2 recruiting, 1 enrolling by invitation. 1 Not applicable, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Enrolling by invitation 1 |
| Phase 4 | 1 | Recruiting 1 |
Follow KBG syndrome: new trials by email Literature Review of KBG syndrome trials Research Report on KBG syndrome
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 |
|---|---|---|---|---|---|---|
| Palliative Care Needs of Children With Rare Diseases and Their Families Family Centered pediatric palliative care for family caregivers of children with rare diseases. - Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia | Not applicable | Enrolling by invitation | Children's National Research Institute | 480 | 2025-10-02 | 2026-01-28 |
| Methylphenidate in KBG Syndrome: N-of-1 Series Methylphenidate Hydrochloride, Placebo - Kbg Syndrome, ADHD - Combined Type | Phase 4 | Recruiting | Radboud University Medical Center | 15 | 2024-11-13 | 2026-08-11 |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2026-10-28 |
Cockayne Syndrome (2) · Rare Diseases (2) · Short Bowel Syndrome (2) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1) · Activated PI3K-delta Syndrome (1) · Addison Disease (1) · Alagille Syndrome (1) · Alstrom Syndrome (1) · Amnesia, Transient Global (1) · Aniridia (1) · Aniridia and Absent Patella (1) · Aniridia, Partial, with Unilateral Renal Agenesis and Psychomotor Retardation (1)
Abnormalities, Multiple (2) · Bone Diseases (2) · Bone Diseases, Developmental (2) · Cardiovascular Abnormalities (2) · Cardiovascular Diseases (2) · Chromosome Disorders (2) · Congenital Abnormalities (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Connective Tissue Diseases (2) · DNA Repair-Deficiency Disorders (2) · Digestive System Diseases (2) · Disease Attributes (2) · Dwarfism (2) · Ear Diseases (2) · Eye Diseases (2)
Methylphenidate (1)
Children's National Research Institute (1) · Radboud University Medical Center (1) · Sanford Health (1)
United States (2) · Netherlands (1)
R01HD117137 (1)