long term follow up of children
Showing 1 - 25 of >10,000
Long Term Follow-Up for RGX-111
Enrolling by invitation
- Mucopolysaccharidosis I
- No Intervention
-
Orange, California
- +2 more
Oct 25, 2023
After Adoptive Transfer of Genetically Modified Cell Products
Recruiting
- Relapsed Hematologic Malignancy
- Refractory Hematologic Malignancy
-
Memphis, TennesseeSt. Jude Children's Research Hospital
Feb 6, 2023
Duchenne Muscular Dystrophy Trial in Los Angeles, Columbus (delandistrogene moxeparvovec)
Not yet recruiting
- Duchenne Muscular Dystrophy
- delandistrogene moxeparvovec
-
Los Angeles, California
- +1 more
Jul 21, 2023
Acute Kidney Injury, Multi Organ Failure, Children, Only Trial in Stockholm
Enrolling by invitation
- Acute Kidney Injury
- +2 more
-
Stockholm, SwedenPediatric Perioperative Medicine and Intensive Care
Nov 28, 2022
Study of Patients With MPS IIIA Treated With ABO-102
Enrolling by invitation
- Mucopolysaccharidosis III-A
- ABO-102
-
Columbus, Ohio
- +2 more
Nov 8, 2022
(LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Active, not recruiting
- Hemophilia B
- +2 more
- SB-318
- +2 more
-
Oakland, California
- +4 more
Jan 30, 2023
Telomere Biology Disorders With Bone Marrow Failure Trial in Cincinnati (EXG34217)
Not yet recruiting
- Telomere Biology Disorders With Bone Marrow Failure
- EXG34217
-
Cincinnati, OhioCincinnati Children's Hospital
May 18, 2023
Subjects Previously Administered HMI 102
Enrolling by invitation
- Phenylketonuria
- PAH Deficiency
- HMI-102
-
Boston, Massachusetts
- +1 more
Jun 28, 2022
Growth and Development Trial in Belgium, Russian Federation, United States (Everolimus, Envirolimus drug class as prescribed by
Active, not recruiting
- Growth and Development
- Everolimus
- Envirolimus drug class as prescribed by Physician
-
Los Angeles, California
- +5 more
Dec 5, 2022
Long Term Follow-Up Gene Therapy Study for XLRP RPGR
Recruiting
- X-Linked Retinitis Pigmentosa
- AAV-RPGR
-
Boston, Massachusetts
- +3 more
Dec 5, 2022
The Alberta BLOOM Long Term Follow Up Study
Terminated
- Infant, Premature, Diseases
- +5 more
-
Calgary, Alberta, CanadaUniversity of Calgary
Mar 29, 2022
Study of Patients With MPS IIIB Treated With ABO-101
Terminated
- Mucopolysaccharidosis III-B
- ABO-101
-
Columbus, Ohio
- +2 more
May 25, 2022
The Long-term Consequences of Neonatal Encephalopathy in
Not yet recruiting
- Neonatal Encephalopathy
- +3 more
- Therapeutic hypothermia
-
Montreal, Quebec, Canada
- +1 more
Feb 23, 2023
Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug
Enrolling by invitation
- Cerebral Adrenoleukodystrophy (CALD)
- +2 more
- Lenti-D
-
Los Angeles, California
- +10 more
Nov 4, 2022
Long Term Follow Up Protocol for NiCord®/CordIn™ Patients
Active, not recruiting
- Patients Transplanted With NiCord/CordIn (Omidubicel)
- NiCord®/CordIn™ (omidubicel)
-
Oakland, California
- +9 more
Oct 31, 2022
Sickle Cell Disease Treated With Ex Vivo Gene Therapy
Enrolling by invitation
- Sickle Cell Disease
- Safety and efficacy assessments
-
Birmingham, Alabama
- +11 more
Oct 31, 2022
CLN6 Batten Disease Subjects Following Gene Transfer
Active, not recruiting
- CLN6
- Batten Disease
- AT-GTX-501
-
Columbus, OhioNationwide Children's Hosptial
Dec 17, 2021
Patients Who Have Received Gene Therapy Or Gene Marked Products
Recruiting
- Severe Combined Immunodeficiency
- +4 more
- Venipuncture
-
Memphis, TennesseeSt. Jude Children's Research Hospital
Dec 10, 2021
Preterm Birth, Child Development Trial in Columbus (Docosahexaenoic Acid + Arachidonic Acid (DHA+AA), Placebo)
Enrolling by invitation
- Preterm Birth
- Child Development
- Docosahexaenoic Acid + Arachidonic Acid (DHA+AA)
- Placebo
-
Columbus, OhioNationwide Children's Hospital
Jan 10, 2022
Study for Patients Who Participated in GOAL Trial (GOAL-Post)
Recruiting
- Rheumatic Heart Disease
- Penicillin G Benzathine
-
Kampala, UgandaUganda Heart Institute
Feb 19, 2022
A Long-term Follow-up Study in Subjects Who Received CTX001
Enrolling by invitation
- Beta-Thalassemia
- +6 more
- CTX001
-
New York, New York
- +12 more
Nov 18, 2022
Long-term Follow-Up for RGX-121
Enrolling by invitation
- Mucopolysaccharidosis II
- Long-term Follow-Up
-
Pittsburgh, PennsylvaniaChildren's Hospital of Pittsburgh - UPMC: Program for Neurodevel
Aug 9, 2021
Prader-Willi Syndrome Trial in Toulouse (Follow-up study of the treated cohort, Follow-up study of the untreated cohort)
Recruiting
- Prader-Willi Syndrome
- Follow-up study of the treated cohort
- Follow-up study of the untreated cohort
-
Toulouse, FranceCentre de réfrence Prader-Willi, Hospital of infants
Sep 27, 2021