Clinical trials: X-Linked
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also…
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- Trial name
- NCT06805695
- Testing
- No Intervention
- Where
- 4 sites in United States, France and 1 more
- Volunteers
- 13 people
- Sponsor
- iECURE, Inc.
Mobility Impairment, Clinical Workflow Optimization
Deployment and Evaluation of Humanoid Robotics in Coalition-Aligned Clinical Care Settings
This clinical trial evaluates the deployment of humanoid robotics in coalition-aligned healthcare settings. The study measures biometric performance, ESG compliance, and operational impact across patient care, sanitation, and…
- Conditions
- Mobility Impairment, Clinical Workflow Optimization, Sanitation Protocol Compliance, ESG-linked Healthcare Robotics Deployment
- Trial name
- HEALTH (NCT07214896)
- Testing
- Skin Cell Dip, Stem Cell Dip, Wage Token Distribution
- Where
- 4 sites in United States, Germany and 1 more
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Truway Health, Inc.
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Expanded Access Program of Bitopertin For Participants With EPP or XLP
The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.
- Trial name
- NCT07603401
- Testing
- DISC-1459
- Where
- 8 sites in United States
- Sponsor
- Disc Medicine, Inc
Metastatic Castration Resistant Prostate Cancer
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)
Himalayas-1 is a randomized, open-label, global, multicenter phase 3 study evaluating whether the combination of rinzimetostat with darolutamide is more effective compared to physician's choice of control; ARPI (darolutamide or…
- Trial name
- Himalayas-1 (NCT07723248)
- Testing
- Rinzimetostat, Darolutamide, Enzalutamide
- Where
- 15 sites in United States, Puerto Rico
- Volunteers
- 600 people
- Sponsor
- ORIC Pharmaceuticals
X-linked Myotubular Myopathy
A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07876804
- Testing
- ASP2957
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations
Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations
This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, or CDC42 mutations.
- Trial name
- MASter-1 (NCT04641442)
- Testing
- MAS825
- Where
- 12 sites in United States, Czechia and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Study of Bitopertin in Participants With EPP or XLP (APOLLO)
The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older.
- Trial name
- APOLLO (NCT06910358)
- Testing
- DISC-1459
- Where
- 27 sites in United States, Australia and 10 more
- Volunteers
- 183 people
- Sponsor
- Disc Medicine, Inc
Muscular Disorders, Atrophic, Muscular Diseases
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Trial name
- NCT07250737
- Testing
- delpacibart zotadirsen
- Where
- 22 sites in United States
- Sponsor
- Avidity Biosciences, Inc.
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Characteristics and Impacts of X-linked Hypohidrotic Ectodermal Dysplasia (XLHED) in Boys: An Observational International Study
This is an observational, multicentre, international study over a 2-year follow-up period. The aim of this study is to understand how XLHED affects the lives of young male patients and their families over time.
- Trial name
- EdeReaLife (NCT07096206)
- Where
- 2 sites in France, Germany
- Volunteers
- 27 people
- Sponsor
- Pierre Fabre Medicament
Prostate Cancer
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer.
- Trial name
- ANDROMEDA (NCT07336446)
- Testing
- AZD9750, AZD5305
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 6 people
- Sponsor
- AstraZeneca
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Trial name
- NCT06255782
- Testing
- ECUR-506
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 20 people
- Sponsor
- iECURE, Inc.
X-Linked Myotubular Myopathy
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness.
- Trial name
- EXCEL (NCT06581146)
- Testing
- No Intervention
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Astellas Gene Therapies
Metastatic Castration-resistant Prostate Cancer
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer
The purpose of this study is to compare the efficacy and safety of BMS-986365 versus the investigator's choice of therapy in participants with Metastatic Castration-resistant Prostate Cancer.
- Trial name
- rechARge (NCT06764485)
- Testing
- BMS-986365, Enzalutamide, Abiraterone
- Where
- 227 sites in United States, Argentina and 24 more
- Volunteers
- 960 people
- Sponsor
- Celgene
Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes.
- Trial name
- NCT07523581
- Testing
- Exaluren
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 24 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
X-linked Retinoschisis
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 5 years of age with RS1-associated X-linked retinoschisis (XLRS).
- Trial name
- LIGHTHOUSE (NCT05878860)
- Testing
- ATSN-201
- Where
- 21 sites in United States, Canada and 1 more
- Volunteers
- 97 people
- Sponsor
- Atsena Therapeutics Inc.
Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of two different treatment combinations of tulmimetostat in participants with de novo or recurrent metastatic hormone-sensitive prostate cancer…
- Trial name
- TulmiSTAR-02 (NCT07190300)
- Testing
- Tulmimetostat, Darolutamide, Abiraterone
- Where
- 31 sites in United States, Australia and 12 more
- Volunteers
- 181 people
- Sponsor
- Novartis Pharmaceuticals
X-Linked Retinitis Pigmentosa
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.
- Trial name
- NCT04312672
- Testing
- AAV5-hRKp.RPGR
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 42 people
- Sponsor
- MeiraGTx Ocular UK Ltd
X-Linked Retinitis Pigmentosa
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Trial name
- NCT04794101
- Testing
- Genetic: AAV5-hRKp.RPGR Intermediate Dose, Genetic: AAV5-hRKp.RPGR Low Dose
- Where
- 28 sites in United States, Belgium and 9 more
- Volunteers
- 97 people
- Sponsor
- MeiraGTx Ocular UK Ltd
X-Linked Retinitis Pigmentosa
A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
- Trial name
- NCT06646289
- Testing
- AAV5-hRKp.RPGR, No intervention (Follow-Up assessment)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 24 people
- Sponsor
- MeiraGTx Ocular UK Ltd