Therachon´s Observational study open for recruitment

TA46-002 is the Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia

and is now public and fully available since 7th January 2019 in the website, with the full designation of "A Multi-center, Prospective, Longitudinal, Observational Study to Investigate the Clinical and Anthropometric Characteristics of Children With the Diagnosis Achondroplasia". This observational and non-interventional study (with no drug administered) will precede the phase 2 clinical trial.

Study description


This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatments. The company estimates to enroll 200 participants globally for this study.

Inclusion Criteria:

  1. Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out
  2. The child is able to provide written informed assent, where this is required according to national legislation, before any study related activity is carried out
  3. The child has been diagnosed as having achondroplasia documented by clinical diagnosis
  4. The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent
  5. The investigator has considered the family and prospective participating child being able to comply with the study procedures

Exclusion Criteria:

  1. The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital [SEDC], pseudoachondroplasia, trisomy 21)
  2. The child has any medical condition that may impact growth or where the treatment is known to impact growth, such as but not limited to hypothyroidism or hyperthyroidism, insulin-requiring diabetes mellitus, autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus [SLE], juvenile dermatomyositis, scleroderma, and others), autonomic neuropathy, or inflammatory bowel disease
  3. Treatment in the previous 12 months prior to consent and assent with growth hormone, insulin-like growth factor 1 (IGF-1), anabolic steroids, or any other drug expected to affect growth velocity
  4. Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months
  5. Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature
  6. Has had bone-related surgery impacting assessment of anthropometric measurements or is expected to have it during the study period. Children with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the date of consent/assent and healing is complete without sequelae as determined by the investigator
  7. Has any condition that in the view of the investigator places the child at high risk of poor compliance with the visit schedule or of not completing the study.
  8. Any concurrent disease or condition that in the view of the investigator would interfere with study participation

List of study centers where this study will take place

United States, Delaware
Alfred I. duPont Hospital for Children Not yet recruiting
Wilmington, Delaware, United States, 419803
United States, Maryland
The John Hopkins Not yet recruiting
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital Not yet recruiting
Boston, Massachusetts, United States, 02115
United States, Utah
University of Utah Health Not yet recruiting
Salt Lake City, Utah, United States, 84132
Antwerp University Hospital Recruiting
Antwerp, Belgium, 2650
The Hospital for Sick Children Recruiting
Toronto, Canada
Righospitalet Recruiting
Copenhagen, Denmark, 2100
Centre Hospitalier Universitaire La Timone Not yet recruiting
Marseille, France, 13385
Hopital Necker-Enfants Malades Not yet recruiting
Paris, France, 75015
University Hospital of Cologne Recruiting
Cologne, Germany, 50937
Otto-von-Guericke- Universitat Magdeburg Recruiting
Magdeburg, Germany, 39120
ASST Lariana Como Not yet recruiting
Como, Italy, 22042
IRCCS Istituto Giannina Gaslini Not yet recruiting
Genova, Italy, 6147
San Raffaele Hospital Not yet recruiting
Milan, Italy, 20132
Fondazione Policlinico Universitario A.Gemelli Not yet recruiting
Rome, Italy, 00168
Hospital Pediatrico de Coimbra Not yet recruiting
Coimbra, Portugal, 3000-602
Hospital Quironsalud Malaga Recruiting
Málaga, Spain
United Kingdom
Bristol Royal Children's Hospital Recruiting
Bristol, United Kingdom, BS2 8BJ
Guys & St Thomas NHS Trust Recruiting
London, United Kingdom, SE1 7EH
Newcastle Hospital NHS Foundation Trust Not yet recruiting
Newcastle, United Kingdom, NE1 3BZ
Sheffield Children's NHS Foundation Trust Recruiting
Sheffield, United Kingdom, S10 2TH

Study responsible in Therachon

Alison Slade - This email address is being protected from spambots. You need JavaScript enabled to view it.

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