9 active not recruiting. 9 Phase 2, 2 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 9 | Active not recruiting 9 |
| Phase 1 | 2 | Active not recruiting 2 |
| Phase 3 | 1 | Active not recruiting 1 |
Follow Chromosome Disorders: new trials by email Literature Review of Chromosome Disorders trials Research Report on Chromosome Disorders
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 |
|---|---|---|---|---|---|---|
| A Study of Setmelanotide in Patients With Prader-Willi Syndrome Setmelanotide - Prader-Willi Syndrome, Obesity | Phase 2 | Active not recruiting | Rhythm Pharmaceuticals, Inc. | 18 | 2025-03-04 | 2026-02-12 |
| Clemastine Treatment in Individuals With Williams Syndrome Open Label Clemastine with a blinded randomize withdrawal - Williams Syndrome | Phase 2 | Active not recruiting | Sheba Medical Center | 30 | 2024-04-01 | 2026-03-06 |
| A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment Part A: RM-718 or placebo (matched to specific RM-718 dose cohort), Part B: RM-718 or placebo (matched to specific RM-718 dose cohort), Part C: RM-718 - Hypothalamic Obesity, Prader-Willi Syndrome | Phase 1 / Phase 2 | Active not recruiting | Rhythm Pharmaceuticals, Inc. | 111 | 2024-03-05 | 2026-10-09 |
| Medications for Obstructive Sleep Apnea to Improve Cognition in Children With Down Syndrome Atomoxetine and Oxybutynin (ato-oxy) - Obstructive Sleep Apnea, Down Syndrome | Phase 2 | Active not recruiting | University of Arizona | 36 | 2023-09-29 | 2026-04-07 |
| A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome Lonapegsomatropin, Somatropin - Turner Syndrome | Phase 2 | Active not recruiting | Ascendis Pharma Endocrinology Division A/S | 48 | 2023-02-15 | 2026-05-13 |
| Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge Baclofen, Memantine, Roflumilast - Fragile X Syndrome | Phase 2 | Active not recruiting | Children's Hospital Medical Center, Cincinnati | 45 | 2022-09-08 | 2025-12-23 |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome ION582 - Angelman Syndrome | Phase 1 / Phase 2 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 71 | 2021-12-22 | 2026-08-20 |
| Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS ZYN002 - Cannabidiol Transdermal Gel - Fragile X Syndrome | Phase 2 / Phase 3 | Active not recruiting | Zynerba Pharmaceuticals, Inc. | 476 | 2018-11-09 | 2026-05-27 |
| ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016 Daunorubicin, Prednisolone, Vincristine - Down Syndrome, Acute Lymphoblastic Leukemia | Phase 2 | Active not recruiting | National Hospital Organization Nagoya Medical Center | 60 | 2017-04-18 | 2026-05-11 |
Down Syndrome (2) · Fragile X Syndrome (2) · Obesity (2) · Prader-Willi Syndrome (2) · Angelman Syndrome (1) · Hyperphagia (1) · Neoplasms (1) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (1) · Sexual Infantilism (1) · Sleep Apnea, Obstructive (1) · Turner Syndrome (1) · Williams Syndrome (1)
Congenital Abnormalities (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Nervous System Diseases (8) · Intellectual Disability (7) · Neurobehavioral Manifestations (7) · Neurologic Manifestations (7) · Abnormalities, Multiple (5) · Imprinting Disorders (3) · Sex Chromosome Disorders (3) · Body Weight (2) · Cardiovascular Diseases (2) · Disorders of Sex Development (2) · Endocrine System Diseases (2)
Atomoxetine Hydrochloride (1) · Baclofen (1) · Cyclophosphamide (1) · Cytarabine (1) · Daunorubicin (1) · Epirubicin (1) · Human Growth Hormone (1) · Hydrocortisone (1) · Memantine (1) · Mercaptopurine (1) · Methotrexate (1) · Prednisolone (1) · Roflumilast (1) · Vincristine (1) · lonapegsomatropin (1)
Rhythm Pharmaceuticals, Inc. (2) · Ascendis Pharma Endocrinology Division A/S (1) · Children's Hospital Medical Center, Cincinnati (1) · Ionis Pharmaceuticals, Inc. (1) · National Hospital Organization Nagoya Medical Center (1) · Sheba Medical Center (1) · University of Arizona (1) · Zynerba Pharmaceuticals, Inc. (1)
United States (7) · Australia (2) · Israel (2) · United Kingdom (2) · France (1) · Hong Kong (1) · Italy (1) · Japan (1) · Malaysia (1) · New Zealand (1) · Singapore (1) · Taiwan (1) · Thailand (1)
Allen Yeoh, MBBS (1) · Craig A. Erickson, M.D. (1) · David Meeker (1) · David Meeker, MD (1)
R33HL151254 (1), U54HD104461 (1)