Clinical trials: Congenital Deformity
Congenital Bleeding Disorder, Haemophilia A
Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A
This trial is conducted globally. The aim of the trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in subjects with Haemophilia A.
- Trial name
- pathfinder™2 (NCT01480180)
- Testing
- turoctocog alfa pegol
- Where
- 93 sites in United States, Australia and 22 more
- Volunteers
- 186 people
- Sponsor
- Novo Nordisk A/S
Hereditary Hemorrhagic Telangiectasia (HHT)
A Trial of TER-1754 in Patients With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1a/1b, multicenter study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of TER-1754 (a novel AKT1 inhibitor) in patients with HHT.
- Trial name
- NCT07255846
- Testing
- TER-1754
- Where
- United States
- Sponsor
- Terremoto Biosciences Inc.
Skin Laxity
Feasibility Study: Compare the Effectiveness Between Two Pain Medications When Used Prior to Ultherapy™ Treatments
This study will compare the effectiveness of two pain medications for reducing discomfort during an Ultherapy treatment.
- Trial name
- NCT01708473
- Testing
- Advil, Lortab, Ulthera System Treatment
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
Leukemia
Cytarabine With or Without VNP40101M in Treating Patients With Relapsed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as cytarabine and VNP40101M, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00112554
- Testing
- cytarabine, laromustine
- Where
- 62 sites in United States, Belgium and 8 more
- Volunteers
- 420 people
- Sponsor
- Vion Pharmaceuticals
Gout
Extension Study of ABP-671 in Participants With Gout
The primary objectives of this study are to assess the safety, tolerability, and efficacy of ABP-671 in lowering serum uric acid (sUA) in participants with gout who roll over from Study ABP-671-301 after they complete the…
- Trial name
- NCT06276556
- Testing
- ABP-671, Allopurinol
- Where
- 54 sites in United States, Australia and 3 more
- Volunteers
- 43 people
- Sponsor
- Atom Therapeutics Co., Ltd
Skin Laxity, Lipodystrophy
CONFORM: Rotational Fractional Resection for Submental Contouring
This study will evaluate the safety and efficacy of rotational fractional resection (RFR) to improve neck contouring. RFR is used to remove loose skin and fat.
- Trial name
- NCT03407313
- Testing
- Rotational fractional resection (1.5mm diameter device)
- Where
- 6 sites in United States
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Recros Medica, Inc.
Hemophilia A
BAX 855 PK-guided Dosing
1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%) 2.
- Trial name
- PROPEL (NCT02585960)
- Testing
- PEGylated Recombinant Factor VIII
- Where
- 83 sites in United States, Australia and 20 more
- Volunteers
- 135 people
- Sponsor
- Baxalta now part of Shire
Hereditary Angioedema (HAE)
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Trial name
- NCT02093923
- Testing
- DX-2930
- Where
- 14 sites in United States, Italy and 1 more
- Volunteers
- 38 people
- Sponsor
- Shire
Factor XIII Deficiency
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00885742
- Testing
- FXIII Concentrate (Human)
- Where
- 23 sites in United States, Spain
- Volunteers
- 41 people
- Sponsor
- CSL Behring
Atopic Dermatitis
A Phase 2 Study of E6005 in Patients With Atopic Dermatitis
The purpose of this study is to evaluate the efficacy and safety profiles of E6005 ointment in patients with atopic dermatitis compared to vehicle. The pharmacokinetic profile after topical application of E6005 is also assessed.
- Trial name
- NCT01461941
- Testing
- E6005, E6005 ointment (vehicle)
- Where
- 10 sites in Japan
- Volunteers
- 78 people
- Sponsor
- Organon and Co
Atopic Dermatitis, Eczema, Atopic
A Study in Subjects With Moderate Atopic Dermatitis
This is a randomized, double-blind, vehicle-controlled study to evaluate the safety and efficacy of ALX-101 Gel 1.5% and 5% and a matching ALX-101 Gel Vehicle when applied topically twice daily for 42 days to adult and adolescent…
- Trial name
- NCT03175354
- Testing
- ALX-101 Gel 1.5% and ALX-101 Gel Vehicle, ALX-101 Gel 5% and ALX-101 Gel Vehicle
- Where
- 35 sites in United States
- Volunteers
- 209 people
- Sponsor
- Ralexar Therapeutics, Inc.
Alport Syndrome, Focal Segmental Glomerulosclerosis
Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis
This is a Phase 2, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary…
- Trial name
- NCT05267262
- Testing
- R3R01
- Where
- 22 sites in United States, Belgium and 4 more
- Volunteers
- 43 people
- Sponsor
- River 3 Renal Corp.
Hemophilia A
Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures
The purpose of the study is to evaluate the efficacy and safety of BAX 855 in severe hemophilia A previously treated (PTP) males, 12 to 65 years of age who are undergoing elective surgical or other invasive procedures.
- Trial name
- NCT01913405
- Testing
- PEGylated Recombinant factor VIII (rFVIII)
- Where
- 22 sites in United States, Bulgaria and 7 more
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Atopic Dermatitis
A Study of Lebrikizumab (LY3650150) in Participants With Moderate-to-Severe Atopic Dermatitis
The purpose of this study is to evaluate the safety and efficacy of lebrikizumab compared with placebo in participants with moderate-to-severe atopic dermatitis.
- Trial name
- NCT03443024
- Testing
- Lebrikizumab
- Where
- 58 sites in United States
- Volunteers
- 280 people
- Sponsor
- Eli Lilly and Company
Hemophilia A
Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq
Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe…
- Trial name
- PREVAIL (NCT03344003)
- Testing
- Wilate or Nuwiq
- Where
- 3 sites in Canada
- Volunteers
- 14 people
- Sponsor
- Octapharma
Glycogen Storage Disease Type Ia
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.
- Trial name
- NCT06636383
- Testing
- No Intervention
- Where
- 19 sites in United States, Brazil and 7 more
- Volunteers
- 140 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in…
- Trial name
- NCT05911763
- Testing
- Efanesoctocog Alfa BIVV001
- Where
- 28 sites in United States
- Volunteers
- 200 people
- Sponsor
- Sanofi
Atopic Dermatitis
Effectiveness of Upadacitinib and Biologics for the Achievement of Minimal Disease Activity in Adults With Moderate-to-Severe Atopic Dermatitis (RAMpUp)
Atopic dermatitis (AD) is a common, chronic, inflammatory skin disease with a significant impact on patients' health-related quality of life.
- Trial name
- RAMpUp (NCT07741838)
- Volunteers
- 900 people
- Sponsor
- AbbVie
Hemophilia A
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
- Trial name
- NCT06111638
- Testing
- Single dose intravenous injection of BBM-H803
- Where
- 10 sites in China
- Volunteers
- 55 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
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