Clinical trials: Birth Defect
Congenital Heart Disease, Periventricular Leucomalacia
Randomized Trial of Maternal Progesterone Therapy
Neurodevelopmental disability is now recognized as the most common long-term complication after cardiac surgery in neonates. Research studies have shown that progesterone is critical to the development of the brain and in a…
- Conditions
- Congenital Heart Disease, Periventricular Leucomalacia, Brain Development, Cardiac Surgery
- Trial name
- NCT02133573
- Testing
- Progesterone, Vaginal lubricant
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- Children's Hospital of Philadelphia
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Iodine I 131 Monoclonal Antibody BC8, Fludarabine Phosphate, Total Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients With Advanced Acute Myeloid Leukemia or Myelodysplastic Syndrome
This phase II trial studies the side effects and best dose of iodine I 131 monoclonal antibody BC8 when given together with fludarabine phosphate, total-body irradiation, and donor stem cell transplant followed by cyclosporine…
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT00119366
- Testing
- iodine I 131 monoclonal antibody BC8, fludarabine phosphate, total-body irradiation
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD).
- Trial name
- NCT03263559
- Testing
- Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG
- Where
- 32 sites in United States
- Volunteers
- 95 people
- Sponsor
- Medical College of Wisconsin
Angelman Syndrome
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.
- Trial name
- NCT02996305
- Testing
- OV101 Regimen 1, OV101 regimen 2
- Where
- 13 sites in United States, Israel
- Volunteers
- 88 people
- Sponsor
- Healx AI
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Sickle Cell Disease, Vaso-occlusive Crisis
Steroid Treatment for Sickle Cell Pain Crisis
The painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication…
- Trial name
- NCT00263562
- Testing
- Steroid arm
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Baylor College of Medicine
Respiratory Distress Syndrome, Newborn
A Safety and Efficacy Study of Lucinactant for Inhalation in Preterm Neonates 26 to 32 Weeks Gestational Age
This study is to evaluate the safety and efficacy of lucinactant for inhalation in conjunction with nCPAP, in comparison to nCPAP alone, in preterm neonates with RDS, as assessed by the incidence of and time to respiratory…
- Trial name
- NCT04264156
- Testing
- Lucinactant for Inhalation, nCPAP Only
- Where
- 3 sites in Poland
- Volunteers
- 12 people
- Sponsor
- Windtree Therapeutics
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Down Syndrome
Promoting Health Through Parent Empowerment and the Activation of Routines (PrO-PEAR)
This study will examine the feasibility of the Promoting Health through Parent Empowerment and the Activation of Routines (Pro-PEAR) intervention using a open case-series for intervention optimization followed by a pilot RCT in…
- Trial name
- Pro-PEAR (NCT05020366)
- Testing
- Promoting Health through Parent Empowerment and the Activation of Routines, Enhanced Usual Care
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Pittsburgh
Cystic Fibrosis
Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB…
- Trial name
- ASSURE (NCT02750501)
- Testing
- RELiZORB (immobilized lipase) cartridge, Impact Peptide 1.5
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Alcresta Therapeutics, Inc.
Tourette Syndrome, Tic Disorder, Chronic Motor or Vocal
Open-label MNS for Tourette Syndrome
A recent report (Morera Maiquez et al 2020) described reduced tic severity in people with Tourette syndrome during 1-minute epochs of median nerve stimulation (MNS) at 10 Hz.
- Trial name
- NCT05016765
- Testing
- Active, self-directed electrical stimulation of the median nerve
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease, Vaso-Occlusive Crisis
Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis
Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the…
- Trial name
- AKTSS (NCT03296345)
- Testing
- Ketamine
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Congenital Myotonic Dystrophy
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
- Trial name
- NCT03692312
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 56 people
- Sponsor
- AMO Pharma Limited
Hereditary Angioedema
C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)
Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities…
- Trial name
- NCT00292981
- Testing
- C1 Esterase Inhibitor
- Where
- 14 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- CSL Behring
Hemophilia A Without Inhibitor
The Hemophilia Inhibitor Prevention Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which Eloctate will be compared with Emicizumab, using adaptive design, to prevent inhibitors in patients with severe hemophilia A.
- Trial name
- NCT04303559
- Testing
- Eloctate Injectable Product, Emicizumab Injection [Hemlibra]
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni
Pulmonary Valve Insufficiency, Pulmonary Regurgitation
COMPASSION - COngenital Multicenter Trial of Pulmonic VAlve Regurgitation Studying the SAPIEN InterventIONal THV
To assess the safety and effectiveness of pulmonic THV implantation in subjects with dysfunctional RVOT conduit requiring treatment for moderate or severe pulmonary regurgitation (≥3+ pulmonary regurgitation) and/or RVOT conduit…
- Conditions
- Pulmonary Valve Insufficiency, Pulmonary Regurgitation, Dysfunctional RVOT Conduit, Pulmonary Obstruction
- Trial name
- NCT00676689
- Testing
- SAPIEN Transcatheter Valve Implantation
- Where
- 7 sites in United States
- Volunteers
- 81 people
- Sponsor
- Edwards Lifesciences
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 6 To 10
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of cognitive dysfunction shown by children with Down syndrome, aged 6 to 10 years.
- Trial name
- NCT00754013
- Testing
- Aricept (Donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Eisai Inc.
Rett Syndrome
Pharmacological Treatment of Rett Syndrome With Statins
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
- Trial name
- Statins (NCT02563860)
- Testing
- Lovastatin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Montefiore Medical Center
Atopic Dermatitis, Ichthyosis Vulgaris
Genetic Screening for Filaggrin Mutation in Atopic Dermatitis and Ichthyosis Vulgaris in the African American Population
The investigators' primary objective is to identify common and rare mutations in the filaggrin gene in African American patients with a diagnosis of atopic dermatitis and ichthyosis vulgaris.
- Trial name
- NCT01016106
- Testing
- Buccal Swab
- Where
- 2 sites in United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Northwestern University
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease.
- Trial name
- PAPAYA (NCT01410890)
- Testing
- alglucosidase alfa
- Where
- 12 sites in United States, Bulgaria and 4 more
- Volunteers
- 21 people
- Sponsor
- Genzyme, a Sanofi Company
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