Clinical trials: Multiple Primary Neoplasm
Chronic Myeloproliferative Disorders, Leukemia
Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer
RATIONALE: Giving chemotherapy before a donor bone marrow transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00134017
- Testing
- Busulfan, Cyclophosphamide
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Progression of Rheumatoid Arthritis, Osteoarthritis of Multiple Joints
To Examine Whether or Not the Primary Causes of OA or RA Might be Bone Alterations
Generally, osteoarthris (OA) is considered as primarily cartilage disease. However, as we and others have previously reported, a portion of OA might be caused primarily by bone alterations.
- Trial name
- NCT02327650
- Testing
- The examinations for bones in joints of OA and RA patients
- Where
- 2 sites in Japan
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Shinshu University
Multiple System Atrophy
Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France
This multicenter open-label extension study conducted in France is designed to provide ATH434 orally BID to eligible patients who completed the Phase 2 study ATH434-201, and who may benefit from this treatment according to the…
- Trial name
- NCT07729852
- Testing
- ATH434
- Where
- 4 sites in France
- Volunteers
- 7 people
- Sponsor
- Alterity Therapeutics
Leiomyomatosis
Official Record of Patients Diagnosed With Lymphangioleiomyomatosis (LAM)
Pulmonary lymphoangioleiomyomatosis (LAM) is a rare destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor.
- Trial name
- NCT00001869
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Lymphoma
Early Detection of Graft-Versus-Host Disease in Patients Undergoing a Donor Bone Marrow Transplant
RATIONALE: Studying samples of blood in the laboratory from patients who have undergone a donor bone marrow transplant may help doctors learn more about changes that occur in DNA and identify biomarkers related to…
- Trial name
- NCT00898612
- Where
- United States
- Sponsor
- Mayo Clinic
Tuberous Sclerosis, Lymphangioleiomyomatosis
TSC Biosample Repository and Natural History Database
The TSC Biosample Repository collects and stores samples of blood, DNA, and tissues that scientists can request to use in their research. The samples we collect are all linked to clinical data in the TSC Natural History Database.
- Trial name
- NCT05676099
- Testing
- Phlebotomy, Buccal (cheek) swab, Genetic Testing
- Where
- 26 sites in United States, Canada
- Volunteers
- 5,000 people
- Sponsor
- National Tuberous Sclerosis Association
Anemia, Chronic Myeloproliferative Disorders
Busulfan and Fludarabine Before Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Anemia, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00301912
- Testing
- filgrastim, busulfan, fludarabine phosphate
- Where
- United States
- Sponsor
- University of California, San Francisco
Chronic Myeloproliferative Disorders, Leukemia
Interleukin-12 Followed by Interferon Alfa in Treating Patients With Advanced Cancer
Phase I trial to study the effectiveness of combining interleukin-12 and interferon alfa in treating patients who have residual, recurrent, or metastatic malignant melanoma or other advanced cancer that has not responded to…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00003451
- Testing
- recombinant interferon alfa, recombinant interleukin-12
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Cancer Institute (NCI)
VHL Pancreatic Neuroendocrine Tumors, Von Hippel-Lindau Disease
Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine Tumors
Background: People with von Hippel-Lindau (VHL) can have problems with a variety of organs, such as the pancreas. The disease can cause tumors of the pancreas. This can result in life-threatening complications.
- Conditions
- VHL Pancreatic Neuroendocrine Tumors, Von Hippel-Lindau Disease, Neuroendocrine Tumors
- Trial name
- NCT04074135
- Testing
- 68-Gallium DOTATATE
- Where
- United States
- Volunteers
- 740 people
- Sponsor
- National Cancer Institute (NCI)
Primary Ciliary Dyskinesia, Kartagener Syndrome
Effect of Game Based Approach on Oxygenation, Functional Capacity and Quality of Life in Primary Ciliary Dyskinesia
Impaired pulmonary function, decreased physical activity, functional capacity and depending on these factors are reported in patients with primary ciliary dyskinesia in recent studies.
- Conditions
- Primary Ciliary Dyskinesia, Kartagener Syndrome, Immotile Cilia Syndrome
- Trial name
- NCT03832491
- Testing
- Home based therapy, Game and home based therapy
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Hacettepe University
Insulin-like Growth Factor-1 Deficiency
rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency
IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary).
- Trial name
- NCT00572156
- Testing
- NutropinAq® (Somatropin [rDNA origin]), Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])
- Where
- United States
- Volunteers
- 106 people
- Sponsor
- Ipsen
Cancer
Heparin or M-EDTA in Preventing Catheter-Related Infections and Blockages in Patients at High Risk for a Catheter-Related Infection
RATIONALE: Heparin or M-EDTA may prevent catheter-related infections and blockages in patients at high risk for a catheter-related infection.
- Trial name
- NCT00378781
- Testing
- Heparin, Minocycline-EDTA
- Sponsor
- M.D. Anderson Cancer Center
Primary Ciliary Dyskinesia, Kartagener Syndrome
Swiss Primary Ciliary Dyskinesia Registry
The Swiss Primary Ciliary Dyskinesia (PCD) Registry is a national patient registry that collects information on diagnosis, symptoms, treatment and follow-up of patients with PCD in Switzerland and provides data for national and…
- Trial name
- CH-PCD (NCT03606200)
- Where
- Switzerland
- Volunteers
- 800 people
- Sponsor
- University of Bern
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
Severe Combined Immunodeficiency
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat…
- Trial name
- NCT01129544
- Testing
- Gene transfer
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- David Williams
Immune Defect, 22q11.2 Deletion Syndrome
Resolution of Primary Immune Defect in 22q11.2 Deletion Syndrome
Evaluate about age of resolution in immune defect in 22q11.2 Deletion Syndrome Incidence of immunodeficiencies in 22q11.2 Deletion Syndrome
- Trial name
- NCT02460328
- Where
- Thailand
- Volunteers
- 43 people
- Sponsor
- Mahidol University
Diphtheria, Tetanus
A Phase II/III Study to Evaluate the Immunogenicity, Safety and Lot-to-lot Consistency of LBVD, a Fully Liquid Hexavalent Diphtheria-tetanus-whole Cell Pertussis-hepatitis B-poliovirus-Haemophilus Influenzae Type b Conjugate (DTwP-HepB-IPV-Hib) Vaccine, in Healthy Infants as Primary Series
The purpose of this study is to evaluate immunogenicity, safety and lot-to-lot consistency of LBVD in comparison to co-administration of Pentavalent vaccine and Poliomyelitis Vaccine (Inactivated) in separate injections at four…
- Conditions
- Diphtheria, Tetanus, Pertussis, Hepatitis B
- Trial name
- NCT06947499
- Testing
- LBVD, Pentavalent vaccine and Inactivated Polio vaccine
- Where
- Philippines
- Volunteers
- 1,186 people, healthy volunteers welcome
- Sponsor
- LG Chem
Rare Disorders
Channelling Algorithm Proficiency To Understand, Recognise and Elucidate Disease
Rare diseases affect over 3.5 million people in the UK. It can take years of multiple referrals, inconclusive tests or incorrect diagnoses, for patients to get a final diagnosis.
- Trial name
- CAPTURED (NCT07130071)
- Volunteers
- 500 people
- Sponsor
- Research in Real-Life Ltd
Tuberous Sclerosis, Neurofibromatoses
Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC)…
- Conditions
- Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
- Trial name
- NCT01031901
- Testing
- Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- PACIFIC (NCT05364021)
- Testing
- LP352
- Where
- 34 sites in United States, Australia
- Volunteers
- 52 people
- Sponsor
- Longboard Pharmaceuticals