University College London - Great Ormond Street Institute of Child Health
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 7 Trials 
9 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Anikster, Yair
NCT06214507: Danon Disease Natural History Study

Recruiting
N/A
46
Europe, US, RoW
No intervention
Rocket Pharmaceuticals Inc.
Danon Disease
08/26
08/26
NCT06474104: Collection of Blood and Skin Samples From Patients With Primary Mitochondrial Diseases and Healthy Volunteers

Recruiting
N/A
100
RoW
Minovia Therapeutics Ltd.
Primary Mitochondrial Diseases
05/25
05/26
Rossano, Joseph
NCT03779711: Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Repair of Hypoplastic Left Heart Syndrome

Active, not recruiting
2b
95
US
Autologous (self) mononuclear cells derived from umbilical cord blood, Stage II Surgical repair
Timothy J Nelson, MD, PhD, University of Oklahoma, Children's Hospital of Philadelphia, Children's Hospital Los Angeles, Children's Hospital Colorado, Children's Hospitals and Clinics of Minnesota, Ochsner Health System, Children's of Alabama, Children's Hospital Medical Center, Cincinnati
Hypoplastic Left Heart Syndrome
05/22
02/26
NCT06092034: A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Active, not recruiting
2
12
Europe, US
RP-A501
Rocket Pharmaceuticals Inc.
Danon Disease
09/25
09/29
NCT04907526: Intramyocardial Injection of Autologous UCB-MNC During Fontan Surgery for SRV Dependent CHD

Active, not recruiting
1
30
US
Autologous mononuclear cells
Timothy J Nelson, MD, PhD, Mayo Clinic, University of Oklahoma, Children's Hospital of Philadelphia, Children's Hospital Los Angeles, Children's Hospitals and Clinics of Minnesota, Children's Hospital Colorado, Ochsner Health System, Children's of Alabama, Children's Hospital Medical Center, Cincinnati, The Hospital for Sick Children
Congenital Heart Disease, SRV Dependent
05/23
06/25
NCT03882437: Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

Active, not recruiting
1
7
US
RP-A501
Rocket Pharmaceuticals Inc.
Danon Disease
03/25
03/25
NCT05885412: A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Recruiting
1
9
US
RP-A601
Rocket Pharmaceuticals Inc.
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
09/25
09/26
NCT06214507: Danon Disease Natural History Study

Recruiting
N/A
46
Europe, US, RoW
No intervention
Rocket Pharmaceuticals Inc.
Danon Disease
08/26
08/26
Garuba, David
NCT06214507: Danon Disease Natural History Study

Recruiting
N/A
46
Europe, US, RoW
No intervention
Rocket Pharmaceuticals Inc.
Danon Disease
08/26
08/26

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