessed by CT Scan at Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Difference of Change from Week 28 to Week 56 as assessed by CT Scan versus from Baseline to Week 28 is reported
Measured: Week 28, Week 56
Period 2: Number of New HO Lesions as Assessed by CT at Week 56 Relative to Baseline (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Number of new HO lesions as assessed by CT at week 56 relative to baseline.
Measured: Baseline, Week 56
Period 2: Number of New HO Lesions as Assessed by CT Only at Week 56 Relative to Baseline (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT Only: Computed Tomography (CT) assessment without reference to Positron-Emission Tomography (PET); Number of new HO lesions as assessed by CT only at week 56 relative baseline is reported.
Measured: Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by CT at Week 56 Relative to Baseline (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percentage of participants with new HO lesions as assessed by CT at week 56 relative to baseline were reported.
Measured: Week 56
Period 2: Number of New HO Lesions as Assessed by 18^F-NAF PET at Week 56 Relative to Baseline (AHO COVID-19 mITT)
18^F-NaF PET is used to assess lesion and disease activity. Number of new HO lesions as assessed by 18^F-NAF PET at week 56 relative to baseline is reported.
Measured: Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Baseline (AHO COVID-19 mITT)
18^F-NaF PET is used to assess lesion and disease activity. Percentage of participants with new HO lesions as assessed by 18^F-NaF PET at week 56 relative to baseline.
Measured: Week 56
Period 2: Total Volume of New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Total volume of new HO lesions as assessed by CT at Week 56 relative to Week 28 scan.
Measured: Week 28, Week 56
Period 2: Total Lesion Activity (TLA) Assessed by 18^F-NaF PET in New HO Lesions at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
TLA is a measure of participant-level cumulative burden of metabolically active HO. Activity of individual HO lesions was calculated as the product of mean standard uptake value (SUVmean) and the PET volume of the active HO lesion. TLA was derived for each participant at each time point as the sum of HO lesion activity of individual target and new active HO lesions.
Measured: Week 28, Week 56
Period 2 vs. Period 1: Percent Change From Week 28 in TLA as Assessed by 18^F-NaF PET to Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
Difference of Percent Change from Week 28 to Week 56 versus from Baseline to Week 28 is reported
Measured: Week 28, Week 56
Period 2 vs. Period 1: Percent Change From Week 28 in the Total Volume of HO Lesions as Assessed by CT to Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Difference of Percent Change from Week 28 to Week 56 versus from Baseline to Week 28 is reported.
Measured: Week 28, Week 56
Period 2: TLA in New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Total Lesion Activity (TLA) is a measure of participant-level cumulative burden of metabolically active HO. TLA in New (Relative to Baseline) Lesions at Week 56 is reported.
Measured: Week 56
Period 2: Total Volume of New HO Lesions as Assessed by CT Only at Week 56 Relative to Baseline (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT Only: Computed Tomography (CT) assessment without reference to Positron-Emission Tomography (PET); Total volume of new HO lesions as assessed by CT only at week 56 relative to baseline is reported.
Measured: Week 56
Period 2: Percent Change From Baseline in TLA as Assessed by 18^F-NaF PET to Week 56 (AHO COVID-19 mITT)
Percent change from baseline in TLA as assessed by 18^F-NaF PET to week 56 were reported.
Measured: Week 56
Period 2: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by CT to Week 56 (AHO COVID-19 mITT)
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions as assessed by CT to Week 56 were reported.
Measured: Week 56
Period 2: Percent Change From Week 28 in SUVmax as Assessed by 18^F-NaF to Week 56 (AHO COVID-19 mITT)
Percent Change from Week 28 to Week 56 is reported.
Measured: Week 28 to Week 56
Period 2: Percent Change From Baseline in 18^F-NaF PET SUVmax to Week 56 (AHO COVID-19 mITT)
Percent change from baseline in 18^F-NaF PET SUVmax to week 56
Measured: Baseline, Week 56
Period 2: Daily Average Pain Due to FOP Measured Using the Daily NRS
The pain NRS is a patient reported outcome used by participants to rate their pain associated with FOP. Participants were asked to rate their pain on a scale that ranges from "0" (no pain) to "10" (worst possible pain), where the highest score indicated worst outcome.
Measured: Week 28 up to Week 56
Period 2: Percentage of Participants With Flare-ups Assessed by Participant E-diary
Percentage of participants with flare-ups starting between week 28 and week 56 as assessed by participant E-diary is reported.
Measured: Week 28 to Week 56
Period 2: Percentage of Participants With Investigator-assessed Flare-ups
Percentage of participants with investigator-assessed flare-ups were reported.
Measured: Week 28 to Week 56
Periods 1, 2, and 3: Concentration of Total Activin A in Serum
Concentration of total activin A in serum over time is reported.
Measured: Week 28, Week 56, Week 76
Periods 1, 2, and 3: Concentrations of Functional REGN2477 in Serum
Concentrations of REGN2477 capable of target binding were measured (functional drug).
Measured: Week 28, Week 56, Week 76
Periods 1, 2, and 3: Number of Participants With Clinical Impact of Treatment-Emergent Anti-drug Antibodies (ADA) to REGN2477
Immunogenicity was characterized by ADA responses \& titers. Responses categories: Negative - ADA negative response at all time points, regardless of missing samples; Pre-existing immunoreactivity - ADA positive response at baseline with all post first dose negative results or positive response at baseline with all post first dose ADA responses < 9-fold over baseline titer levels; Treatment-boosted response - positive response in the assay post first dose, >= 9-fold over baseline titer levels, when baseline results are positive; Treatment-emergent response - ADA positive response in the REGN2477 ADA assay post first dose when baseline results = negative or missing.
Measured: Up to Week 76
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2017
- Study start26 Feb 2018
- Primary completion16 Sep 2019
- Study completion16 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Fibrodysplasia Ossificans ProgressivaStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03188666
- Sponsor's protocol ID
- R2477-FOP-1623
- Other IDs
- 2016-005035-33, 2016
- Acronym
- LUMINA-1
- Status last verified
- Nov 2022
- First submitted
- 13 Jun 2017
- Last updated
- 2 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Baldasaro J, Sanchez RJ, Hartford C, Dahir KM, Keen R, Funck-Brentano T, Pignolo RJ, Davis M, Altman DE. Content Validation of the Flares Diary: A Qualitative Analysis of the Flares Experience Within the Fibrodysplasia Ossificans Progressiva (FOP) Population. Adv Ther. 2026 Apr;43(4):1827-1844. doi: 10.1007/s12325-026-03506-6. Epub 2026 Mar 10.
PubMed 41806277de Ruiter RD, Botman E, Teunissen BP, Lammertsma AA, Boellaard R, Raijmakers PG, Schwarte LA, Nieuwenhuijzen JA, Gonzalez Trotter D, Eekhoff EMW, Yaqub M. Performance of simplified methods for quantification of [18F]NaF uptake in fibrodysplasia ossificans progressiva. Front Nucl Med. 2024 Jul 22;4:1406947. doi: 10.3389/fnume.2024.1406947. eCollection 2024.
PubMed 39381032Keen R, Dahir KM, McGinniss J, Sanchez RJ, Mellis S, Economides AN, Di Rocco M, Orcel P, Roux C, Tabarkiewicz J, Bachiller-Corral J, Cheung AM, Al Mukaddam M, Mohammadi K, Gu J, Srinivasan D, Trotter DG, Eekhoff EMW, Kaplan FS, Pignolo RJ. Characterization of flare-ups and impact of garetosmab in adults with fibrodysplasia ossificans progressiva: a post hoc analysis of the randomized, double-blind, placebo-controlled LUMINA-1 trial. J Bone Miner Res. 2024 Sep 26;39(10):1486-1492. doi: 10.1093/jbmr/zjae140.
PubMed 39216107Di Rocco M, Forleo-Neto E, Pignolo RJ, Keen R, Orcel P, Funck-Brentano T, Roux C, Kolta S, Madeo A, Bubbear JS, Tabarkiewicz J, Szczepanek M, Bachiller-Corral J, Cheung AM, Dahir KM, Botman E, Raijmakers PG, Al Mukaddam M, Tile L, Portal-Celhay C, Sarkar N, Hou P, Musser BJ, Boyapati A, Mohammadi K, Mellis SJ, Rankin AJ, Economides AN, Trotter DG, Herman GA, O'Meara SJ, DelGizzi R, Weinreich DM, Yancopoulos GD, Eekhoff EMW, Kaplan FS. Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial. Nat Med. 2023 Oct;29(10):2615-2624. doi: 10.1038/s41591-023-02561-8. Epub 2023 Sep 28.
PubMed 37770652Vanhoutte F, Liang S, Ruddy M, Zhao A, Drewery T, Wang Y, DelGizzi R, Forleo-Neto E, Rajadhyaksha M, Herman G, Davis JD. Pharmacokinetics and Pharmacodynamics of Garetosmab (Anti-Activin A): Results From a First-in-Human Phase 1 Study. J Clin Pharmacol. 2020 Nov;60(11):1424-1431. doi: 10.1002/jcph.1638. Epub 2020 Jun 18.
PubMed 32557665Aykul S, Corpina RA, Goebel EJ, Cunanan CJ, Dimitriou A, Kim HJ, Zhang Q, Rafique A, Leidich R, Wang X, McClain J, Jimenez J, Nannuru KC, Rothman NJ, Lees-Shepard JB, Martinez-Hackert E, Murphy AJ, Thompson TB, Economides AN, Idone V. Activin A forms a non-signaling complex with ACVR1 and type II Activin/BMP receptors via its finger 2 tip loop. Elife. 2020 Jun 9;9:e54582. doi: 10.7554/eLife.54582.
PubMed 32515349
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
- ClinicalTrials.gov ID
- NCT03188666
- Acronym / sponsor ID
- LUMINA-1 / R2477-FOP-1623
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 26 Feb 2018 (actual)
- Primary completion
- 16 Sep 2019 (actual)
- Study completion
- 16 Sep 2021 (actual)
- First posted
- 15 Jun 2017 (actual)
- Results
- Posted 2 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3).
Primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).
Primary efficacy objective of the study is to assess the effect of REGN2477 versus placebo on the change from baseline in heterotopic ossification (HO) in patients with FOP, as determined by 18-NaF uptake in HO lesions by positron emission tomography (PET) and in total volume of HO lesions by computed tomography (CT).
Key Secondary objectives are:
- To compare the effect of REGN2477 versus placebo on pain due to FOP, as measured by the area under the curve (AUC) for pain based on daily pain numeric rating scale (NRS) scores
- To assess the effect of REGN2477 versus placebo on the change from baseline in HO, as determined by the number of new HO lesions identified by 18F-NaF PET or by CT
- To assess the effect of REGN2477 versus placebo on the change from baseline in 18F-NaF standardized uptake value maximum (SUVmax) of individual active HO site(s) by PET
- To assess the effect of REGN2477, between week 28 and week 56, on the number, activity, and volume of HO lesions identified by 18F-NaF PET or by CT in patients who switch from placebo to REGN2477 at week 28 versus the same patients between baseline and week 28
- To assess the effect of REGN2477 versus placebo on the change from baseline in biochemical markers of bone formation
- To characterize the concentrations of total activin A at baseline and over time following the first dose of study drug
- To characterize the concentration-time profile (pharmacokinetics [PK]) of REGN2477 in patients with FOP
- To assess the immunogenicity of REGN2477
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| REGN2477 | Experimental | Drug: REGN2477 |
| Placebo | Experimental | Drug: Matching placebo |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Men and women 18 to 60 years of age at screening.
- Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive heterotopic ossification (HO)).
- Confirmation of FOP diagnosis with documentation of any ACVR1 mutation.
- FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, and other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of heterotopic ossifications (increase in site or number of HO lesions) with/without being associated with flare-up episodes.
- Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study.
Key exclusion criteria (8 listed)
- Significant concomitant illness or history of significant illness such as, but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study.
- Previous history or diagnosis of cancer.
- Use of bisphosphonate within 1 year of screening.
- Concurrent participation in another interventional clinical study, or a non-interventional study with radiographic measures or invasive procedures (e.g. collection of blood or tissue samples). Participation in the FOP Connection Registry or other studies in which participants complete study questionnaires are allowed.
- Treatment with another investigational drug, denosumab, imatinib or isotretinoin in the last 30 days or within 5 half-lives of the investigational drug, whichever is longer.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| REGN2477Pharmaceutical form: liquid product for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment periods 1 and 2. | Drug | REGN2477 |
| Matching placeboPharmaceutical form: Liquid product for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment period 1 only. | Drug | Placebo |
Primary outcome
Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Up to Week 28
Treatment-emergent adverse events (TEAEs) are adverse events not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period. A serious TEAE was defined as any untoward medical occurrence that resulted in any of following outcomes not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. Number of participants with TEAEs and Serious TEAEs are reported.
Period 1: Number of Participants With TEAEs by Severity
Time frame: Up to Week 28
Severity of TEAEs were graded as follows: Mild: Does not interfere in a significant manner with the participant's normal functioning level. It may be an annoyance. Prescription drugs are not ordinarily needed for relief of symptoms but may be given because of personality of the participants. Moderate: Produces some impairment of functioning but is not hazardous to health. It was uncomfortable or an embarrassment. Treatment for symptom may be needed. Severe: Produces significant impairment of functioning or incapacitation and was a definite hazard to the participant's health. Treatment for symptom may be given and/or participants hospitalized. Number of participants with TEAEs by severity is reported.
Period 1: Time-Weighted Average (Standardized Area Under the Curve [AUC]) of the Percent Change From Baseline in Total Lesion Activity by Fluorine-18-labeled Sodium Fluoride (18^F-NaF) Positron Emission Tomography (PET) at Week 28 (AHO)
Time frame: Baseline and Week 28
18^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (standardized area under the curve [AUC]) of the percent change from baseline in total lesion activity by 18^F-NaF PET up to Week 28 in AHO analysis set is reported.
Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by Computed Tomography (CT) at Week 28 (AHO)
Time frame: Week 28
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions as assessed by CT during Period 1 at Week 28 is reported.
Period 2: Number of New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. HO detectable by CT that developed after baseline are referred to as "new HO lesions." Number of new HO lesions as assessed by CT at Week 56 relative to Week 28 scan is reported.
Period 1: Time-weighted Average (Standardized AUC) of the Percent Change From Baseline in Total Lesion Activity Assessed by 18^F-NaF PET at Week 28 (AHOC)
Time frame: Week 28
18^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (Standardized AUC) of the percent change from baseline in total lesion activity as assessed by 18^F-NaF PET in Active HO Classic ACVR1 Mutation (AHOC) analysis set up to Week 28 is reported.
Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by CT at Week 28 (AHOC)
Time frame: Week 28
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions was assessed by CT at Week 28 in AHOC analysis set is reported.
36 secondary outcomes
Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to Fibrodysplasia Ossificans Progressiva (FOP) Assessed by Daily Numeric Rating Scale (NRS) at Week 28 (AHO)
Time frame: Week 28
Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to FOP, Assessed by Daily NRS at Week 28 (AHOC)
Time frame: Week 28
Period 1: Percent Change From Baseline in 18^F-NaF SUVmax of Individual Active HO Site(s) Assessed by 18^F-NaF PET at Week 8 (AHOC)
Time frame: Week 8
Period 1: Percent Change From Baseline in 18^F-NaF SUVmax of Individual Active HO Site(s) as Assessed by 18^F-NaFPET at Week 8 (AHO)
Time frame: Week 8
Period 1: Change From Baseline in Number of HO Lesions as Assessed by 18^F-NaF PET at Week 28 (AHOC)
Time frame: Week 28
Period 1: Change From Baseline in Number of HO Lesions as Assessed by 18^F-NaF PET at Week 28 (AHO)
Time frame: Week 28
Period 1: Change From Baseline in Number of HO Lesions Detectable by CT at Week 28 (AHOC)
Time frame: Week 28
Period 1: Change From Baseline in Number of HO Lesions Detectable by CT at Week 28 (AHO)
Time frame: Week 28
Period 2: Number of New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2: Number of New HO Lesions as Assessed by CT Only at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by CT Only at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2 vs. Period 1: Change From Week 28 in Number of Active HO Lesions as Assessed by 18^F-NaF PET to Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2 vs. Period 1: Change From Week 28 in Number of Active HO Lesions as Assessed by CT Scan at Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2: Number of New HO Lesions as Assessed by CT at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Time frame: Baseline, Week 56
Period 2: Number of New HO Lesions as Assessed by CT Only at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by CT at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Number of New HO Lesions as Assessed by 18^F-NAF PET at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Percentage of Participants With New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Total Volume of New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2: Total Lesion Activity (TLA) Assessed by 18^F-NaF PET in New HO Lesions at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2 vs. Period 1: Percent Change From Week 28 in TLA as Assessed by 18^F-NaF PET to Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2 vs. Period 1: Percent Change From Week 28 in the Total Volume of HO Lesions as Assessed by CT to Week 56 (Period 2) Versus the Same Participants Between Baseline and Week 28 (Period 1) (AHO COVID-19 mITT)
Time frame: Week 28, Week 56
Period 2: TLA in New HO Lesions as Assessed by 18^F-NaF PET at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Total Volume of New HO Lesions as Assessed by CT Only at Week 56 Relative to Baseline (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Percent Change From Baseline in TLA as Assessed by 18^F-NaF PET to Week 56 (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by CT to Week 56 (AHO COVID-19 mITT)
Time frame: Week 56
Period 2: Percent Change From Week 28 in SUVmax as Assessed by 18^F-NaF to Week 56 (AHO COVID-19 mITT)
Time frame: Week 28 to Week 56
Period 2: Percent Change From Baseline in 18^F-NaF PET SUVmax to Week 56 (AHO COVID-19 mITT)
Time frame: Baseline, Week 56
Period 2: Daily Average Pain Due to FOP Measured Using the Daily NRS
Time frame: Week 28 up to Week 56
Period 2: Percentage of Participants With Flare-ups Assessed by Participant E-diary
Time frame: Week 28 to Week 56
Period 2: Percentage of Participants With Investigator-assessed Flare-ups
Time frame: Week 28 to Week 56
Periods 1, 2, and 3: Concentration of Total Activin A in Serum
Time frame: Week 28, Week 56, Week 76
Periods 1, 2, and 3: Concentrations of Functional REGN2477 in Serum
Time frame: Week 28, Week 56, Week 76
Periods 1, 2, and 3: Number of Participants With Clinical Impact of Treatment-Emergent Anti-drug Antibodies (ADA) to REGN2477
Time frame: Up to Week 76
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41806277Baldasaro J, Sanchez RJ, Hartford C, Dahir KM, Keen R, Funck-Brentano T, Pignolo RJ, Davis M, Altman DE. Content Validation of the Flares Diary: A Qualitative Analysis of the Flares Experience Within the Fibrodysplasia Ossificans Progressiva (FOP) Population. Adv Ther. 2026 Apr;43(4):1827-1844. doi: 10.1007/s12325-026-03506-6. Epub 2026 Mar 10.
- Derived from this study: PubMed 39381032de Ruiter RD, Botman E, Teunissen BP, Lammertsma AA, Boellaard R, Raijmakers PG, Schwarte LA, Nieuwenhuijzen JA, Gonzalez Trotter D, Eekhoff EMW, Yaqub M. Performance of simplified methods for quantification of [18F]NaF uptake in fibrodysplasia ossificans progressiva. Front Nucl Med. 2024 Jul 22;4:1406947. doi: 10.3389/fnume.2024.1406947. eCollection 2024.
- Derived from this study: PubMed 39216107Keen R, Dahir KM, McGinniss J, Sanchez RJ, Mellis S, Economides AN, Di Rocco M, Orcel P, Roux C, Tabarkiewicz J, Bachiller-Corral J, Cheung AM, Al Mukaddam M, Mohammadi K, Gu J, Srinivasan D, Trotter DG, Eekhoff EMW, Kaplan FS, Pignolo RJ. Characterization of flare-ups and impact of garetosmab in adults with fibrodysplasia ossificans progressiva: a post hoc analysis of the randomized, double-blind, placebo-controlled LUMINA-1 trial. J Bone Miner Res. 2024 Sep 26;39(10):1486-1492. doi: 10.1093/jbmr/zjae140.
- Derived from this study: PubMed 37770652Di Rocco M, Forleo-Neto E, Pignolo RJ, Keen R, Orcel P, Funck-Brentano T, Roux C, Kolta S, Madeo A, Bubbear JS, Tabarkiewicz J, Szczepanek M, Bachiller-Corral J, Cheung AM, Dahir KM, Botman E, Raijmakers PG, Al Mukaddam M, Tile L, Portal-Celhay C, Sarkar N, Hou P, Musser BJ, Boyapati A, Mohammadi K, Mellis SJ, Rankin AJ, Economides AN, Trotter DG, Herman GA, O'Meara SJ, DelGizzi R, Weinreich DM, Yancopoulos GD, Eekhoff EMW, Kaplan FS. Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial. Nat Med. 2023 Oct;29(10):2615-2624. doi: 10.1038/s41591-023-02561-8. Epub 2023 Sep 28.
- Derived from this study: PubMed 32557665Vanhoutte F, Liang S, Ruddy M, Zhao A, Drewery T, Wang Y, DelGizzi R, Forleo-Neto E, Rajadhyaksha M, Herman G, Davis JD. Pharmacokinetics and Pharmacodynamics of Garetosmab (Anti-Activin A): Results From a First-in-Human Phase 1 Study. J Clin Pharmacol. 2020 Nov;60(11):1424-1431. doi: 10.1002/jcph.1638. Epub 2020 Jun 18.
- Derived from this study: PubMed 32515349Aykul S, Corpina RA, Goebel EJ, Cunanan CJ, Dimitriou A, Kim HJ, Zhang Q, Rafique A, Leidich R, Wang X, McClain J, Jimenez J, Nannuru KC, Rothman NJ, Lees-Shepard JB, Martinez-Hackert E, Murphy AJ, Thompson TB, Economides AN, Idone V. Activin A forms a non-signaling complex with ACVR1 and type II Activin/BMP receptors via its finger 2 tip loop. Elife. 2020 Jun 9;9:e54582. doi: 10.7554/eLife.54582.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
11 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing REGN2477 in people with Fibrodysplasia Ossificans Progressiva, run by Regeneron Pharmaceuticals at 11 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, France, Canada, Italy, Netherlands and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs (time frame: Up to Week 28). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03188666, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Dec 2022.