Clinical trials: Congenital Deformity
Hemophilia A
Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A
This study is to describe the safety and efficacy of Xyntha® during the usual care setting.
- Trial name
- NCT01790828
- Testing
- Xyntha : coagulation factor IIIV (recombinant)
- Where
- 4 sites in South Korea
- Volunteers
- 42 people
- Sponsor
- Pfizer
NF1 Mutation, Neurofibroma Plexiform
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1).
- Conditions
- NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)
- Trial name
- NCT06961565
- Testing
- PAS-004 Tablets, PAS-004 Capsule
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 56 people
- Sponsor
- Pasithea Therapeutics Corp.
Hereditary Angioedema (HAE)
Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With Hereditary Angioedema Who Have Completed the CSL312_3003 Study
This protocol for post study access allows pediatric participants (2-11 years old at the time of consent) with HAE who have completed study CSL312_3003 (NCT05819775) to continue treatment with CSL312 for routine prevention of HAE…
- Trial name
- NCT07159464
- Testing
- CSL312
- Sponsor
- CSL Behring
Atopic Dermatitis
Study of Abrocitinib Compared With Dupilumab in Adults With Moderate to Severe Atopic Dermatitis on Background Topical Therapy
This is a randomized, double-blind, double-dummy, active-controlled, multi-center study to assess the efficacy and safety of abrocitinib 200 mg (2 x 100 mg tablets) administered orally QD compared with dupilumab 300 mg…
- Trial name
- NCT04345367
- Testing
- Abrocitinib 200 mg, Dupilumab 300 mg
- Where
- 151 sites in United States, Australia and 13 more
- Volunteers
- 727 people
- Sponsor
- Pfizer
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Facioscapulohumeral Muscular Dystrophy, FSHD
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
- Trial name
- NCT07435129
- Testing
- Apitegromab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Scholar Rock, Inc.
Dermatitis, Atopic
Drug Drug Interaction Study Evaluating the Effect of Fluvoxamine or Fluconazole on PK and Safety of PF-04965842.
A Phase 1, Open Label, Parallel-Cohort, Randomized, Fixed Sequence Study To Evaluate The Pharmacokinetic Drug Drug Interaction Between PF-04965842 and Fkuvoxamine (cohort 1) or Fluconazole (Cohort 2) in healthy subjects.
- Trial name
- NCT03634345
- Testing
- Period 1 - Day 1: PF-04965842 administered, Period 2: Cohort 1: Fluvoxamine & PF-04965842, Period 2: Cohort 2: Fluconazole & PF-04965842
- Where
- Belgium
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Pfizer
Thyroid Eye Disease, Graves' Orbitopathy
Treatment of Graves' Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis With Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study
The overall objective is to investigate the efficacy, tolerability, and safety of teprotumumab (a fully human monoclonal antibody [mAb] inhibitor of the insulin-like growth factor-1 receptor [IGF-1R]) administered once every 3…
- Trial name
- OPTIC (NCT03298867)
- Testing
- Teprotumumab
- Where
- 14 sites in United States, Germany and 1 more
- Volunteers
- 83 people
- Sponsor
- Amgen
Cystic Fibrosis Lung
4D-710 in Adult Patients With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Trial name
- CF (NCT05248230)
- Testing
- 4D-710
- Where
- 20 sites in United States
- Volunteers
- 30 people
- Sponsor
- 4D Molecular Therapeutics
Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
A Study on Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in Children, Teenagers and Adults in Portugal
The main aim of this study is to learn about the percentage of persons diagnosed with Dravet Syndrome (DS) and Lennox-Gastaut-Syndrome (LGS) in 2022 and of persons newly diagnosed in 2021 and 2022 compared to the overall…
- Trial name
- DRALEGA-PT (NCT06395792)
- Testing
- No Intervention
- Sponsor
- Takeda
Erythropoietic Protoporphyria
Phase III Confirmatory Study in Erythropoietic Protoporphyria
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Between 75 and 100 eligible patients will be enrolled.
- Trial name
- NCT01605136
- Testing
- Afamelanotide
- Where
- 7 sites in United States
- Volunteers
- 93 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Thyroid Eye Disease
An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)
This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, VRDN-003, in participants with active Thyroid Eye Disease (TED).
- Trial name
- REVEAL-1 (NCT06625411)
- Testing
- VRDN-003
- Where
- 48 sites in United States, Germany and 3 more
- Volunteers
- 132 people
- Sponsor
- Viridian Therapeutics, Inc.
Severe Hemophilia A
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant…
- Trial name
- NCT04046848
- Testing
- OCTA101
- Where
- Bulgaria
- Volunteers
- 36 people
- Sponsor
- Octapharma
Hemophilia A, Hemophilia B
Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors.
- Trial name
- MATCHBOX (NCT01921855)
- Testing
- BAY Factor VII (BAY86-6150)
- Where
- 4 sites in Poland, South Africa and 1 more
- Volunteers
- 16 people
- Sponsor
- Bayer
Hydrocephalus, Nervous System Diseases
Study of Shunt Flow Sensor Accuracy in Extra-ventricular Drains.
The study hypothesis is that an ultrasonic flow sensor can accurately measure flow in hydrocephalic shunts. The ultrasonic sensor will measure cerebrospinal fluid drainage in hydrocephalus patients with external ventriculostomies…
- Conditions
- Hydrocephalus, Nervous System Diseases, Infant, Newborn Diseases
- Trial name
- NCT01108965
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Transonic Systems Inc.
Cystic Fibrosis
Study of Denufosol Inhalation Solution in Patients With Mild to Moderate Cystic Fibrosis Lung Disease
The purpose of this trial is to evaluate the effects of a single dose of denufosol versus placebo on mucociliary clearance in patients with mild to moderate CF lung disease
- Trial name
- NCT00425165
- Testing
- denufosol tetrasodium (INS37217) Inhalation Solution
- Volunteers
- 6 people
- Sponsor
- Merck Sharp & Dohme LLC
ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant…
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- Trial name
- NCT05030831
- Testing
- INZ-701
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Inozyme Pharma
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
Rheumatoid Arthritis, Osteoarthritis
A Single Centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Ceramic-on-ceramic Bearing in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the Pinnacle™ Cup with a ceramic-on-ceramic bearing combination in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00872222
- Testing
- Pinnacle™ Acetabular System
- Where
- Czechia
- Volunteers
- 98 people
- Sponsor
- DePuy International
Atopic Dermatitis
Safety, Tolerance and Pharmacokinetics Clinical Study of VC005 in Healthy Subjects and Patients with Mild to Moderate Atopic Dermatitis
This study is a single center, randomized, double-blind, Vehicle controlled,, single and multiple dose clinical study.
- Trial name
- NCT06009094
- Testing
- VC005
- Where
- China
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Jiangsu vcare pharmaceutical technology co., LTD
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