Phase 2 study of long-term safety, tolerability, PK and efficacy of recifercept in achondroplasia

Phase 1

• Conditions: Achondroplasia; MedDRA version: 25.0Level: LLTClassification code 10000452Term: AchondroplasiaSystem Organ Class: 100000004850; Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]

• Registration Number: EUCTR2021-003149-39-DK
• Lead Sponsor: Pfizer Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-03-10
• Last Update Posted: 8 August 2022

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 63

Inclusion Criteria

Age and Sex:
1.Male and female participants between the ages of =15 months to =12 years inclusive, at Visit 1 (Screen 1).
•Refer to Appendix 4 for reproductive criteria for male (Section 10.4.1) and female (Section 10.4.2) participants.

Type of Participant and Disease Characteristics:
2.Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests lifestyle considerations and other study procedures.
3.Completed the C4181005 Phase 2 study.
4.Able to stand independently for height measurements (if =2 years of age at enrollment).

Informed Consent:
5.Capable of giving signed informed consent/assent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the ICD and in this protocol.
6.Following receipt of oral and written information about the trial, the child (depending on local IRB/independent EC requirements) must provide assent, and 1 or both (according to local regulations) parent(s) or legal guardians of the child must provide signed informed consent before any trial-related activity is carried out.

Are the trial subjects under 18? yes
Number of subjects for this age range: 63
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Medical Conditions:
1.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.
2.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.
3.Presence of severe obesity (BMI >95th percentile on Hoover-Fong BMI charts) [Hoover-Fong et al, 2008].
4.Known closure of long bone growth plates (cessation of height growth).
5.Body weight = 45 kg.
6.History of hypersensitivity to study intervention or any excipients.

Prior/Concomitant Therapy:
7.Current use of any prohibited concomitant medication(s) or those unwilling/unable to use a permitted concomitant medication(s). Refer to Section 6.8 Concomitant Therapy.
8.History of any prior treatment with human growth hormone or related products (including IGF-1).
9.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 µg/day beclomethasone equivalent) and medication for attention deficit hyperactivity disorder).
10.History of limb lengthening surgery (defined as distraction osteogenesis/Ilizarov/callostasis technique following submetaphyseal osteotomy to extend bone length).
11.Any limb lengthening/corrective orthopaedic surgery planned at any point during the trial period.
12.Less than 6 months since fracture or surgical procedure of any bone determined from the screening visit date.
13.Presence of any internal guided growth plates/devices.
14.History of removal of internal guided growth plates/devices within less than 6 months.

Prior/Concurrent Clinical Study Experience:
15.History of receipt of any other (except recifercept) IP for achondroplasia or that may affect growth/interpretation of growth parameters.
16.Previous administration with an investigational drug (not for achondroplasia/growth affecting) within 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).

Other Exclusions:
17.Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified