Clinical trials: Congenital Anomaly
Hepatic Encephalopathy, Urea Cycle Disorders
A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group
The purpose of this study is to determine the safety and tolerability of GT4P administered orally as a single dose, and twice daily for 7 consecutive days, to subjects with hepatic impairment with cirrhosis (Child-Pugh scores of…
- Trial name
- NCT00986895
- Testing
- HPN-100
- Where
- 2 sites in Ukraine
- Volunteers
- 32 people
- Sponsor
- Amgen
Hemophilia A
A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480)
- Trial name
- NCT03061201
- Testing
- SB-525 (PF-07055480)
- Where
- 22 sites in United States
- Volunteers
- 13 people
- Sponsor
- Pfizer
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
Congenital Adrenal Hyperplasia
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.
- Trial name
- NCT04783181
- Testing
- AAV BBP-631
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Adrenas Therapeutics Inc
Cystic Fibrosis, Advanced Lung Disease
Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation
- Trial name
- NCT02390219
- Testing
- Lumacaftor, Ivacaftor
- Where
- 6 sites in United States
- Volunteers
- 46 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Atopic Dermatitis, Sleep Disturbance
A Study to Assess the Effect of Upadacitinib in Atopic Dermatitis-induced Sleep Disturbance in Adults With Moderate to Severe Atopic Dermatitis
Many people with atopic dermatitis (AD) experience sleep disturbances. Greater sleep disturbances are associated with greater burden including increased sick days and impaired cognition.
- Trial name
- Sleep Up (NCT06390722)
- Testing
- Upadacitinib
- Sponsor
- AbbVie
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant
Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation
The purpose of this study is to characterize the disease progression of confirmed OPA1 mutation-associated autosomal dominant optic atrophy (ADOA) by evaluating the changes in ocular structural and functional outcomes.
- Conditions
- Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
- Trial name
- NCT06140329
- Where
- 7 sites in United States, Australia and 4 more
- Volunteers
- 1 people
- Sponsor
- PYC Therapeutics
Hemophilia A
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older.
- Trial name
- HAVEN 3 (NCT02847637)
- Testing
- Emicizumab, Factor VIII (FVIII)
- Where
- 39 sites in United States, Australia and 12 more
- Volunteers
- 152 people
- Sponsor
- Hoffmann-La Roche
Growth Hormone Deficiency, Turner Syndrome
Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
The purpose of this study is to evaluate the long-term safety and effectiveness of growth hormone (Eutropin Inj./Eutropin plus Inj.) treatment with GHD (Growth Hormone Deficiency), TS (Turner Syndrome),CRF (Chronic Renal…
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- LGS (NCT01604395)
- Where
- South Korea
- Volunteers
- 6,000 people
- Sponsor
- LG Chem
Phenylketonurias, PKU
Evaluation of the Express Plus Range
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM.
- Conditions
- Phenylketonurias, PKU, Homocystinuria, Maple Syrup Urine Disease
- Trial name
- express plus (NCT05051657)
- Testing
- PKU express plus
- Where
- 6 sites in United Kingdom
- Volunteers
- 28 people
- Sponsor
- Vitaflo International, Ltd
Cystic Fibrosis
Rollover Study of VX-770 in Cystic Fibrosis Subjects
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF).
- Trial name
- NCT01117012
- Testing
- Ivacaftor
- Where
- 58 sites in United States, Australia and 6 more
- Volunteers
- 192 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
Fed
Bioequivalence Study of Olanzapine Orally Disintegrating Tablets, 5 mg Under Fed Condition
The purpose of this study is to compare the single oral dose bioequivalence of olanzapine 5 mg OD tablets with Zyprexa Zydis 5 mg tablets under fed conditions and to monitor adverse events and ensure safety of subjects.
- Trial name
- NCT01503450
- Testing
- Olanzapine OD Tablets 5 mg
- Where
- India
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with…
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05176717
- Testing
- QR-421a
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Laboratoires Thea
Osteogenesis Imperfecta
Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta
This is a randomized, open label intra-patient dose escalation study to evaluate safety and tolerability, pharmacokinetics, and pharmacodynamics of BPS804 in adults with osteogenesis imperfecta (OI).
- Trial name
- NCT01417091
- Testing
- BPS804
- Where
- 6 sites in United States, Belgium and 2 more
- Volunteers
- 10 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Pelizaeus-Merzbacher Disease
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
- Trial name
- NCT06150716
- Testing
- ION356
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Non-squamous Metastatic Non-Small-Cell Lung Carcinoma
NIS to Examine the Effectiveness of TDC in Patients With Metastatic Non-squamous NSCLC and High-risk Genetic Alterations
This prospective, multicenter, non-interventional study (NIS) in Germany aims to collect real-life data of patients with non-squamous (NSQ) metastatic non-small cell lung cancer (mNSCLC) (incl.
- Trial name
- NAUTIC (NCT06494540)
- Where
- 30 sites in Germany
- Volunteers
- 600 people
- Sponsor
- AstraZeneca
Hereditary Angioedema, Pediatric
Berotralstat Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic…
- Trial name
- APeX-P (NCT05453968)
- Testing
- Berotralstat
- Where
- 15 sites in Austria, Canada and 8 more
- Volunteers
- 29 people
- Sponsor
- BioCryst Pharmaceuticals
Cystic Fibrosis, Steatorrhea
An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have…
- Trial name
- NCT00217204
- Testing
- Pancrelipase microtablets
- Volunteers
- 18 people
- Sponsor
- McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
Hemophilia B
Registry For Patients Treated With BeneFix In Usual Care Setting In Germany
The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.
- Trial name
- NCT00714415
- Testing
- BeneFIX
- Where
- 21 sites in Austria, Germany
- Volunteers
- 80 people
- Sponsor
- Pfizer
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