Clinical trials: Congenital Disorder
Retinitis Pigmentosa
Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell.
- Trial name
- NCT04604899
- Testing
- human retinal progenitor cells
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- jCyte, Inc
Thyroid Eye Disease
A Study of IBI311 in Subjects With Active Thyroid Eye Disease
This is a multicenter, randomized, double-masked, placebo-controlled Phase II/III study in subjects with active thyroid eye disease.
- Trial name
- NCT05795621
- Testing
- IBI311
- Where
- China
- Volunteers
- 115 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Study of Pancreatic Enzyme Product in Pediatric Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic…
- Trial name
- NCT00981214
- Testing
- EUR-1008 (APT-1008)
- Where
- 14 sites in United States
- Volunteers
- 19 people
- Sponsor
- Forest Laboratories
Haemophilia A
Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A. Participants will be treated with N8-GP.
- Trial name
- Pathfinder10 (NCT05082116)
- Testing
- turoctocog alfa pegol (N8-GP)
- Where
- 11 sites in China
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Dilated Cardiomyopathy
A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
The purpose of the study is to assess the safety, tolerability and the pharmacokinetics (PK) of AZD4063 after single dose administration in participants with phospholamban (PLN) R14del dilated cardiomyopathy.
- Trial name
- PULSE (NCT07241104)
- Testing
- AZD4063
- Where
- 4 sites in Netherlands
- Volunteers
- 23 people
- Sponsor
- AstraZeneca
Fabry Disease
A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00283933
- Testing
- migalastat HCl
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Amicus Therapeutics
Cystic Fibrosis
Long Term Administration of Inhaled Mannitol in Cystic Fibrosis
The purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis.
- Trial name
- NCT00630812
- Testing
- inhaled mannitol
- Where
- 53 sites in United States, Argentina and 5 more
- Volunteers
- 318 people
- Sponsor
- Syntara
Epidermolysis Bullosa
Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
- Trial name
- NCT02670330
- Testing
- SD-101-6.0 cream
- Where
- 30 sites in United States, Australia and 10 more
- Volunteers
- 152 people
- Sponsor
- Scioderm, Inc.
Congenital Bleeding Disorder, Haemophilia A
A Single Dose Trial of Recombinant Factor VIII (N8) in Japanese Subjects With Haemophilia A: An Extension to Trial NN7008-3543
This trial is conducted in Japan. The aim of this clinical trial is to investigate the pharmacokinetics (the effect of the investigated drug on the body) and safety of turoctocog alfa (recombinant factor VIII (N8)) in Japanese…
- Trial name
- NCT01238367
- Testing
- turoctocog alfa
- Where
- 6 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Novo Nordisk A/S
Duchenne Muscular Dystrophy (DMD)
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101.
- Trial name
- NCT06867107
- Testing
- SAT-3247
- Where
- 6 sites in United States, Australia
- Volunteers
- 30 people
- Sponsor
- Satellos Bioscience, Inc.
Pachyonychia Congenita
Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
A study to evaluate safety and efficacy of topical sirolimus to treat plantar keratoderma in adults with PC. Subjects may receive either placebo or treatment with at least 1 foot receiving topical sirolimus at some time.
- Trial name
- NCT02152007
- Testing
- 1% sirolimus cream (TD201 1%)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- TransDerm, Inc.
Homozygous Familial Hypercholesterolemia
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
The primary objectives of the study are: To evaluate the long-term safety and tolerability of evinacumab in patients with Homozygous Familial Hypercholesterolemia (HoFH) To evaluate the long-term safety and tolerability of…
- Trial name
- NCT03409744
- Testing
- evinacumab
- Where
- 38 sites in United States, Australia and 10 more
- Volunteers
- 116 people
- Sponsor
- Regeneron Pharmaceuticals
Wrinkle, Acne Scars - Mixed Atrophic and Hypertrophic
AV-23-001 AVAVA MIRIA Pilot Study
This non-significant risk study is intended to demonstrate the use of the MIRIA Skin Treatment System can show improvement in dermatologic conditions currently indicated for treatment with a laser (such as, but not limited to…
- Conditions
- Wrinkle, Acne Scars - Mixed Atrophic and Hypertrophic, Pigmented Lesions, Skin Laxity
- Trial name
- NCT06557434
- Testing
- MIRIA
- Where
- 2 sites in United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- AVAVA, Inc.
Netherton Syndrome
Natural History of Netherton Syndrome
The goal of this non-interventional study (NIS) is to collect real-world data to describe the natural history of Netherton Syndrome (NS).
- Trial name
- NCT05902663
- Where
- 4 sites in United States, Italy
- Volunteers
- 4 people
- Sponsor
- Boehringer Ingelheim
Atopic Dermatitis
Administration for Placebo Group and Evalution the Safety and Efficacy in Atopic Dermatitis Patients
A Multi-center, Open, Single-arm Clinical Trial to Evaluate the Safety and Efficacy of FURESTEM-AD inj. in Patients with Moderate to Severe Chronic Atopic Dermatitis Who Participated in a Placebo Group in K0102 Clinical Trial…
- Trial name
- NCT04087863
- Testing
- Not applicable(observational study)
- Where
- South Korea
- Volunteers
- 99 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Hemophilia A
Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A
The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A.
- Trial name
- Kids ALONG (NCT01458106)
- Testing
- BIIB031 (rFVIIIFc), FVIII (PK subgroup only)
- Where
- 23 sites in United States, Australia and 6 more
- Volunteers
- 71 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hyperlipoproteinemia Type II, Homozygous Familial Hypercholesterolemia
Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042)
This study will evaluate the safety and effect of anacetrapib on low-density lipoprotein-cholesterol (LDL-C) when added to ongoing lipid-lowering therapy.
- Trial name
- NCT01841684
- Testing
- Anacetrapib
- Volunteers
- 2 people
- Sponsor
- Merck Sharp & Dohme LLC
Growth, Colic
Safety and Tolerance Study of Alpha-Lactalbumin Enriched and Probiotic-Supplemented Infant Formula in Infants With Colic
The purpose of this study is to evaluate the nutritional adequacy, the digestive tolerance and the effect on colic of an alpha-lactalbumin-enriched and probiotic-supplemented infant formula.
- Trial name
- NCT00929292
- Testing
- Modilac Dahlia 1, Modilac 1
- Where
- 12 sites in France
- Volunteers
- 66 people
- Sponsor
- Sodilac
Achondroplasia
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life…
- Trial name
- NCT04035811
- Where
- 32 sites in United States, Argentina and 9 more
- Volunteers
- 271 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Niemann-Pick Disease, Type C
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.
- Trial name
- NCT02534844
- Testing
- Parts A/B: Adrabetadex
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 56 people
- Sponsor
- Mandos LLC
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