Clinical trials: Newborn Diseases
Atopic Dermatitis
Safety and Bioequivalence of Pimecrolimus Cream 1% and Elidel R in Treatment of Mild to Moderate Atopic Dermatitis
A phase 3, double-blind, randomized, parallel-group, placebo-controlled, multicenter study to evaluate the safety and bioequivalence of Par Pharmaceutical Inc.'s Pimecrolimus Cream, 1% and Reference Listed Elidel® (Pimecrolimus…
- Trial name
- NCT03297502
- Testing
- pimecrolimus cream 1%, Elidel (pimecrolimus) cream 1%
- Volunteers
- 582 people
- Sponsor
- Par Pharmaceutical, Inc.
Junctional Epidermolysis Bullosa
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17)
Prospective open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a gamma-retroviral vector carrying…
- Trial name
- HOLOGENE17 (NCT03490331)
- Testing
- Transplantation surgery of genetically corrected cultured epidermal autograft (ATMP)
- Where
- Austria
- Volunteers
- 1 people
- Sponsor
- Holostem s.r.l.
Duchenne Muscular Dystrophy
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies.
- Trial name
- NCT02525302
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Akashi Therapeutics
Hemophilia A
Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies
This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives.
- Trial name
- PARkER (NCT03405337)
- Testing
- FVIII products, Conventional FVIII replacement therapies
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- Bayer
Multiple Myeloma
WT1 Vaccine Treatment of Patients With Multiple Myeloma After Autologous Stem Cell Transplantation
The purpose of this trial is to assess the immune response after vaccination with a peptide vaccine called galinpepimut-S (or GPS) in a type of blood cancer called multiple myeloma.
- Trial name
- NCT01827137
- Testing
- Galinpepimut-S, GM-CSF, Montanide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sellas Life Sciences Group
Atopic Dermatitis
The Study of CM326 in Adult Subjects With Atopic Dermatitis
This is a open-label, multi-center study to evaluate the safety and efficacy of CM326 in atopic adrmatitis subjects.
- Trial name
- NCT05671445
- Testing
- CM326
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Keymed Biosciences Co.Ltd
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD).
- Conditions
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
- Trial name
- NCT06610201
- Testing
- Clinical outcomes of patients with VWD, Type 1, Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 200 people
- Sponsor
- Hemab ApS
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Heart Diseases, ASD - Atrial Septal Defect
Abbott Structural Heart Device Registry
The Abbott Structural Heart (SH) Registry is being conducted to confirm the safety and performance of Abbott's SH devices in a post-market, real-world setting.
- Conditions
- Heart Diseases, ASD - Atrial Septal Defect, VSD - Muscular Ventricular Septal Defect, PFO - Patent Foramen Ovale
- Trial name
- SH-Registry (NCT06590467)
- Testing
- Amplatzer™ Occlusion Devices, Epic™ Surgical Tissue Heart Valve devices
- Where
- 36 sites in United States, Denmark and 8 more
- Volunteers
- 2,500 people
- Sponsor
- Abbott Medical Devices
Atopic Dermatitis
Drug-drug Interaction Trial With Tralokinumab in Moderate to Severe Atopic Dermatitis - ECZTRA 4
The purpose of this trial is to investigate if tralokinumab changes the metabolism of selected CYP substrates in adults with moderate-to-severe AD after: 14 weeks of treatment with tralokinumab a single dose of tralokinumab
- Trial name
- NCT03556592
- Testing
- Tralokinumab, Caffeine, Warfarin
- Where
- 12 sites in United States, France and 1 more
- Volunteers
- 40 people
- Sponsor
- LEO Pharma
Adrenal Insufficiency
Extension Study for Patients Entered Into Study Infacort 003
A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia…
- Trial name
- NCT02733367
- Testing
- Infacort®
- Where
- Germany
- Volunteers
- 18 people
- Sponsor
- Neurocrine UK Limited
Muscular Dystrophy, Duchenne
Safety Study of BLS-M22 in Healthy Volunteers
BLS-M22 is being developed as an anti-myostatin agent for the treatment of Duchenne Muscular Dystrophy (Muscular Dystrophy). A total of 37 subjects participated in this study to confirm the safety of BLS-M22.
- Trial name
- NCT03789734
- Testing
- BLS-M22
- Where
- South Korea
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- BioLeaders Corporation
Hereditary Angioedema
Efficacy and Safety of GNR-038 vs Berinert® in Patients With Hereditary Angioedema
It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema
- Trial name
- NCT04898309
- Testing
- GNR-038, 50 МЕ/ kg, GNR-038, 100 МЕ/ kg, Berinert®, 20 МЕ/ kg
- Where
- 5 sites in Russia
- Sponsor
- AO GENERIUM
Cardiomyopathy, Dilated
Immunoadsorption Therapy for Patients With Non-Ischemic Dilated Cardiomyopathy (DCM)
The purpose of this study is to evaluate the clinical safety and feasibility of Mysorba in patients with chronic non-ischemic dilated cardiomyopathy (DCM).
- Trial name
- NCT01478087
- Testing
- Mysorba
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Asahi Kasei Medical Co., Ltd.
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Clofarabine, Cytarabine, and Filgrastim Followed by Infusion of Non-HLA Matched Ex Vivo Expanded Cord Blood Progenitors in Treating Patients With Acute Myeloid Leukemia
This phase I trial is studying the safety and potential efficacy of infusing non-human leukocyte antigen (HLA) matched ex vivo expanded cord blood progenitors following treatment with clofarabine and cytarabine for patients with…
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01031368
- Testing
- cytarabine, clofarabine, filgrastim
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Nohla Therapeutics, Inc.
Hemophilia A
Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A
This study is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who…
- Trial name
- NCT03815318
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 73 people
- Sponsor
- Sinocelltech Ltd.
Periventricular Leukomalacia, White Matter Disease
Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia
The goal of this study is to explore a new treatment that may help repair brain damage in individuals with periventricular leukomalacia (PVL), a condition that affects white matter in the brain.
- Conditions
- Periventricular Leukomalacia, White Matter Disease, Hypoxic-Ischemic Encephalopathy, Demyelinating Diseases
- Trial name
- NCT06985303
- Testing
- Investigational Combination Product
- Sponsor
- MGAM LLC
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
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