Clinical trials: Neoplastic Syndromes, Hereditary
Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
This clinical trial tests next generation sequencing (NGS) for the detection of precursor features of pre-myeloid cancers and bone marrow failure syndromes.
- Conditions
- Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome, Clonal Expansion, Cytopenia
- Trial name
- NCT02958462
- Testing
- Biospecimen Collection, Bone Marrow Biopsy, Punch Biopsy
- Where
- 3 sites in United States
- Volunteers
- 2,000 people
- Sponsor
- Mayo Clinic
Tuberous Sclerosis Complex
Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)
The investigators are enrolling 3-12 month old infants with a diagnosis of tuberous sclerosis complex (TSC) for a new study on early markers of autism.
- Trial name
- NCT01780441
- Where
- 5 sites in United States
- Volunteers
- 166 people
- Sponsor
- Boston Children's Hospital
Basal Cell Nevus Syndrome
Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.
- Trial name
- NCT04308395
- Testing
- Patidegib Topical Gel, 2%
- Where
- 37 sites in United States, Belgium and 7 more
- Volunteers
- 108 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Colorectal Cancer
WT1 in Colorectal Adenocarcinoma
Colorectal carcinoma is a major bublic health problem. It is due to several genetic mutations. Wilms' tumor1 (WT1) gene is a tumor suppressor gene that has been mutated in several human tumors.
- Trial name
- NCT07778628
- Testing
- immunohistochemical staining with WT1 antibody
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Sohag University
Leukemia, Lymphoma
Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00818961
- Testing
- alemtuzumab, graft-versus-tumor induction therapy, rituximab
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Northside Hospital, Inc.
Solid Tumor
PF-07284892 in Participants With Advanced Solid Tumors
The purpose of this first-in-patient, open label study is to determine the maximum tolerated dose and/or recommended dose for further study of PF-07284892 as a single agent and in combination with lorlatinib, encorafenib and…
- Trial name
- NCT04800822
- Testing
- PF-07284892, lorlatinib, binimetinib
- Where
- 11 sites in United States
- Volunteers
- 53 people
- Sponsor
- Pfizer
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Cutaneous Neurofibroma
Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
This study will evaluate the tolerability and effectiveness of three FDA-approved treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 1064nm laser, a 755nm laser, and a Kybella injection.
- Trial name
- NCT04730583
- Testing
- Kybella, 1064nm laser, 755nm Alexandrite Laser
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Massachusetts General Hospital
Hereditary Breast and Ovarian Cancer Syndrome
Impact of a Psychoeducational Intervention on Expectations and Coping in Young Women Exposed to a High HBOC Risk
Young female counselees (18-40 years) belonging to HBOC families with a known mutation on BRCA-genes or not, receive a lot of information regarding their cancer risk. Information sources are numerous and sometimes contradictory.
- Trial name
- NCT02705924
- Testing
- psychoeducation
- Where
- France
- Volunteers
- 32 people
- Sponsor
- Centre Jean Perrin
Neurofibromatoses
Resiliency Training for Adolescent Neurofibromatosis Patients Via Videoconferencing With Skype
The aim of this research is to adapt the NF-specific adult version of the Skype 3RP for use with adolescents, and to test its feasibility, acceptability, and preliminary effect in improving quality of life, and in decreasing…
- Trial name
- NCT02387177
- Testing
- Stress Management Group 1, Stress Management Group 2
- Where
- United States
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia
Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults
This is a Phase 1, open-label, 2-part study to evaluate the effect of multiple doses of oral carbamazepine or oral itraconazole on the plasma pharmacokinetic profile of radiprodil in healthy adult participants.
- Conditions
- Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia, Other Neurological Disorders
- Trial name
- NCT06965881
- Testing
- Radiprodil with Carbamazepine, Radiprodil with Itraconazole
- Where
- 2 sites in Australia
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- GRIN Therapeutics, Inc.
Secondary or Primary Immunodeficiency
Tolerance and Patients' Satisfaction With IGHy
Primary immunodeficiencies (PID) represent more than 150 diseases affecting the immune system. More than 50% of PIDs are due to a lack or an insufficiency in antibody production.
- Trial name
- NCT03668288
- Testing
- Data record and questionnaires passation
- Where
- France
- Volunteers
- 20 people
- Sponsor
- CHU de Reims
Malignant Peripheral Nerve Sheath Tumors
Imatinib Mesylate Treatment of Patients With Malignant Peripheral Nerve Sheath Tumors
This study assesses the safety and efficacy of imatinib mesylate treatment of patients with malignant peripheral nerve sheath tumors
- Trial name
- NCT00427583
- Testing
- imatinib mesylate
- Where
- 2 sites in Germany
- Volunteers
- 11 people
- Sponsor
- Novartis Pharmaceuticals
Breast Cancer, Hereditary Breast and Ovarian Cancer Syndrome
Protexa® Versus TiLoopBra® in Immediate Breast Reconstruction- A Pilot Study
Randomized controlled trial to look at early complications, cosmetic outcome, patient satisfaction and thickness of the tissue overlying the implant at the lower pole in immediate implant based breast reconstruction after Skin or…
- Trial name
- NCT02562170
- Testing
- TiLoop Bra, Protexa
- Where
- Austria
- Volunteers
- 50 people
- Sponsor
- Medical University of Vienna
Breast Cancer, Chronic Myeloproliferative Disorders
Lactobacillus in Preventing Infection in Patients Undergoing a Donor Stem Cell Transplant for Hematologic Cancer or Myelodysplastic Syndrome
RATIONALE: Probiotics, such as Lactobacillus, may be effective in preventing infections in patients with suppressed immune systems.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00946283
- Testing
- Lactobacillus rhamnosus GG
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Rutgers, The State University of New Jersey
Parenting, Alopecia
Mindful Parenting - A Single Case Experimental Design Study
Some parents of children living with a visible difference can experience heightened stress due to the associated challenges of this. Parent's views of the child's visible difference and their responses to the child are important.
- Conditions
- Parenting, Alopecia, Skin Condition, Limb Difference
- Trial name
- NCT05779527
- Testing
- Mindful Parenting Programme
- Where
- United Kingdom
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Cardiff University
Li Fraumeni Syndrome
LIFSCREEN : Evaluation of Whole Body MRI for Early Detection of Cancers in Subjects With P53 Mutation (Li-Fraumeni Syndrome)
The purpose of this study is to compare the diagnostic efficacy of two follow-up schedules for the early detection of cancers in the population under study on cancer incidence at 3 years.
- Trial name
- LIFSCREEN (NCT01464086)
- Testing
- whole body MRI
- Where
- France
- Volunteers
- 107 people
- Sponsor
- Gustave Roussy, Cancer Campus, Grand Paris
Hereditary Nonpolyposis Colorectal Cancer, Lynch Syndrome
Registry for Women Who Are At Risk Or May Have Lynch Syndrome
The goal of this study is to create a registry of information about women who have or are at risk for Lynch syndrome, in order to study gynecologic cancer risks. This is an investigational study.
- Trial name
- NCT00508573
- Testing
- Questionnaire
- Where
- United States
- Volunteers
- 323 people
- Sponsor
- M.D. Anderson Cancer Center
Lynch Syndrome
Multi-Organ Screening Recommendations in Patients With Lynch Syndrome
The purpose of this study is to examine how people with a family history of colon cancer and other related cancers respond to recommendations for cancer screening after genetic counseling.
- Trial name
- NCT00582296
- Testing
- questionnaires, telephone interview, optional blood drawn, questionnaires, telephone interview and optional blood drawn
- Where
- 8 sites in United States
- Volunteers
- 1,530 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neurofibromatosis 1
Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1
The main goal of this protocol is to develop a well-phenotyped genetic biobank to identify genetic variants associated with the heterogeneous clinical presentations of Neurofibromatosis Type 1 (NF1).
- Trial name
- NCT04941027
- Where
- 2 sites in United States
- Volunteers
- 1,046 people
- Sponsor
- Stanford University
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