A Study Of Safety, Tolerability And Effectiveness Of Reciferecept In Children With Achondroplasia
NCT04638153
ABOUT THIS STUDY
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
Pfizer Clinical Trials Contact Center
1-800-718-1021
- Aged ≥3 months to <11 years (up to the day before 11th 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.
- 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.
- 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).
NEED INFO?
Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative
TRY A NEW SEARCH
Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your city or zip code to find studies near you.
Based on your search, you may also be interested in
- Edegem,
- Vitoria-Gasteiz, Alava
- Los Angeles, California
- Columbia, Missouri
- Cincinnati, Ohio
- Houston, Texas
- Seattle, Washington
- Melbourne,
- Barcelona,
- Lausanne,
- Wilmington, Delaware
- Baltimore, Maryland
- Boston, Massachusetts
- Salt Lake City, Utah
- Antwerp,
- Toronto,
- Copenhagen,
- Marseille,
- Paris,
- Cologne,
- Magdeburg,
- Como,
- Genova,
- Milan,
- Rome,
- Coimbra,
- Málaga,
- Bristol,
- London,
- Newcastle,
- Sheffield,
Descriptive Information | |||||||
---|---|---|---|---|---|---|---|
Brief Title ICMJE | A Study Of Safety, Tolerability And Effectiveness Of Reciferecept In Children With Achondroplasia | ||||||
Official Title ICMJE | A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA | ||||||
Brief Summary | Approximately 63 participants will be randomized to one of three doses to receive Recifercept either
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. | ||||||
Detailed Description | This is a phase 2 randomized, 3 arm (3 active doses of Recifercept), parallel group dose finding study of safety, tolerability, PK and efficacy The total number of participants is 63 in 2 age straified cohorts of 0-2 years and 6-10 years old. The study will enroll approximately 54 children with achondroplasia aged 2-10 years (inclusive) who will be enrolled and randomized to receive one of three doses of recifercept
A total of 18 participants will be enrolled per dose 18 per dosesuch that at least 15 participants per dose are evaluable. An interim analysis is planned when at least 15 participants per dose aged ?2 to <11 years have received 6 months of treatment with recifercept. eDMC will review safety, PK and efficacy data to confirm ongoing positive benefit:risk in participants. Additionally, an exploratory cohort of approximately 9 children with achondroplasia, ages 0-2 years, will be enrolled later in the study (n=3 per dose). Enrollment will follow an age and dose-staggered approach (descending age and ascending dose) with review of safety and PK data by the study team before progression to the next enrollment block If certain pre-defined safety signals occur then a meeting of the eDMC will be convened to make a decision on progression of enrollment. The PK data collected in block A will be used in the PopPK model (developed using healthy adult data) to confirm the dosing for younger children (ie, ?2 to <6 years and 0-<2 years). 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 All participants will receive 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. | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 2 | ||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Single (Outcomes Assessor) Masking Description: Anthropometric Measurements Assessor Primary Purpose: Prevention
| ||||||
Condition ICMJE | Achondroplasia | ||||||
Intervention ICMJE | Biological: Recifercept
Recifercept | ||||||
Study Arms ICMJE |
| ||||||
Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||||
Recruitment Information | |||||||
Recruitment Status ICMJE | Recruiting | ||||||
Estimated Enrollment ICMJE | 63 | ||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||
Estimated Study Completion Date ICMJE | January 20, 2023 | ||||||
Estimated Primary Completion Date | January 20, 2023 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
| ||||||
Sex/Gender ICMJE |
| ||||||
Ages ICMJE | 3 Months to 10 Years (Child) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE |
| ||||||
Listed Location Countries ICMJE | Belgium, Spain | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT04638153 | ||||||
Other Study ID Numbers ICMJE | C4181005 2020-001189-13 ( EudraCT Number ) | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
| ||||||
IPD Sharing Statement ICMJE |
| ||||||
Responsible Party | Pfizer | ||||||
Study Sponsor ICMJE | Pfizer | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
| ||||||
PRS Account | Pfizer | ||||||
Verification Date | December 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |