Clinical trials: Fetal Developmental Abnormality
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Alvocidib, Cytarabine, and Mitoxantrone Hydrochloride or Cytarabine and Daunorubicin Hydrochloride in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
This randomized phase II trial is studying how alvocidib, cytarabine, and mitoxantrone hydrochloride work compared to cytarabine and daunorubicin hydrochloride in treating patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01349972
- Testing
- alvocidib, daunorubicin hydrochloride, mitoxantrone hydrochloride
- Where
- 11 sites in United States
- Volunteers
- 172 people
- Sponsor
- National Cancer Institute (NCI)
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
Equinus Contracture
The Effect of Botulinum Toxin A Injections on Ankle Dorsiflexion Following Internal Fixation of Tibial Pilon Fractures
The purpose of this study is to determine if botulinum toxin type A (Botox) injections, at the time of surgery for pilon fractures, will improve ankle range-of-motion and functionality.
- Trial name
- Botox (NCT02051933)
- Testing
- Botulinum toxin type A, sodium chloride
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Wake Forest University Health Sciences
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
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
Gastroschisis
Evaluation of Glycerin Suppositories to Improve Bowel Function in Gastroschisis
Gastroschisis is a congenital defect of the abdominal wall that leads to evisceration of various amounts of the abdominal organs.
- Trial name
- NCT03355326
- Testing
- Glycerin Suppository
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Indiana University
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
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
Dry Age-related Macular Degeneration
EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005
The purpose of this clinical study was to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration…
- Trial name
- EXPLORE (NCT04437368)
- Testing
- GT005
- Where
- 55 sites in United States, Australia and 6 more
- Volunteers
- 98 people
- Sponsor
- Gyroscope Therapeutics Limited
Impetigo, Folliculitis
Twice Daily Altabax Application for the Treatment of Uncomplicated Soft Tissue Infection
The purpose of this study is to document the clinical and bacteriological efficacy of retapamulin in the treatment of subjects with bacterial infections, including impetigo, folliculitis, and minor soft tissue infections…
- Conditions
- Impetigo, Folliculitis, Secondarily Infected Eczema, Minor Soft Tissue Infections
- Trial name
- NCT01126268
- Testing
- Retapamulin (Altabax)
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- The University of Texas Health Science Center, Houston
Maxillary Retrusion, Nasal Airway Obstruction
Evaluation of the Effect of Three Types of Rapid Maxillary Expanders (Conventional, Hybrid and MSE)
Aim of the study: To compare radiographically the morphometric changes in the nasal airway after using three types of rapid maxillary expansion (RME) conventional hyrax (CH), hybrid hyrax (HH) and maxillary skeletal expander…
- Trial name
- NCT05446714
- Testing
- CH 4 band, HH 2 bands, MSE 2 bands and 4
- Where
- Egypt
- Volunteers
- 54 people
- Sponsor
- Al-Azhar University
Intracranial Arteriovenous Malformations (AVM)
Protocol for Staged Stereotactic Radiosurgery for Large Arteriovenous Malformations
Stereotactic radiosurgery is a well established treatment option for arteriovenous malformations (AVMs). The potential complications related to radiosurgery are well documented and are predominately related to radiation effects…
- Trial name
- NCT02576535
- Testing
- Fractionated stereotactic radiosurgery, Leskell gamma unit
- Volunteers
- 18 people
- Sponsor
- NYU Langone Health
Homozygous Familial Hypercholesterolemia
Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor
The primary objective of this study is to evaluate the safety and tolerability of 4 doses of lomitapide (AEGR-733; BMS-201038) given as an initial low dose and then escalated through an additional 3 dose levels over a 16-week…
- Trial name
- NCT01556906
- Testing
- Lomitapide
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617847
- Testing
- WVE-120101
- Where
- 18 sites in Australia, Canada and 5 more
- Volunteers
- 27 people
- Sponsor
- Wave Life Sciences USA, Inc.
Atopic Dermatitis
Dupilumab Skin Barrier Function Study in Atopic Dermatitis
Primary Objective: Evaluate changes in skin barrier function with transepidermal water loss (TEWL) assessed after skin tape stripping (STS) in pre-defined lesional skin in participants with moderate to severe atopic dermatitis…
- Trial name
- BALISTAD (NCT04447417)
- Testing
- Dupilumab SAR231893
- Where
- 2 sites in United States, Canada
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Sanofi
Craniosynostosis
The Efficacy and Population Pharmacokinetics of Tranexamic Acid for Craniosynostosis Surgery
This research study is being performed to evaluate two different doses of Tranexamic acid (TXA) in children who have craniosynostosis and have been referred to Boston Children's Hospital for corrective surgery.
- Trial name
- NCT02188576
- Testing
- high dose TXA, Low dose TXA
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Boston Children's Hospital
Wolfram Syndrome, Diabetes Mellitus
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration.
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Trial name
- NCT02829268
- Testing
- dantrolene sodium
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Hemophilia
Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery
This is an interventional, prospective, international, multicenter, single-arm, Phase 3, and sequential efficacy and safety study in adolescents and adults with congenital hemophilia A or B with inhibitors to factor VIII (FVIII)…
- Trial name
- SCOPE HIM (NCT05695391)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 15 sites in United States, Malaysia and 4 more
- Volunteers
- 2 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Congenital Diaphragmatic Hernia, Pulmonary Hypoplasia
Fetoscopic Endoluminal Tracheal Occlusion in Severe Left Congenital Diaphragmatic Hernia
CDH is a birth defect characterized by the development, very early in gestation, of a hole in the diaphragm, the breathing muscle that separates the chest from the abdomen.
- Trial name
- CHOP_FETO (NCT02549820)
- Testing
- GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Children's Hospital of Philadelphia
Epidermolysis Bullosa, Epidermolysis Bullosa Simplex
Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
The primary objective of this study is to evaluate the long term safety and tolerability of diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously participated in the CCP-020-301 or the CCP-020-101…
- Trial name
- NCT03389308
- Testing
- diacerein 1% ointment
- Where
- 3 sites in United States
- Volunteers
- 51 people
- Sponsor
- Castle Creek Pharmaceuticals, LLC
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