Clinical trials: Craniofacial Abnormalities
- Condition Craniofacial Abnormalities
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
SGA, Turner Syndrome
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
The purpose of this study is to find out if somapacitan is safe and how well somapacitan works in children either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature.
- Conditions
- SGA, Turner Syndrome, Noonan Syndrome, ISS
- Trial name
- REAL 9 (NCT05723835)
- Testing
- Somapacitan
- Where
- 18 sites in United States, Malaysia and 4 more
- Volunteers
- 47 people
- Sponsor
- Novo Nordisk A/S
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
Noonan Syndrome
Effect of MAXOMAT ® on the Growth of Small Children to NOONAN's Syndrome
1. Clinical Objective : To improve the growth of these children 2. Genetic objective : A study of the genetics of the syndrome
- Trial name
- NCT00452725
- Testing
- MAXOMAT ®, biosynthetic growth hormone
- Where
- France
- Volunteers
- 36 people
- Sponsor
- Sanofi
DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
Thymus Transplantation in DiGeorge Syndrome #668
The study purpose is to determine whether cultured thymus tissue implantation (CTTI) is effective in treating typical complete DiGeorge syndrome.
- Trial name
- NCT00576407
- Testing
- Cultured Thymus Tissue for Implantation (CTTI)
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
SGA, Turner Syndrome, Noonan Syndrome, ISS
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow
The study compares two medicines for treatment of children born small and who stay small, or with Turner Syndrome, Noonan Syndrome, or idiopathic short stature.
- Trial name
- REAL 8 (NCT05330325)
- Testing
- Somapacitan, Norditropin®
- Where
- 199 sites in United States, Austria and 31 more
- Volunteers
- 412 people
- Sponsor
- Novo Nordisk A/S
DiGeorge Anomaly
Serum-Free Thymus Transplantation in DiGeorge Anomaly
The study purpose is to determine if thymus tissue cultured in a serum-free (SF) solution is a safe and effective treatment for atypical and typical complete DiGeorge anomaly. [Funding Source - FDA OOPD]
- Trial name
- SerumFree (NCT00849888)
- Testing
- Serum Free Thymus Transplantation with Immunosuppression, Serum Free Thymus Transplantation without immunosuppression
- Volunteers
- 2 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Microcephaly
Zika and Microcephaly: Case-control Study
It is suspected that zika virus infection during pregnancy is associated with microcephaly. The objective of this study is to investigate the occurrence of rash accompanied by three or more dengue-like signs and symptoms during…
- Trial name
- NCT02741882
- Testing
- Questionary
- Where
- Brazil
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Laboratório Sabin
22q11.2 Deletion Syndrome
Safety and Efficacy Study of NFC-1 in Subjects Aged 12-17 Years With 22q11.2DS & Associated Neuropsychiatric Conditions
This is a 5-week, multi-center, open-label, dose optimization trial in subjects aged 12-17 years with 22q11DS who have a diagnosis of anxiety disorder, and/or ADHD, and/or ASD.
- Trial name
- NCT02895906
- Testing
- NFC-1
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
DiGeorge Syndrome, DiGeorge Anomaly
Thymus Transplantation With Immunosuppression
The research purpose is to determine if thymus transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly.
- Conditions
- DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- 884 (NCT00579709)
- Testing
- Thymus Tissue for Transplantation
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Plagiocephaly, Brachycephaly
Cranial Remolding Orthosis Registry
A registry of infants fitted with a cranial remolding orthosis.
- Conditions
- Plagiocephaly, Brachycephaly, Asymmetric Head
- Trial name
- NCT06762691
- Testing
- MyCRO Band, Traditional CRO
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Otto Bock HealthCare LP
22q.11.2 Deletion/Duplication
Non-Invasive Chromosomal Evaluation of 22q11.2
This study is being conducted to develop and evaluate a cell-free fetal DNA test (Harmony) for non-invasive prenatal detection of 22q11.2 chromosomal deletion or duplication.
- Trial name
- 22Q (NCT02541058)
- Where
- Belgium
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- Cindy Cisneros
Noonan Syndrome
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment.
- Trial name
- NCT06668805
- Testing
- Vosoritide Injection
- Where
- 38 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- BioMarin Pharmaceutical
Spasticity, Muscle, Microcephaly
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss.
- Conditions
- Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation
- Trial name
- NCT05518188
- Testing
- MELPIDA
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Elpida Therapeutics SPC
Genetic Disorder, Noonan Syndrome
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as…
- Trial name
- NCT03435627
- Testing
- Somatropin
- Where
- 22 sites in Japan
- Volunteers
- 71 people
- Sponsor
- Novo Nordisk A/S
Atopic Dermatitis
A Study of Crisaborole Ointment 2%; Crisaborole Vehicle; TCS and TCI in Subjects Aged ≥ 2 Years, With Mild-moderate AD
This 4-week study will evaluate the safety and efficacy of crisaborole ointment 2%; crisaborole vehicle; topical corticosteroid and topical calcineurin inhibitor, applied twice daily (BID) in subjects who are at least 2 years of…
- Trial name
- NCT03539601
- Testing
- Crisaborole ointment, 2%, Hydrocortisone butyrate cream, 0.1%, Pimecrolimus cream, 1%
- Where
- 32 sites in United States, Germany and 5 more
- Volunteers
- 237 people
- Sponsor
- Pfizer
22q11 Deletion Syndrome
NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
This is a Phase 2, randomized, placebo-controlled crossover trial to assess the safety and efficacy of NB-001 in children and adolescents with 22q11DS that manifest commonly associated neuropsychiatric symptoms.
- Trial name
- NCT05290493
- Testing
- NB-001
- Where
- 4 sites in United States, Canada
- Volunteers
- 37 people
- Sponsor
- Nobias Therapeutics, Inc.
Genetic Disorder, Noonan Syndrome
Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome
This trial is conducted in Asia. The aim of the trial is to investigate the long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome.
- Trial name
- NCT01927861
- Testing
- somatropin
- Where
- 27 sites in Japan
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Microcephaly, Congenital Infection
Surveillance of Zika-related Microcephaly in Sub-Saharan Africa and Asia
This study will explore whether ZIKV is currently responsible for neurological complications, and particularly microcephaly, in Aedes-infested regions of Sub-Saharan Africa (SSA) and Asia.
- Trial name
- NCT03922594
- Testing
- Differential diagnosis for infectious causes of microcephaly
- Where
- 8 sites in Cameroon, China and 3 more
- Volunteers
- 150 people
- Sponsor
- Institut Pasteur
Plagiocephaly, Positional, Plagiocephaly, Nonsynostotic
Effectiveness of Ottobock Helmet Therapy in Infants With Plagiocephaly, Brachycephaly or Combination of Both
The objective of the research is to measure the effectiveness of the Ottobock helmet, an innovative 3D-printed cranial orthosis used to correct infant cranial positional deformation: plagiocephaly, brachycephaly or combination of…
- Conditions
- Plagiocephaly, Positional, Plagiocephaly, Nonsynostotic, Brachycephaly
- Trial name
- NCT05154175
- Testing
- Ottobock helmet
- Where
- 12 sites in France
- Volunteers
- 452 people
- Sponsor
- Otto Bock France SNC
Orthognathic Surgery, Maxillofacial Abnormalities
Study Evaluating the Performance and Safety of Global D Implants Indicated for Orthognathic Surgery
This study is set up within the framework of the European Union regulation 2017/745 on medical devices. Its objective is to confirm the performance and safety of the Global D implants (ORTRAUTEK® and MINITEK®) used for…
- Conditions
- Orthognathic Surgery, Maxillofacial Abnormalities, Maxilla Fracture
- Trial name
- CMF-ORTHO (NCT05271383)
- Testing
- Orthognathic surgery using Global D's implants.
- Where
- France
- Volunteers
- 198 people
- Sponsor
- Global D