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Study of Infigratinib in Children With Achondroplasia

Primary Purpose

Achondroplasia

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Infigratinib 0.016 mg/kg
Infigratinib 0.032 mg/kg
Infigratinib 0.064 mg/kg
Infigratinib 0.128 mg/kg
Infigratinib 0.25 mg/kg
Sponsored by
QED Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Achondroplasia focused on measuring Skeletal dysplasia, Endochondral ossification, ACH, Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism, dwarfism, Bone disease, Bone diseases, developmental, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, inborn, Inborn

Eligibility Criteria

3 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).
  2. Diagnosis of ACH, documented clinically and confirmed by genetic testing.
  3. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
  4. Ambulatory and able to stand without assistance
  5. Able to swallow oral medication.

Exclusion Criteria:

  1. Hypochondroplasia or short stature condition other than ACH.
  2. In females, having had their menarche.
  3. Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH.
  4. Significant concurrent disease or condition that, in the view of the Investigator and/or Sponsor, would confound assessment of efficacy or safety of infigratinib.
  5. Current evidence of corneal or retinal disorder/keratopathy.
  6. History of malignancy.
  7. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration.
  8. Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time.
  9. Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time.
  10. Regular long-term treatment (>3 weeks) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
  11. Treatment with any other investigational product or investigational medical device for the treatment of ACH or short stature.
  12. Previous limb-lengthening surgery or guided growth surgery.
  13. Fracture within 6 months of screening.

Sites / Locations

  • UCSF Benioff Children's HospitalRecruiting
  • Nemours Alfred I. Dupont Hospital for ChildrenRecruiting
  • Johns Hopkins School of MedicineRecruiting
  • Cincinnati Children's Hospital Medical CenterRecruiting
  • Vanderbilt University Medical CenterRecruiting
  • Murdoch Children's HospitalRecruiting
  • Stollery Children's HospitalRecruiting
  • Hopital Femme Mere EnfantRecruiting
  • Hopital Necker-Enfants MaladesRecruiting
  • Hopital des EnfantsRecruiting
  • Hospital Universitario La PazRecruiting
  • Hospital Universitario Virgen de la VictoriaRecruiting
  • Vithas Hospital San JoséRecruiting
  • Sheffield Children's HospitalRecruiting
  • Birmingham Children's HospitalRecruiting
  • University Hospitals Bristol and Weston NHS Foundation TrustRecruiting
  • Queen Elizabeth University HospitalRecruiting
  • Evelina London Children's HospitalRecruiting
  • Manchester University Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

Infigratinib 0.016 mg/kg

Infigratinib 0.032 mg/kg

Infigratinib 0.064 mg/kg

Infigratinib 0.128 mg/kg

Infigratinib 0.25 mg/kg

Arm Description

Dose Escalation: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.

Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.

Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.

Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. Dose Expansion: Upon identification of the recommended dose from all cohorts analyzed, an expansion cohort of 20 subjects may begin enrollment to further determine safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the selected dose.

Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.

Outcomes

Primary Outcome Measures

Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Change from baseline in annualized height velocity
PK parameters of infigratinib (Cmax- PK substudy only)
PK parameters of infigratinib (Clast- PK substudy only)
PK parameters of infigratinib (Tmax- PK substudy only)
PK parameters of infigratinib (AUC24- PK substudy only)
PK parameters of infigratinib (T1/2- PK substudy only)
PK parameters of infigratinib (AUCinf- PK substudy only)
PK parameters of infigratinib (CL/F- PK substudy only)
PK parameters of infigratinib (Vz/F- PK substudy only)
PK parameters of infigratinib (Racc- PK substudy only)

Secondary Outcome Measures

Incidence of adverse events (AEs) and serious adverse events (SAEs) as a measure of safety and tolerability
Absolute height velocity (annualized to cm/year), expressed numerically and as Z-score in relation to ACH and non-ACH tables
Absolute and change from baseline in weight (kg)
Absolute and change from baseline in sitting height (cm)
Absolute and change from baseline in head circumference (cm)
Absolute and change from baseline in upper and lower arm length (cm)
Absolute and change from baseline in thigh length (cm)
Absolute and change from baseline in knee height (cm)
Absolute and change from baseline in arm span (cm)
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Changes in pharmacodynamic parameters by assessing collagen X marker
Changes in pharmacodynamic parameters by assessing serum type 1 collagen c-telopeptide
Changes in pharmacodynamic parameters by assessing procollagen type 1 N-telopeptide
Changes in pharmacodynamic parameters by assessing bone-specific alkaline phosphatase

Full Information

First Posted
January 29, 2020
Last Updated
February 6, 2023
Sponsor
QED Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04265651
Brief Title
Study of Infigratinib in Children With Achondroplasia
Official Title
Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 10, 2020 (Actual)
Primary Completion Date
May 2023 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
QED Therapeutics, Inc.

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Achondroplasia
Keywords
Skeletal dysplasia, Endochondral ossification, ACH, Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism, dwarfism, Bone disease, Bone diseases, developmental, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, inborn, Inborn

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
108 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Infigratinib 0.016 mg/kg
Arm Type
Experimental
Arm Description
Dose Escalation: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Arm Title
Infigratinib 0.032 mg/kg
Arm Type
Experimental
Arm Description
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Arm Title
Infigratinib 0.064 mg/kg
Arm Type
Experimental
Arm Description
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Arm Title
Infigratinib 0.128 mg/kg
Arm Type
Experimental
Arm Description
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. Dose Expansion: Upon identification of the recommended dose from all cohorts analyzed, an expansion cohort of 20 subjects may begin enrollment to further determine safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the selected dose.
Arm Title
Infigratinib 0.25 mg/kg
Arm Type
Experimental
Arm Description
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Intervention Type
Drug
Intervention Name(s)
Infigratinib 0.016 mg/kg
Intervention Description
Initial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Intervention Type
Drug
Intervention Name(s)
Infigratinib 0.032 mg/kg
Intervention Description
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Intervention Type
Drug
Intervention Name(s)
Infigratinib 0.064 mg/kg
Intervention Description
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Intervention Type
Drug
Intervention Name(s)
Infigratinib 0.128 mg/kg
Intervention Description
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Intervention Type
Drug
Intervention Name(s)
Infigratinib 0.25 mg/kg
Intervention Description
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Time Frame
Up to 18 months
Title
Change from baseline in annualized height velocity
Time Frame
Up to 18 months
Title
PK parameters of infigratinib (Cmax- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (Clast- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (Tmax- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (AUC24- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (T1/2- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (AUCinf- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (CL/F- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (Vz/F- PK substudy only)
Time Frame
21 days
Title
PK parameters of infigratinib (Racc- PK substudy only)
Time Frame
21 days
Secondary Outcome Measure Information:
Title
Incidence of adverse events (AEs) and serious adverse events (SAEs) as a measure of safety and tolerability
Time Frame
Up to 18 months
Title
Absolute height velocity (annualized to cm/year), expressed numerically and as Z-score in relation to ACH and non-ACH tables
Time Frame
Up to 18 months
Title
Absolute and change from baseline in weight (kg)
Time Frame
Up to 18 months
Title
Absolute and change from baseline in sitting height (cm)
Time Frame
Up to 18 months
Title
Absolute and change from baseline in head circumference (cm)
Time Frame
Up to 18 months
Title
Absolute and change from baseline in upper and lower arm length (cm)
Time Frame
Up to 18 months
Title
Absolute and change from baseline in thigh length (cm)
Time Frame
Up to 18 months
Title
Absolute and change from baseline in knee height (cm)
Time Frame
Up to 18 months
Title
Absolute and change from baseline in arm span (cm)
Time Frame
Up to 18 months
Title
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Time Frame
Up to 18 months
Title
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Time Frame
Up to 18 months
Title
Changes in pharmacodynamic parameters by assessing collagen X marker
Time Frame
Up to 18 months
Title
Changes in pharmacodynamic parameters by assessing serum type 1 collagen c-telopeptide
Time Frame
Up to 18 months
Title
Changes in pharmacodynamic parameters by assessing procollagen type 1 N-telopeptide
Time Frame
Up to 18 months
Title
Changes in pharmacodynamic parameters by assessing bone-specific alkaline phosphatase
Time Frame
Up to 18 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable). Diagnosis of ACH, documented clinically and confirmed by genetic testing. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry. Ambulatory and able to stand without assistance Able to swallow oral medication. Exclusion Criteria: Hypochondroplasia or short stature condition other than ACH. In females, having had their menarche. Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH. Significant concurrent disease or condition that, in the view of the Investigator and/or Sponsor, would confound assessment of efficacy or safety of infigratinib. Current evidence of corneal or retinal disorder/keratopathy. History of malignancy. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration. Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time. Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time. Regular long-term treatment (>3 weeks) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable). Treatment with any other investigational product or investigational medical device for the treatment of ACH or short stature. Previous limb-lengthening surgery or guided growth surgery. Fracture within 12 months of screening.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
QED Therapeutics VP, Clinical Development
Phone
1-877-280-5655
Email
PROPELstudyinfo@QEDTX.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
QED Therapeutics VP, Clinical Development
Organizational Affiliation
QED Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
UCSF Benioff Children's Hospital
City
Oakland
State/Province
California
ZIP/Postal Code
94618
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Leslie Lynch
Phone
510-428-3885
Ext
x7217
Email
leslie.lynch@ucsf.edu
First Name & Middle Initial & Last Name & Degree
Paul Harmatz
Facility Name
Nemours Alfred I. Dupont Hospital for Children
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michelle Norton
Phone
302-298-7978
Email
michelle.norton@nemours.org
First Name & Middle Initial & Last Name & Degree
Michael Bober
Facility Name
Johns Hopkins School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21211
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kristina Wade
Phone
410-502-2298
Email
klwade@jhmi.edu
First Name & Middle Initial & Last Name & Degree
Julie Hoover-Fong, MD
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rachael Powers
Phone
513-803-1177
Email
racheal.powers@cchmc.org
First Name & Middle Initial & Last Name & Degree
Howard Saal
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
LeeAnna Melton
Phone
615-343-6761
Email
leeanna.melton@vumc.org
First Name & Middle Initial & Last Name & Degree
John Phillips, III
Facility Name
Murdoch Children's Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Cassandra Choy
Phone
+61 3 9936 6541
Email
ravi.savarirayan@vcgs.org.au
First Name & Middle Initial & Last Name & Degree
Ravi Savarirayan
Facility Name
Stollery Children's Hospital
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2H7
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lee-Ann Carroll
Phone
(780) 492-6621
Email
peter.kannu@albertahealthservices.ca
First Name & Middle Initial & Last Name & Degree
Peter Kannu
Facility Name
Hopital Femme Mere Enfant
City
Lyon
Country
France
Individual Site Status
Recruiting
Facility Name
Hopital Necker-Enfants Malades
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nathalie Couteau
Phone
00 33 5 67 77 13 69
Email
couteau.n@chu-toulouse.fr
First Name & Middle Initial & Last Name & Degree
Jean Pierre Salles
Facility Name
Hopital des Enfants
City
Toulouse
Country
France
Individual Site Status
Recruiting
Facility Name
Hospital Universitario La Paz
City
Madrid
ZIP/Postal Code
24086
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Maria Salcedo
Phone
+34 650138642
Email
maria.salcedo@salud.madrid.org
First Name & Middle Initial & Last Name & Degree
Maria Salcedo
Facility Name
Hospital Universitario Virgen de la Victoria
City
Málaga
Country
Spain
Individual Site Status
Recruiting
Facility Name
Vithas Hospital San José
City
Vitoria-Gasteiz
State/Province
Álava
ZIP/Postal Code
01012
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ireme Gimeno
Phone
+34 945252077
Email
irene.gimeno@ucatrauma.com
First Name & Middle Initial & Last Name & Degree
Jorge Knörr
Facility Name
Sheffield Children's Hospital
City
Sheffield
State/Province
England
ZIP/Postal Code
S10 2TH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jayne Evans
Email
r.innovation@nhs.net
First Name & Middle Initial & Last Name & Degree
Paul Arundel
Facility Name
Birmingham Children's Hospital
City
Birmingham
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rita Chilton
Phone
0121 333 9550
Email
rita.chilton1@nhs.net
First Name & Middle Initial & Last Name & Degree
Vrinda Saraff
Facility Name
University Hospitals Bristol and Weston NHS Foundation Trust
City
Bristol
ZIP/Postal Code
BS1 3NU
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Arya Kumari
Phone
+44 01173420196
Email
Arya.Kumari@uhbw.nhs.uk
First Name & Middle Initial & Last Name & Degree
Toby Candler
Facility Name
Queen Elizabeth University Hospital
City
Glasgow
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hannah Williamson
Phone
01412327600
Email
glasgow.crf@ggc.nhs.uk
First Name & Middle Initial & Last Name & Degree
Helen McDevitt
Facility Name
Evelina London Children's Hospital
City
London
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sophie Williams
Phone
+44 2071887188
Email
sophie.williams@gsst.nhs.uk
First Name & Middle Initial & Last Name & Degree
Melita Irving
Facility Name
Manchester University Children's Hospital
City
Manchester
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Citations:
PubMed Identifier
35342457
Citation
Savarirayan R, De Bergua JM, Arundel P, McDevitt H, Cormier-Daire V, Saraff V, Skae M, Delgado B, Leiva-Gea A, Santos-Simarro F, Salles JP, Nicolino M, Rossi M, Kannu P, Bober MB, Phillips J 3rd, Saal H, Harmatz P, Burren C, Gotway G, Cho T, Muslimova E, Weng R, Rogoff D, Hoover-Fong J, Irving M. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022 Mar 21;14:1759720X221084848. doi: 10.1177/1759720X221084848. eCollection 2022.
Results Reference
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Study of Infigratinib in Children With Achondroplasia

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