Clinical trials: Crigler-Najjar Syndrome
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Crigler-Najjar Syndrome Type I
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GT-UGT1A1-AAV8-02 in patients with Crigler-Najjar type 1 aged ≤10 years and requiring phototherapy.
- Trial name
- NCT06641154
- Testing
- GT-UGT1A1-AAV8-02
- Where
- 2 sites in Russia
- Volunteers
- 5 people
- Sponsor
- Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
Crigler-Najjar Syndrome
Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
Clinical trial rationale: CNS is an ultra-rare (<1/1 million newborns), autosomal recessive disorder of bilirubin conjugation caused by mutation in the gene coding for uridine 5'-diphosphate glucuronosyltransferase (UGT1A1), that…
- Trial name
- NCT06518005
- Testing
- Imlifidase, GNT0003
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Genethon
Urea Cycle Disorder, Crigler-Najjar Syndrome
HepaStem Long-Term Safety Registry
All patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences
- Conditions
- Urea Cycle Disorder, Crigler-Najjar Syndrome, Acute on Chronic Liver Failure
- Trial name
- PROLONGSTEM (NCT03343756)
- Where
- 29 sites in Belgium, Bulgaria and 3 more
- Volunteers
- 44 people
- Sponsor
- Cellaion SA
Crigler-Najjar Syndrome
Gene Therapy for Severe Crigler Najjar Syndrome
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy.
- Trial name
- CareCN (NCT03466463)
- Testing
- GNT0003
- Where
- 4 sites in France, Italy and 1 more
- Volunteers
- 17 people
- Sponsor
- Genethon
Metabolic Diseases
Hepatocyte Transplantation for Liver Based Metabolic Disorders
The purpose of this research study is to determine whether partial irradiation of the liver and liver cell transplantation can provide help for patients with life-threatening liver-based metabolic diseases who are unlikely to…
- Trial name
- NCT01345578
- Testing
- human hepatocyte transplantation, Preparative Radiation Therapy
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Ira Fox
Crigler-Najjar Syndrome
Gene Transfer Clinical Study in Crigler-Najjar Syndrome
This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT342 in subjects with Crigler-Najjar aged ≥1 year.
- Trial name
- VALENS (NCT03223194)
- Testing
- AT342
- Where
- 4 sites in United States, Israel and 1 more
- Volunteers
- 1 people
- Sponsor
- Audentes Therapeutics
Urea Cycle Disorders, Crigler Najjar Syndrome
Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)
The purpose of this study is to assess the safety and to appraise the efficacy of one cycle of Hepastem (Heterologous Human Adult Liver-derived Progenitor Cells, HHALPC) infusions in paediatric patients suffering from CN or UCD.
- Trial name
- HEP001 (NCT01765283)
- Testing
- HepaStem
- Where
- 11 sites in Belgium, France and 3 more
- Volunteers
- 20 people
- Sponsor
- Cellaion SA
Urea Cycle Disorders, Crigler Najjar Syndrome
Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
- Trial name
- NCT02051049
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Cellaion SA
Crigler-Najjar Syndrome
Clinical Assessment Study in Crigler-Najjar Syndrome
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.
- Trial name
- LUSTRO (NCT03078881)
- Where
- 4 sites in United States, Israel and 1 more
- Volunteers
- 8 people
- Sponsor
- Audentes Therapeutics
Crigler Najjar Syndrome
Immunity Against AAV in Crigler Najjar Patient
The proposed research protocol aims at addressing these points by pre-screening CN patients for their AAV serology in link with their medical history and current medical status.
- Trial name
- NCT02302690
- Testing
- Blood sample for immunology
- Where
- France
- Volunteers
- 49 people
- Sponsor
- Genethon
Crigler Najjar Syndrome, Children
New Phototherapy Device to Treat Patients With Crigler-Najjar Disease
Evaluating the efficacity of a new device phototherapy by comparing it with conventional phototherapy. Jaundice occurs in many newborns, and is, in most cases benign, However, owing to the potential neurotoxicity of unconjugated…
- Trial name
- DRAP (NCT02356978)
- Testing
- Arm1 homemade phototherapy treatment, DRAP
- Where
- France
- Volunteers
- 6 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Crigler-Najjar Syndrome
Orlistat Treatment of Crigler-Najjar Disease
The purpose of this study was to determine whether orlistat is effective in decreasing plasma unconjugated bilirubin levels in patients with Crigler-Najjar disease.
- Trial name
- NCT00461799
- Testing
- orlistat
- Where
- Netherlands
- Volunteers
- 16 people
- Sponsor
- University Medical Center Groningen