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Trial record 10 of 29 for:    fop

A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva (LUMINA-1)

This study is not yet open for participant recruitment.
Verified June 2017 by Regeneron Pharmaceuticals
Sponsor:
ClinicalTrials.gov Identifier:
NCT03188666
First Posted: June 15, 2017
Last Update Posted: June 15, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Regeneron Pharmaceuticals
  Purpose

This is a two 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).

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).

Secondary objectives are:

  • 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 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 numeric rating scale (NRS) scores
  • 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

Condition Intervention Phase
Fibrodysplasia Ossificans Progressiva Drug: REGN2477 Drug: Matching placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Regeneron Pharmaceuticals:

Primary Outcome Measures:
  • Incidence and severity of treatment-emergent adverse events (TEAEs) through the end of the Treatment Period 1 at week 28 [ Time Frame: Baseline to week 28 ]
  • Time-weighted average (standardized area under the curve [AUC]) percent change from baseline in total lesion activity by 18F-NaF PET over 28 weeks [ Time Frame: Baseline to week 28 ]
  • Percent change from baseline in the total volume of HO lesions as assessed by CT at week 28 [ Time Frame: Baseline to week 28 ]

Secondary Outcome Measures:
  • Percent change from baseline in 18F-NaF SUVmax of individual active HO site(s) by PET at week 8 [ Time Frame: Baseline to week 8 ]
  • Change from baseline in number of HO lesions detectable by CT at week 28 [ Time Frame: Baseline to week 28 ]
  • Time-weighted average (standardized AUC) change from baseline in daily pain due to FOP, as measured using the daily NRS over 28 weeks [ Time Frame: Baseline to week 28 ]
  • Change from baseline in the volume of individual HO lesions as assessed by CT at week 28 [ Time Frame: Baseline to week 28 ]
  • Incidence and severity of TEAEs through the end of study [ Time Frame: Baseline to week 76 (end of study) ]
  • Time weighted average (standardized AUC) percent change from baseline in biomarkers of bone formation levels in serum over time [ Time Frame: Baseline to week 76 ]
  • Concentration of total activin A in serum over time [ Time Frame: Baseline to week 76 ]
  • PK profile of REGN2477, assessed as concentrations of REGN2477 in serum over time [ Time Frame: Baseline to week 76 ]
  • Immunogenicity of REGN2477 [ Time Frame: Baseline to week 76 ]
    As determined by the incidence, titer, and clinical impact of treatment-emergent ADA to REGN2477 over time


Estimated Enrollment: 40
Anticipated Study Start Date: October 9, 2017
Estimated Study Completion Date: April 9, 2020
Estimated Primary Completion Date: May 9, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: REGN2477 Drug: REGN2477
Pharmaceutical form: Powder for solution for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment periods 1 and 2.
Experimental: Placebo Drug: Matching placebo
Pharmaceutical form: Powder for solution for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment period 1 only.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Men and women 18 to 60 years of age at screening.
  2. Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive heterotopic ossification).
  3. Confirmation of classic FOP diagnosis with documentation of the ACVR1[R206H] mutation.
  4. 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.
  5. Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study.

Key Exclusion Criteria:

  1. 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.
  2. Use of bisphosphonate within 1 year of screening.
  3. 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 patients complete study questionnaires are allowed.
  4. Pregnant or breastfeeding women.
  5. Male and women of childbearing potential patients who are unwilling to practice highly effective contraception.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03188666


Contacts
Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

Sponsors and Collaborators
Regeneron Pharmaceuticals
Investigators
Study Director: Clinical Trial Management Regeneron Pharmaceuticals
  More Information

Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03188666     History of Changes
Other Study ID Numbers: R2477-FOP-1623
2016-005035-33 ( EudraCT Number )
First Submitted: June 13, 2017
First Posted: June 15, 2017
Last Update Posted: June 15, 2017
Last Verified: June 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:
Myositis Ossificans
Myositis
Muscular Diseases
Musculoskeletal Diseases