Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
NCT ID: NCT05116046
Last Updated: 2024-02-08
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
35 participants
INTERVENTIONAL
2021-12-24
2023-03-30
Brief Summary
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Approximately 63 participants will be offered to continue at the previously received dose of Recifercept either
Low Dose Medium Dose High Dose
or at the therapeutic dose once it is identified.
Participants will attend the clinic monthly for 24 months. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements \& patient/caregiver quality of life questionnaires.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Low Dose
Low Dose
Recifercept
Recifercept
Medium Dose
Medium Dose
Recifercept
Recifercept
High Dose
High Dose
Recifercept
Recifercept
Interventions
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Recifercept
Recifercept
Eligibility Criteria
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Inclusion Criteria
* Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests lifestyle considerations and other study procedures.
* Completed the C4181005 Phase 2 study.
* Able to stand independently for height measurements (if ≥2 years of age at enrollment).
Exclusion Criteria
* Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
* Presence of severe obesity (body mass index (BMI) \>95th percentile on Hoover-Fong BMI charts) \[Hoover-Fong et al, 2008\].
* Known closure of long bone growth plates (cessation of height growth).
* Body weight \>45 kg.
* History of hypersensitivity to study intervention or any excipients.
* History of any prior treatment with human growth hormone or related products (including insulin-like growth factor 1 \[IGF-1\]).
* History of receipt of any treatment that are known to potentially affect growth (including oral steroids \>5 days in the last 6 months, high dose inhaled corticosteroids (\>800 mcg/day beclometasone equivalent) and medication for attention deficit hyperactivity disorder).
* History of limb lengthening surgery (defined as distraction osteogenesis/Ilizarov/callostasis technique following submetaphyseal osteotomy to extend bone length).
* Any limb lengthening/corrective orthopaedic surgery planned at any point during the trial period.
* Less than 6 months since fracture or surgical procedure of any bone determined from the screening visit date.
* Presence of any internal guided growth plates/devices.
* History of removal of internal guided growth plates/devices within less than 6 months.
* History of receipt of any other (except recifercept) investigational product for achondroplasia or that may affect growth/interpretation of growth parameters.
* History of receipt of an investigational drug (not for achondroplasia/growth affecting) within the last 30 days (or as determined by the local requirement) or 5 half-lives preceding the first dose of study intervention used in this study (whichever is longer).
15 Months
12 Years
ALL
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Ocean Sleep Medicine
Irvine, California, United States
Long Beach Memorial Medical Center
Long Beach, California, United States
MemorialCare Sleep Disorders Center at Long Beach Memorial Medical Center
Long Beach, California, United States
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Torrance, California, United States
Nemours Children's Hospital, Delaware
Wilmington, Delaware, United States
Texas Childrens Hospital/Baylor College of Medicine
Houston, Texas, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
UZ Leuven - Center of Human Genetics
Leuven, Flanders, Belgium
Antwerp University Hospital
Edegem, , Belgium
Bispebjerg Hospital
Copenhagen, , Denmark
Bispebjerg Hospital
Copenhagen, , Denmark
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Università Cattolica del Sacro Cuore
Roma, , Italy
Centro Hospitalar e Universitário de Coimbra - Hospital Pediátrico
Coimbra, , Portugal
Hospital Vithas San Jose
Vitoria-Gasteiz, Alava, Spain
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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2021-003149-39
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
C4181008
Identifier Type: -
Identifier Source: org_study_id
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