31 completed, 8 terminated, 7 recruiting, 6 unknown. 60 Phase 2, 14 Phase 1, 4 Phase 3.
Follow Craniofacial Abnormalities: new trials by email Literature Review of Craniofacial Abnormalities trials Research Report on Craniofacial Abnormalities
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME) SVG103 - Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC) | Phase 1 / Phase 2 | Recruiting | Sovargen | 12 | 2026-07-14 | 2026-08-10 |
| Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD) Hematopoietic stem cell transplant (HSCT) - Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder | Phase 2 | Recruiting | Paul Szabolcs | 25 | 2026-05-04 | 2026-05-15 |
| A Phase 1b/2 Study of the Safety and Efficacy of the Monoclonal Antibody OM-RCA-01 in Patients With Metastatic Tumors Expressing Fibroblast Growth Factor Receptor 1 humanized monoclonal anti-FGFR1 antibody OM-RCA-01 - Renal Cell Carcinoma Metastatic, Prostate Cancer Metastatic | Phase 1 / Phase 2 | Recruiting | Kidney Cancer Research Bureau | 64 | 2025-12-02 | 2026-11-10 |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment Vosoritide Injection - Noonan Syndrome | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 30 | 2024-11-22 | 2026-09-23 |
| Evaluating the Accuracy of Soft Tissue Prediction in Patients With Mandibular Prognathism After Orthognathic Surgery Using Handheld Scanner Versus Optical Scanner A Prospective Clinical Trial face scanning pre and post orthognathic surgery - Mandibular Prognathism, Orthognathic Surgery | Phase 1 / Phase 2 | Enrolling by invitation | Cairo University | 11 | 2024-09-24 | 2024-12-09 |
| Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders Sertraline, Placebo - Neurodevelopmental Disorders, Autism | Phase 2 | Recruiting | Holland Bloorview Kids Rehabilitation Hospital | 130 | 2024-09-16 | 2026-07-27 |
| MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies Trametinib tablet, Standard therapy - Cardiomegaly, Noonan Syndrome | Phase 2 | Recruiting | Medical University of Warsaw | 40 | 2024-08-01 | 2024-08-15 |
| Augmented Reality for Orthognatic Surgery Patient Education Patient consultation - Malocclusion, Dentofacial Deformities | Phase 2 / Phase 3 | Completed | University Medical Center Groningen | 60 | 2023-10-01 | 2025-03-30 |
| Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) MELPIDA - Spasticity, Muscle, Microcephaly | Phase 1 / Phase 2 | Recruiting | Elpida Therapeutics SPC | 4 | 2023-02-15 | 2026-07-08 |
| BR790 in Combination With Anlotinib in Adult Subjects With Advanced Non-Small Cell Lung Cancer BR790+anlotinib - Non-Small Cell Lung Cancer | Phase 1 / Phase 2 | Unknown | Shanghai Gopherwood Biotech Co., Ltd. | 90 | 2023-02-01 | 2023-02-10 |
| Pharmacological Treatment on the Recovery of Neurosensory Disturbance After Bilateral Sagittal Split Osteotomy Orthognathic Surgery, Melatonin 10 MG Oral Tablet, Hydroxycobalamin - Retrognathism, Prognathism | Phase 2 / Phase 3 | Unknown | Universitat Internacional de Catalunya | 220 | 2022-09-30 | 2022-09-13 |
| Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Relapsed or Refractory Advanced Non-Small Cell Lung Cancer With an FGFR Alteration Pemigatinib - Non-Small Cell Lung Cancer (NSCLC) | Phase 2 | Completed | Incyte Corporation | 8 | 2022-04-29 | 2025-08-05 |
| NB-001 in Children and Adolescents With 22q11 Deletion Syndrome NB-001, Placebo - 22q11 Deletion Syndrome | Phase 2 | Completed | Nobias Therapeutics, Inc. | 37 | 2022-02-10 | 2025-02-10 |
| NCCH2006/MK010 Trial (FORTUNE Trial) E7090 - Advanced or Recurrent Solid Tumors, FGFR Gene Alterations | Phase 2 | Recruiting | National Cancer Center, Japan | 75 | 2021-06-15 | 2025-10-03 |
| Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma Derazantinib, Derazantinib-paclitaxel-ramucirumab combination - Gastric Adenocarcinoma | Phase 1 / Phase 2 | Terminated | Basilea Pharmaceutica | 47 | 2020-10-06 | 2024-04-04 |
| Vosoritide for Selected Genetic Causes of Short Stature Vosoritide - Short Stature | Phase 2 | Active not recruiting | Andrew Dauber | 56 | 2020-08-04 | 2026-03-18 |
| Dexmedetomidine Versus Sevoflurane in Children With Anticipated Difficult Intubation dexmedetomidine, sevoflurane - Craniofacial Abnormalities, Mandibular Hypoplasia | Phase 2 | Unknown | Alexandria University | 40 | 2020-06-01 | 2020-06-12 |
| Pemigatinib + Pembrolizumab vs Pemigatinib Alone vs Standard of Care for Urothelial Carcinoma (FIGHT-205) Pemigatinib, Pembrolizumab, Gemcitabine - Metastatic Urothelial Carcinoma, Unresectable Urothelial Carcinoma | Phase 2 | Terminated | Incyte Corporation | 7 | 2020-05-14 | 2025-11-04 |
| Infigratinib in Subjects With GC or GEJ With FGFR2 Amplification or Other Solid Tumors With Other FGFR Alterations Infigratinib - Gastric Cancer, Gastroesophageal Junction Adenocarcinoma | Phase 2 | Unknown | LianBio LLC | 80 | 2020-05-13 | 2022-06-27 |
| Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE) ZYN002 - 22Q11.2 Deletion Syndrome | Phase 2 | Completed | Harmony Biosciences Management, Inc. | 20 | 2020-02-19 | 2026-03-09 |
Showing 20 of 60. Sign in to see all of them.
Acrocephalosyndactylia (17) · Cleft Palate (9) · DiGeorge Syndrome (9) · Noonan Syndrome (9) · Carcinoma, Non-Small-Cell Lung (6) · Carcinoma, Transitional Cell (5) · Neoplasms (5) · Cleft Lip (4) · 22q11 Deletion Syndrome (3) · Cholangiocarcinoma (3) · Colorectal Neoplasms (3) · Dentofacial Deformities (3) · Head and Neck Neoplasms (3) · Immunologic Deficiency Syndromes (3) · Retrognathia (3)
Congenital Abnormalities (60) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (60) · Musculoskeletal Abnormalities (60) · Musculoskeletal Diseases (59) · Bone Diseases (23) · Bone Diseases, Developmental (23) · Dysostoses (21) · Cardiovascular Abnormalities (19) · Cardiovascular Diseases (19) · Heart Defects, Congenital (19) · Heart Diseases (19) · Synostosis (18) · Craniosynostoses (17) · Genetic Diseases, Inborn (17)
pemigatinib (5) · Saline Solution (3) · Acetaminophen (2) · Bupivacaine (2) · Dexmedetomidine (2) · Drug Implants (2) · Melatonin (2) · Orthognathic Surgery (2) · Rogaratinib (2) · Standard of Care (2) · Thymus Extracts (2) · derazantinib (2) · vosoritide (2) · 5-((2-(6-Amino-9H-purin-9-yl) ethyl) amino)-1-pentanol (1) · Aminocaproic Acid (1)
Incyte Corporation (5) · Sumitomo Pharma Switzerland GmbH (3) · Assiut University (2) · Basilea Pharmaceutica (2) · Teresa Helsten, MD (2) · The University of Hong Kong (2) · Alexandria University (1) · Andrew Dauber (1) · Array Biopharma, now a wholly owned subsidiary of Pfizer (1) · Bambino Gesù Hospital and Research Institute (1) · Bausch Health Americas, Inc. (1) · Bayer (1) · BioMarin Pharmaceutical (1) · Cairo University (1) · Children's National Research Institute (1)
United States (32) · Germany (10) · France (9) · Spain (9) · Italy (8) · United Kingdom (7) · Japan (6) · South Korea (6) · Australia (5) · Canada (5) · Belgium (4) · Brazil (4) · China (4) · Egypt (4) · Poland (4)
M. Louise Markert, MD, PhD (3) · Teresa Helsten, MD (2) · Alfredo Addeo, MD (1) · Andrew Dauber, MS MMSc (1) · Baohui Han (1) · Crystal TY Lee, BDS (HKU) (1) · Daniela Francescato Veiga (1) · Daniela Y Tanikawa, MD (1) · Darnell Kaigler, DDS, PhD (1) · Dr. Evdokia Anagnostou (1) · Ekaterine Asatiani, MD (1)
PMID 18035553, PMID 18424759, PMID 18557726, PMID 19066739, PMID 20236866, PMID 17284531, PMID 12702512, PMID 17763894, PMID 18155964, PMID 18513295, PMID 19912236, PMID 20443823, PMID 20978268, PMID 21120277, PMID 21565561, PMID 21999260, PMID 23117614, PMID 23607606, PMID 23914737, PMID 28748333, PMID 30570744, PMID 31599480, PMID 32203698, PMID 35544727, PMID 35945547, PMID 36206041, PMID 37280688, PMID 37956738, PMID 38039831, PMID 38627238, PMID 38710951, PMID 38813446, PMID 39138436, PMID 40041314, PMID 41491634, PMID 41888667, PMID 42069961, PMID 42337043
2016 (3), 2019 (3), K12HD043494 (3), R01AI047040 (3), R01AI054843 (3), R56AI047040 (2), 2018 (1), R01DE010437 (1)