163 matching studies

Sponsor Condition of Interest
Pediatric cGVHD Symptom Scale
National Cancer Institute (NCI) Graft vs Host Disease
Background: Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life.... expand

Background: Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life. The questionnaires that measure thesymptoms caused by cGVHD are designed for adults. Children/adolescents may not describe their symptoms in the same way. The goal of this research is to improve the way we measure how bothersome these symptoms are for children/adolescents living with cGVHD. Objective: To develop a questionnaire (The Pediatric cGHVD Symptom Scale) for children/adolescents living with cGVHD to identify the symptoms they are experiencing and describe how bothersome those symptoms are to them. An additional goal is to design a parent/guardian companion questionnaire that can be used to capture the symptom experiences of very young children who may not be able to complete a questionnaire. Eligibility: Children/adolescents ages 5-17 who are receiving treatment for cGVHD after a stem cell transplant, and their parent/guardian.. Design: This study consists of 2 projects. Children/adolescents with cGVHD and their parent/guardianparticipants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17. In project 1, participants will complete an age-appropriate questionnaire about cGVHD symptoms. The questionnaire will ask about the child/adolescent s physical functioning and emotional well-being. The parent/guardian will out fill out a companion questionnaire online. The child/adolescent will then review their completed questionnaire during an interview with a researcher and will be asked whether the questions about their symptoms were difficult to understand. The parent/guardian and child/adolescent will then be interviewed together to further explore their responses to the questionnaires. Interviews will be done in person, by phone, and online. . Based on what is learned through these interviews, the wording of the questionnaire will be improved for better comprehension and ease of response. In project 2, participants will complete this revised questionnairefor their age group along with some other questionnaires that ask about quality of life. Both the child/adolescent and parent/guardian will fill out the questionnaires online at three separate time points. In both projects, children/adolescents with cGVHD and their parent/guardian participants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17.

Type: Observational

Start Date: Oct 2019

open study

Clinical and Laboratory Study of Methylmalonic Acidemia
National Human Genome Research Institute (NHGRI) Organic Acidemia Methylmalonic Acidemia Inborn Errors of Metabolism
This study will evaluate patients with methylmalonic acidemia (MMA) to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it. People with MMA may have problems with learning and development... expand

This study will evaluate patients with methylmalonic acidemia (MMA) to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it. People with MMA may have problems with learning and development and kidney malfunctioning. They can become seriously ill, sometimes with little warning. There is no cure for any MMA, but special diets and vitamin therapies are used for treatment. Patients between 2 and 70 years of age with MMA may be eligible for this study. Participants are admitted to the NIH Clinical Center for 4-5 days each year for 5-10 years for the following tests and procedures: - Medical history, physical examination, eye examination - Consultations from dentists and specialists in the nervous system, digestive tract, endocrine, and kidney, as needed. - 24-hour urine collection to examine for methylmalonic acid, other acids, sugar, and proteins for measuring kidney function. - Blood test to assess liver and thyroid function, blood counts and blood chemistries, methylmalonic acid levels, and for genetic tests and basic research studies. - Blood test to measure growth hormone production. For this test, a very small amount of blood is collected overnight (every 20-30 minutes from 8:00 PM to 8:00 AM) through an intravenous catheter (plastic tube placed in a vein). The total amount of blood drawn is approximately 1 tablespoon. Patients who have stopped growing or whose weight does not permit collection of 1 tablespoon of blood do not undergo this procedure. - Frequent blood pressure measurements, including overnight monitoring - Skin biopsy for cell culture (cells to grow in the laboratory for future testing). For this procedure, an area of skin is numbed with an anesthetic such as lidocaine. A 4-mm diameter circular area is then removed using a sharp punch and scissors. The wound is dressed and usually heals within a week. - Photographs of the face and body (wearing underwear) to help track growth and appearance. - Ultrasound of the kidneys - Hand x-ray to determine bone age - Dual energy x-ray absorptionometry (DEXA) scan to assess bone density. For the DEXA scan, the patient lies still on a table while the spine and hip are scanned using a small amount of radiation. Any patient who becomes seriously ill during the evaluation may be cared for at the NIH or transferred to another hospital if it is deemed advisable.

Type: Observational

Start Date: Jun 2004

open study

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis...
National Cancer Institute (NCI) Neurofibromatosis 1 Plexiform Neurofibromas
Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (PNs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives: To examine... expand

Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (PNs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives: To examine and improve questionnaires about daily living for people with NF1 and PNs. Eligibility: People ages 5 and older with NF1 and a PN Design: Participants will be screened with medical history. This study will have 2 phases. Phase 1 participants will talk about existing pain assessment questionnaires and how PNs affect their life. They will have group discussions of up to 8 people of a similar age with NF1 and PNs, or the parents of children with it. These will last about 90 minutes. Children ages 5 to 7 and their parents will have one-on-one meetings instead. These will last about 45 minutes. Discussions will be audiotaped. After the questionnaires have been changed, individual interviews will discuss the new wording, instructions, questions, and electronic format of the new forms. Phase 1 participants may be invited to Phase 2. Phase 2 participants will complete the new questionnaires. These may be pen-and-paper or electronic. The questionnaires will take about 30 minutes for adults and teens. Children will work one-on-one with a staff member and may need up to 45 minutes. A small group of participants will be complete the forms twice in clinic and 1 month later at home. Also, a small group who start a new pain treatment or have a dose increase in their treatment will complete the forms twice before the treatment change and 1 month later.

Type: Observational

Start Date: Nov 2015

open study

Measuring Analgesic Interventions
Julia Finkel Pain
It is generally recognized that pain assessment and management especially in newborns, children and other nonverbal populations is an unmet need. According to the American Medical Association, "the pediatric population is at risk of inadequate pain management, with age-related... expand

It is generally recognized that pain assessment and management especially in newborns, children and other nonverbal populations is an unmet need. According to the American Medical Association, "the pediatric population is at risk of inadequate pain management, with age-related factors affecting pain management in children. Children are often given minimal or no analgesia for procedures that would routinely be treated aggressively in adults. Although much is now known about pain management in children, it has not been widely or effectively translated into routine clinical practice". These two factors combine to emphasize the necessity for an objective tool to quantify pain and monitor the effectiveness of analgesia, especially during treatments. Further, it is reported that many patients require a combination of treatments, and it is often necessary to test a variety of treatments before the personal match for treatment is found. The method in place to change the care on a subjective basis is difficult, time consuming, and not easily individualized. This pilot study is part of an ongoing effort to develop a method to objectively assess response to specific analgesic interventions. It specifically aims to discern the impact of analgesic interventions on sensory nerve fiber sensitivity in a diverse patient population.

Type: Observational

Start Date: Oct 2018

open study

Objective Assessment of Sensory Nerve Fiber Sensitivity
Julia Finkel Nerve Pain
This pilot study utilizes a unique technology to determine nerve fiber sensitivity. This will allow us to determine whether this method and device can discern changes in pain intensity and type, which is predicated on differing nerve fiber sensitivities. expand

This pilot study utilizes a unique technology to determine nerve fiber sensitivity. This will allow us to determine whether this method and device can discern changes in pain intensity and type, which is predicated on differing nerve fiber sensitivities.

Type: Observational

Start Date: Jun 2018

open study

Treatment of Children With Recurrent Refractory Brain/Solid Tumors and Recurrent Ependymoma
The Hospital for Sick Children Recurrent Childhood CNS Tumor Ependymoma, Recurrent Childhood Childhood Solid Tumor
Many pediatric brain and solid tumors have altered epigenetic landscapes, and altered DNA methylation. As such this study is a Phase I/Ib study of combined 5'Azacitidine with an escalating dose of carboplatin for all recurrent/refractory pediatric brain and solid tumors. The... expand

Many pediatric brain and solid tumors have altered epigenetic landscapes, and altered DNA methylation. As such this study is a Phase I/Ib study of combined 5'Azacitidine with an escalating dose of carboplatin for all recurrent/refractory pediatric brain and solid tumors. The phase I component will establish with maximum tolerated dose of carboplatin with azacytidine. An expansion cohort will be recruited of up to 30 patients will follow consisting of 20 recurrent posterior fossa ependymoma and 10 recurrent supratentorial ependymoma.

Type: Interventional

Start Date: Aug 2017

open study

A Study of Nivolumab Plus Brentuximab Vedotin in Patients Between 5 and 30 Years Old, With Hodgkin's...
Bristol-Myers Squibb Hodgkin Disease
The purpose of this study is to determine whether nivolumab plus brentuximab vedotin (followed by brentuximab vedotin plus bendamustine in patient with suboptimal response) is safe and effective in treating patients with Hodgkin's lymphoma (cHL). Eligible patients are children,... expand

The purpose of this study is to determine whether nivolumab plus brentuximab vedotin (followed by brentuximab vedotin plus bendamustine in patient with suboptimal response) is safe and effective in treating patients with Hodgkin's lymphoma (cHL). Eligible patients are children, adolescents, and young adults relapsed or refractory to first line.

Type: Interventional

Start Date: Mar 2017

open study

Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn
United Therapeutics Persistent Pulmonary Hypertension of the Newborn
This pilot study aims to assess the safety and treatment effect of acute dosing with IV Remodulin in neonates with persistent pulmonary hypertension of the newborn (PPHN). expand

This pilot study aims to assess the safety and treatment effect of acute dosing with IV Remodulin in neonates with persistent pulmonary hypertension of the newborn (PPHN).

Type: Interventional

Start Date: May 2015

open study

Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
Levo Therapeutics, Inc. Prader-Willi Syndrome
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin). This study will also evaluate the safety and tolerability of... expand

This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin). This study will also evaluate the safety and tolerability of LV-101.

Type: Interventional

Start Date: Nov 2018

open study

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Center for International Blood and Marrow Transplant Research Hematologic Malignancies Inherited Disorders of Metabolism Inherited Abnormalities of Platelets Histiocytic Disorders Acute Myelogenous Leukemia (AML or ANLL)
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. expand

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Type: Observational

Start Date: Oct 2011

open study

A Safety and Efficacy Study of Ibrutinib in Pediatric and Young Adult Participants With Relapsed or Refractory...
Janssen Research & Development, LLC Lymphoma, Non-Hodgkin
The purpose of this study is to confirm that the pharmacokinetics of ibrutinib in pediatric participants is consistent with that in adults (part 1) and to assess efficacy (event-free survival [EFS]) of ibrutinib in combination with rituximab, ifosfamide, carboplatin, and etoposide... expand

The purpose of this study is to confirm that the pharmacokinetics of ibrutinib in pediatric participants is consistent with that in adults (part 1) and to assess efficacy (event-free survival [EFS]) of ibrutinib in combination with rituximab, ifosfamide, carboplatin, and etoposide (RICE) or rituximab, vincristine, ifosfamide, carboplatin, and idarubicin (RVICI) background therapy compared to RICE or RVICI background therapy alone (part 2).

Type: Interventional

Start Date: Jul 2016

open study

Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN...
Medical College of Wisconsin Sickle Cell Disease
This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival. expand

This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival.

Type: Interventional

Start Date: Nov 2016

open study

T-Lymphocytes for Prevention or Treatment of Viral Infections Following Hematopoietic Stem Cell Transplantation
Children's Research Institute Viral Infections Bone Marrow Transplant Infection
This Phase I dose-escalation trial is designed to evaluate the safety of rapidly generated multivirus-specific T-cell products with antiviral activity against CMV, EBV, adenovirus, HHV6, BK virus, JC virus, and human parainfluenza-3 (HPIV3), derived from eligible HSCT donors.... expand

This Phase I dose-escalation trial is designed to evaluate the safety of rapidly generated multivirus-specific T-cell products with antiviral activity against CMV, EBV, adenovirus, HHV6, BK virus, JC virus, and human parainfluenza-3 (HPIV3), derived from eligible HSCT donors. In this trial, the investigators will utilize a rapid generation protocol for broad spectrum multivirus-specific T cells for infusion to recipients of allogeneic hematopoietic stem cell transplant (HSCT), who are at risk of developing EBV, CMV, adenovirus, HHV6, BKV and/or HPIV3, or with PCR/culture confirmed infection(s). These cells will be derived from HSCT donors, and the study agent will be assessed at each dose for evidence of dose-limiting toxicities (DLT). This study will have two arms: Arm A will include patients who receive prophylactic treatment, and Arm B will include patients who receive VSTs for one or more active infections with targeted viruses. Determination of the study arm will be determined by the patient's clinical status. Study arms will each be analyzed for safety endpoints and secondary endpoints.

Type: Interventional

Start Date: Feb 2017

open study

Registry for Asthma Characterization and Recruitment 2
National Institute of Allergy and Infectious Diseases (NIAID) Asthma
There is a need for people to take part in research studies to learn more about diseases and how to treat them. The Registry for Asthma Characterization and Recruitment 2 (RACR2) will create a database of participants with asthma and nasal allergies, or risk factors for these... expand

There is a need for people to take part in research studies to learn more about diseases and how to treat them. The Registry for Asthma Characterization and Recruitment 2 (RACR2) will create a database of participants with asthma and nasal allergies, or risk factors for these conditions, who are potentially eligible for future Inner City Asthma Consortium (ICAC) trials. The registry database will include assessments of various asthma and allergy characteristics to achieve a more efficient, selective recruitment of these participants for other protocols.

Type: Observational [Patient Registry]

Start Date: Jun 2015

open study

Phase II Pediatric Study With Dabrafenib in Combination With Trametinib in Patients With HGG and LGG
Novartis Pharmaceuticals Diffuse Astrocytoma Anaplastic Astrocytoma Astrocytoma Oligodendroglioma, Childhood Anaplastic Oligodendroglioma
The purpose of this study is to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma or relapsed or refractory high grade glioma. expand

The purpose of this study is to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma or relapsed or refractory high grade glioma.

Type: Interventional

Start Date: Dec 2017

open study

Developing a Method to Objectively Measure Opioid Analgesia
Julia Finkel Analgesics, Antipyretics and Anti-Inflammatory Drugs Causing Adverse Effects in Therapeutic Use
Inappropriate prescribing is the fundamental upstream driver of the opioid epidemic. Objective measures to determine the appropriateness of an opioid intervention, provide monitoring of the therapy for adequacy of dose and detection of tolerance or hyperalgesia would eliminate... expand

Inappropriate prescribing is the fundamental upstream driver of the opioid epidemic. Objective measures to determine the appropriateness of an opioid intervention, provide monitoring of the therapy for adequacy of dose and detection of tolerance or hyperalgesia would eliminate the subjective nature of opioid mediated pain management and obviate iatrogenic facilitation of opioid abuse. The present study is designed to objectively determine whether our device can pain type and determine analgesic efficacy thereby optimizing treatment selection and opioid management.

Type: Observational

Start Date: Jul 2018

open study

A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function
David Nichols, MD Cystic Fibrosis
This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT) in people with one or more copies of the F508del mutation, study the effects of TCT across... expand

This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT) in people with one or more copies of the F508del mutation, study the effects of TCT across a number of CF disease manifestations, and collect specimens for future research. Subjects in the study will have one "before TCT" visit within 30 days before initiation of the therapy and five "after TCT" visits over a 24-month follow-up period. Most participating sites will be divided into sub-study groups; each sub-study group will have specific non-optional procedures conducted in addition to the "Core" procedures. Finally there are four optional procedures (pH pill, transient elastography, and nasal cell procurement) that will be offered to subjects at certain sites. The duration of participation for each subject is 25 months. NOTE: FDA is currently reviewing the New Drug Application (NDA) for the TCT. Study will not begin unless and until FDA approval is granted.

Type: Observational

Start Date: Oct 2019

open study

Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry...
Bayer Hemophilia A
This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry or... expand

This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry or Jivi. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.

Type: Observational

Start Date: Aug 2018

open study

Allogeneic Virus-specific T Cell Lines (VSTs)
Catherine Bollard Viral Infections After HSCT
The primary purpose of the study is to evaluate whether most closely HLA-matched multivirus-specific T cell lines obtained from a bank of allogeneic virus-specific T cell lines (VSTs) have antiviral activity against three viruses: EBV, CMV and adenovirus. Reconstitution of anti-viral... expand

The primary purpose of the study is to evaluate whether most closely HLA-matched multivirus-specific T cell lines obtained from a bank of allogeneic virus-specific T cell lines (VSTs) have antiviral activity against three viruses: EBV, CMV and adenovirus. Reconstitution of anti-viral immunity by donor-derived VSTs has shown promise in preventing and treating infections associated with CMV, EBV and adenovirus post-transplant. However, the time taken to prepare patient-specific products and lack of virus-specific memory T cells in cord blood and seronegative donors, limits their value. An alternative is to use banked partially HLA-matched allogeneic VSTs. A prior phase II study at our institution using trivirus-specific VSTs generated using monocytes and EBV-transformed B cells gene-modified with a clinical grade adenoviral vector expressing CMV-pp65 to activate and expand specific T cells showed the feasibility, safety and activity of this approach for the treatment of refractory CMV, EBV and Adenovirus infections. However, the production process was lengthy, requiring 8-12 weeks, with exposure to biohazards (B95.8 EBV viral strain and adenovector), while antigenic competition between different viral components precluded increasing the spectrum of specificity beyond these three viruses. Investigator have overcome these limitations and in the current trial, they will evaluate whether rapidly generated, allogeneic most closely HLA-matched multivirus-specific VSTs, activated using overlapping peptide libraries spanning immunogenic antigens from CMV, adenovirus and EBV will be safe and produce anti-viral effects in allogeneic HSCT recipients infected with one of more of the targeted viruses that are persistent despite conventional anti-viral therapy. The study agent will be assessed for safety (stopping rules defined) and antiviral activity.

Type: Interventional

Start Date: Oct 2014

open study

Axitinib and Nivolumab in Treating Patients With Unresectable or Metastatic TFE/Translocation Renal Cell...
National Cancer Institute (NCI) Metastatic Renal Cell Carcinoma Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions Stage III Renal Cell Cancer AJCC v8 Stage IV Renal Cell Cancer AJCC v8 Unresectable Renal Cell Carcinoma
This phase II trial studies how well axitinib and nivolumab works in treating patients with TFE/translocation renal cell carcinoma that cannot be removed by surgery or has spread to other places in the body. Axitinib may stop the growth of tumor cells by blocking some of the... expand

This phase II trial studies how well axitinib and nivolumab works in treating patients with TFE/translocation renal cell carcinoma that cannot be removed by surgery or has spread to other places in the body. Axitinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving axitinib and nivolumab may work better in treating patients with TFE/translocation renal cell carcinoma compared to standard treatment, including surgery, chemotherapy, or immunotherapy.

Type: Interventional

Start Date: Oct 2018

open study

Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus...
Pharmacyclics LLC. Chronic Graft Versus Host Disease
Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD) expand

Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)

Type: Interventional

Start Date: Nov 2018

open study

Clinical Feasibility Study of Preoperative Surgical Planning
Gary F. Rogers, MD Craniosynostoses
Most children diagnosed with craniosynostosis undergo a relatively extensive cranial vault remodeling procedure. The decision of performing surgical cranial shape correction for patients with craniosynostosis typically rests on a subjective visual assessment of the severity of... expand

Most children diagnosed with craniosynostosis undergo a relatively extensive cranial vault remodeling procedure. The decision of performing surgical cranial shape correction for patients with craniosynostosis typically rests on a subjective visual assessment of the severity of the cranial malformation and the main goal of this procedure is to reduce the risk of elevated intracranial pressure and to provide a more normal cranial shape and volume. Personalized surgical planning systems to optimize intervention and leverage surgical expertise in the reconstruction of the cranial vault do not exist. Thus, the expertise of the surgeon is paramount for the success of the surgical correction of craniosynostosis. The goal of our project is to evaluate the feasibility and utility of a surgical plan derived from software developed at Children's National, iCSPlan.

Type: Interventional

Start Date: Jan 2020

open study

Technical Development for Pediatric Cardiovascular MRI
Children's Research Institute Congenital Heart Disease Cardiovascular Disease
This study will explore new ways of using magnetic resonance imaging (MRI) to evaluate pediatric patients with cardiovascular disease,congenital heart disease in patients of all ages, fetuses undergoing clinically indicated MR imaging. expand

This study will explore new ways of using magnetic resonance imaging (MRI) to evaluate pediatric patients with cardiovascular disease,congenital heart disease in patients of all ages, fetuses undergoing clinically indicated MR imaging.

Type: Observational

Start Date: May 2013

open study

A Study of Dulaglutide (LY2189265) in Children and Adolescents With Type 2 Diabetes
Eli Lilly and Company Type 2 Diabetes
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of the study drug dulaglutide compared to placebo in pediatric participants with type 2 diabetes. The study duration is approximately 60 weeks. expand

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of the study drug dulaglutide compared to placebo in pediatric participants with type 2 diabetes. The study duration is approximately 60 weeks.

Type: Interventional

Start Date: Dec 2016

open study

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMT CTN 1507)
Medical College of Wisconsin Sickle Cell Disease
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). Based on their age and entry criteria patients are stratified into two groups: (1)... expand

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). Based on their age and entry criteria patients are stratified into two groups: (1) children with SCD with strokes; and (2) adults with severe SCD.

Type: Interventional

Start Date: Oct 2017

open study