Clinical trials: Congenital Disorder
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study.
- Trial name
- NCT03363321
- Testing
- PF-06741086
- Where
- 8 sites in United States, Brazil and 5 more
- Volunteers
- 20 people
- Sponsor
- Pfizer
Thyroid Eye Disease (TED)
MHB018A Treatment in Patients With Chronic Thyroid Eye Disease
The primary objective of this study is to investigate the efficacy, safety, and tolerability of MHB018A, a humanized anti-IGF1R antibody, administered Q4W for 6 months, in comparison to placebo, in the treatment of participants…
- Trial name
- Interventional (NCT07257185)
- Testing
- MHB018A injection
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Minghui Pharmaceutical (Hangzhou) Ltd
Congenital Bleeding Disorder, Haemophilia A
Study for Turoctocog Alfa Treatment Regimen in Iraqi Haemophilia A Patients
The study has descriptive purposes, with aim of assessing how turoctocog alfa is used in the everyday practice and to provide a baseline for the management of haemophilia A and does not involve any change in the clinical…
- Trial name
- NCT06574984
- Testing
- Turoctocog alfa
- Where
- 7 sites in Iraq
- Volunteers
- 329 people
- Sponsor
- Novo Nordisk A/S
Leukemia
Vaccine Therapy and GM-CSF in Treating Patients With Acute Myeloid Leukemia in Remission
RATIONALE: Vaccines made from a peptide may help the body build an effective immune response to kill cancer cells. Colony-stimulating factors, such as GM-CSF, increase the number of white blood cells and platelets found in bone…
- Trial name
- NCT00454168
- Testing
- PR1 leukemia peptide vaccine, sargramostim
- Where
- 15 sites in United States
- Volunteers
- 244 people
- Sponsor
- The Vaccine Company
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary…
- Trial name
- NCT07332091
- Testing
- Vamifeport
- Where
- 100 sites in United States, Australia and 16 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
Glanzmann Thrombasthenia
A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia
The goal of this clinical trial is to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia.
- Trial name
- NCT06211634
- Testing
- HMB-001
- Where
- 17 sites in United States, Belgium and 4 more
- Volunteers
- 57 people
- Sponsor
- Hemab ApS
Dilated Cardiomyopathy (DCM)
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.
- Trial name
- NCT07137338
- Testing
- RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Von Willebrand Disease
Extension Study of Biostate in Subjects With Von Willebrand Disease
The aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or…
- Trial name
- NCT01224808
- Testing
- Biostate
- Where
- 6 sites in Bulgaria, Germany and 3 more
- Volunteers
- 20 people
- Sponsor
- CSL Behring
Hemophilia A
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts.
- Trial name
- NCT00157040
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
Alpha-Mannosidosis
Evaluation of Long-term Efficacy of Treatment With Lamazym
The overall objective is to evaluate the long-term efficacy of Lamazym i.v. treatment in patients with alpha-Mannosidosis previously enrolled in Lamazym trials and currently receiving the treatment according to the AfterCare…
- Trial name
- rhLAMAN-10 (NCT02478840)
- Testing
- Lamazym
- Where
- Denmark
- Volunteers
- 18 people
- Sponsor
- Zymenex A/S
Hemophilia A
Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years)…
- Trial name
- NCT02930317
- Testing
- Recombinant Factor VIII (50 IU/kg), Recombinant Factor VIII (On-demand treatment)
- Where
- 4 sites in China
- Volunteers
- 60 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Heart Defects, Congenital
Safety and Performance of the COR-VG-001 Conduit in Pediatric Patients for Extracardiac Total Cavopulmonary Connection
The extracardiac Fontan surgery/procedure involves diverting the venous blood from the inferior vena cava to the pulmonary arteries without passing through the morphologic right ventricle.
- Trial name
- EC-TCPC (NCT02377674)
- Testing
- Vascular Graft, Model COR-VG-001
- Where
- Russia
- Volunteers
- 5 people
- Sponsor
- Xeltis
Cystic Fibrosis
Study to Validate the Instructions for Use of TOBI® Podhaler™ in Cystic Fibrosis Patients
The purpose of this actual use human factors (HF) study is to validate the approved US TOBI Podhaler Instructions for Use (IFU), by establishing that the IFU effectively communicates the information necessary to achieve safe and…
- Trial name
- NCT02178540
- Testing
- Tobi Podhaler
- Where
- 13 sites in United States
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Hypochondroplasia
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
- Trial name
- CANOPY-HCH-3 (NCT06455059)
- Testing
- Vosoritide
- Where
- 23 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- BioMarin Pharmaceutical
Acute Gouty Arthritis
A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty Arthritis
The purpose of this study is to evaluate how anakinra relieves pain for patients with acute gout that cannot take non-steroidal anti-inflammatory drugs (NSAIDs) and colchicine.
- Trial name
- anaGO (NCT03002974)
- Testing
- Anakinra 100 mg, Triamcinolone Acetonide 40 mg
- Where
- 37 sites in United States
- Volunteers
- 165 people
- Sponsor
- Swedish Orphan Biovitrum
Hereditary Angioedema, HAE
STOP-HAE: A Phase 3 Study of ADX-324 in HAE
This study will evaluate the efficacy and safety of ADX-324 in participants with Type 1 or Type 2 hereditary angioedema. The study will also evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related…
- Conditions
- Hereditary Angioedema, HAE, Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2
- Trial name
- STOP-HAE (NCT06960213)
- Testing
- ADX-324
- Where
- 70 sites in United States, Argentina and 22 more
- Volunteers
- 90 people
- Sponsor
- ADARx Pharmaceuticals, Inc.
Congenital Heart Disease, Tetralogy of Fallot
Study of the Native Outflow Tract Transcatheter Pulmonary Valve (TPV)
The purpose of this early feasibility study is to determine how a new transcatheter pulmonary valve will move and perform once implanted in the right ventricular outflow tract.
- Trial name
- NCT01762124
- Testing
- Native Outflow Tract TPV
- Where
- 3 sites in United States, Canada
- Volunteers
- 20 people
- Sponsor
- Medtronic Heart Valves
PIK3CA-Related Overgrowth Spectrum (PROS)
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the…
- Trial name
- EPIK-P1 (NCT04285723)
- Testing
- alpelisib
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Healthy, Long QT Syndrome
Study Evaluating the Effects of Multiple Oral Doses of ERB-041 on Cardiac Repolarization in Healthy Subjects
To assess the effect of ERB-041 after multiple oral doses on cardiac repolarization as assessed by the QTc interval.
- Trial name
- NCT00316459
- Testing
- ERB-041
- Where
- United States
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Leber's Hereditary Optic Neuropathy
Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study
Expanded Access Program for Idebenone in Patients with Leber's Hereditary Optic Neuropathy who completed the LEROS Study
- Trial name
- US EAP LHON (NCT04381091)
- Testing
- Idebenone 150 MG Oral Tablet
- Sponsor
- Santhera Pharmaceuticals
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