A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

Stopped early · Phase 2

Conditions studied: Achondroplasia

In brief

Approximately 63 participants will be randomized to one of three doses to receive Recifercept either * Low Dose * Medium Dose * High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2, 3 6, 9 \& 12. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements \& patient/caregiver quality of life questionnaires Participants will received treatment with Recifercept for 12 months. All participants who complete the study and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll into an open-label extension (OLE) study. A PK cohort will include 12 participants who will randomly receive a single dose of 3 mg/kg of Phase 2 study (process 1c) formulation and a single dose of 3 mg/kg of the proposed Phase 3 (process 2) study formulation in a cross over study. Dose of the cohort could be changed due to emerging safety and efficacy data in the study.

Key facts

Study ID

NCT04638153

Run by

Pfizer

People needed

60

Starts

2020-12-02

Expected to finish

2023-03-27

Last updated by the study team

2024-02-15

Who can join

Age: 0 and older, up to 10. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Main cohort: Aged ≥2 years to <11 years (up to the day before 11th birthday inclusive) at time of enrollment; or exploratory cohort: aged ≥3 months to <2 years (up to the day before 2nd birthday inclusive) at time of enrollment
• Documented, confirmed genetic diagnosis of achondroplasia from historical medical records prior to entry into this trial (test must have been performed at a laboratory fully accredited for genetic testing under local regulations).
• Completed the C4181001 natural history study with at least 2 valid height/length measurements (at least 3 months apart) prior to enrollment in this study. One of these measurement timepoints must be within the 3 months prior to enrollment in C4181005.
• Tanner stage 1 based on investigator assessment during physical examination (must include assessment of breast development for females, testicular stage for males).
• Able to stand independently for height measurements (if ≥2 years of age at enrollment).
• If aged <2 years at enrollment, has a documented historical MRI brain/cervical spine performed in the previous 12 months.

You may not qualify if…

• Presence of co-morbid conditions or circumstances that, in the opinion of the investigator, would affect interpretation of growth data or ability to complete the trial procedures.
• 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 (BMI >95th percentile on Hoover-Fong BMI charts) [Hoover-Fong et al, 2008].14
• Known closure of long bone growth plates (cessation of height growth).
• Body weight <7 kg or >30 kg.
• Moderate or severe renal impairment CrCL GFR <60 mL/min/1.73m2 (Calculated GFR based on updated "bedside" Schwartz formula for pediatric patients (CrCL (mL/min/1.73 m2) = 0.413 * Height (cms)/ Serum cr (mg/dL) or hepatic impairment (AST/ALT >1.5 ULN).
• 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 beclametasone 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 investigational product for achondroplasia or that may affect growth/interpretation of growth parameters.
• History of receipt of an investigational product (not for achondroplasia/growth affecting) within the last 30 days or 5 half-lives (whichever is longer).

Where it is running

• Ocean Sleep Medicine — Aliso Viejo, California, United States
• Ocean Sleep Medicine — Irvine, 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 Alfred I duPont Hospital for Children — Wilmington, Delaware, United States
• Texas Children's Hospital — Houston, Texas, United States
• Murdoch Children's Research Institute — Melbourne, Victoria, Australia
• Murdoch Children's Research Institute — Parkville, Victoria, Australia
• Universitair Ziekenhuis Antwerpen — Edegem, Belgium
• Universitaire Ziekenhuizen Leuven (UZ Leuven) — Leuven, Belgium
• DanTrials ApS — Copenhagen NV, Denmark
• Fondazione Policlinico Universitario Agostino - Gemelli IRCCS — Roma, Italy
• Osaka University Hospital — Suita, Osaka, Japan
• Okayama University Hospital — Okayama, Japan
• Centro Hospitalar e Universitário de Coimbra - Hospital Pediátrico — Coimbra, Portugal
• Hospital Vithas San Jose — Vitoria-Gasteiz, Alava, Spain

Full record on ClinicalTrials.gov

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