Nervous System Diseases

All Conditions

Symptoms and General Pathology

Neoplasms

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Epilepsy

Sclerosis

Disease Progression

Tuberous Sclerosis

Brain Diseases

Central Nervous System Diseases

Hamartoma

Neoplasms, Multiple Primary

Syndrome

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Tuberous Sclerosis Complex

All Drugs and Chemicals

Anti-Infective Agents

Antineoplastic Agents

Tin Fluorides

Sirolimus

Anti-Bacterial Agents

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antifungal Agents

Immunosuppressive Agents

Immunologic Factors

The investigational drug product to be used in this study is sirolimus, provided in oral suspension.

Placebo

Molly S Griffith, BA

Jessica Krefting, RN

E. Martina Bebin, MD, MPA

Angela Martinez

Rajsekar Rajamaran, MD, MS

Jennifer Winterbottom

Brenda Porter, MD

Emine Arcasoy

Mustafa Sahin, MD, PhD

Olga Novak

Michael Wong, MD, PhD

Hannah Riehl

Jamie Capal, MD

Darcy A Krueger, MD, PhD

Alexis Rodriguez

Hope Northrup, MD

Mikaela Morales

Stephanie Randle, MD

Martina Bebin, MD, MPA

This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.

This study is supported by research funding from the Office of Orphan Products Division (OOPD) of the US Food and Drug Administration (FDA).

Tuberous Sclerosis Complex (TSC) is caused by genetic mutation in TSC1 or TSC2, resulting in dysregulation of the mechanistic target of rapamycin (mTOR) signaling pathway. Age at time of seizure onset in TSC infants has been linked to long-term neurodevelopmental outcome in this high-risk population. Sirolimus is an mTOR inhibitor used to treat many of the symptoms of TSC, including epilepsy. This will be the first study to truly evaluate a targeted, disease-modifying drug therapy for preventing or delaying seizure onset in TSC using a rational, mechanism-based therapeutic approach.

This trial will employ a randomized, double-blind, placebo-controlled multisite design to evaluate the safety and efficacy of early sirolimus treatment to prevent or delay seizure onset in TSC infants.

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

Time to seizure onset, comparing sirolimus with placebo

Percentage of subjects reporting severe (CTCAE v5.0 grade \>= 3) adverse event (AE) or serious adverse event (SAE), comparing sirolimus with placebo.

Neurodevelopmental outcomes at the end of treatment, comparing sirolimus with placebo.

Patient and caregiver quality of life, comparing sirolimus with placebo.

EEG measures of neuronal connectivity, comparing sirolimus with placebo.

MRI measures of neuronal connectivity, comparing sirolimus with placebo.

Validate the feasibility and effectiveness of sirolimus precision dosing in infants with TSC

Darcy Krueger

IND Sponsor/Lead Principal Investigator

Hemangioma

Hemangioma, Capillary

Port-Wine Stain

Neoplasms, Vascular Tissue

Neoplasms by Histologic Type

Skin Abnormalities

Skin Diseases

Pharmaceutical Solutions

Anti-Arrhythmia Agents

Antihypertensive Agents

Timolol

Adrenergic beta-Antagonists

Adrenergic Agents

Adrenergic Antagonists

Neurotransmitter Agents

Half of the enrolled subjects (intervention group) will receive topical 0.5% Timolol.

Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily

topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily

topical 0.5% Timolol

Control (placebo) group

Alfons L. Krol, M.D.

The purpose of this study is to learn about a new potential use for topical timolol 0.5% aqueous solution that may help treat small uncomplicated infantile hemangiomas. This study would examine whether topical timolol could be a potential therapy.

At the first visit, subjects will fill out a questionnaire that asks about the child's history and the hemangioma. Photographs and measurements will be taken at this and each subsequent visit. At the first visit, subjects will have a physical exam, including vital signs, height and weight. An EKG will also be done. Study drug will be dispensed and instructions given. Half of the subjects will receive a placebo. This is a blinded randomized study. Follow-up visits will be weekly for the first two weeks (three visits total including the first visit), then monthly until the study is over. Photographs and measurements will again be taken at each visit. Subjects will be asked to evaluate the size and the color of the hemangioma against the first photographs and fill out forms regarding any changes in behavior and asked about any side effects.

Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas

Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas: A Prospective Double Blinded Placebo Controlled Study

This will be generated by asking each of the assessors to score the improvement using a visual analog scale (VAS) assessing the decrease in size of hemangioma by comparing photographs at different times of treatment. The assessors will score this improvement into one of the following categories: 0-24%, 25-49%, 50-74%, \>75%.

1. The proportion of subjects in the treatment group as compared to the placebo control group with at least 50% improvement in the extent of the hemangioma.
2. The difference between the extent/size of the hemangioma as an outcome measure versus color changes.
3. Frequency of adverse events (e.g. hypotension, behavioral changes, etc.), collected by the investigator and reported by the parents.

Oregon Health and Science University

Professor

Half of the enrolled subjects (intervention group) will receive topical 0.5% Timolol.

topical 0.5% Timolol: topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily

Topical 0.5% Timolol

Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily

Control (placebo) group: Control (placebo) group

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Dr. Alfons Krol

Proportion of Subjects in Treatment Group Compared to Placebo Group With at Least 75% Improvement in the Extent of the Hemangioma as Compared to Baseline Photos.

Compare Treatment Group to Control Group Improvement Assessments

Overall Study

Blood and Lymph Conditions

Immune System Diseases

Leukemia

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Lymphoma

Hematologic Diseases

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Acute Graft Versus Host Disease

Childhood Acute Lymphoblastic Leukemia

Lymphosarcoma

Immunomodulating Agents

Blinatumomab

two-stage stratification into risk groups: Initially and According to the results of induction therapy MRD-positive patients receive a course of blinatumomab and HSCT.

the risk-adapted choice of therapy and the use of a combination of chemotherapy with immunotherapy and hematopoietic stem cell transplantation for patients with risk factors.

Natalya f Myakova, PD

The innovation of this protocol is the risk-adapted choice of therapy and the use of a combination of chemotherapy with immunotherapy and hematopoietic stem cell transplantation for patients with risk factors. we have proposed a two-stage stratification into risk groups:

Initially:

* Standard risk: patients with no rearrangement of the KMT2A gene.
* Intermediate risk: patients with rearrangement of the KMT2A gene without damage to the central nervous system.
* High risk: patients with rearrangement of the KMT2A gene with lesions of the central nervous system.

According to the results of induction therapy:

* The high-risk group includes patients from the standard risk group with an MRD level of more than 0.1% after the induction course and from the intermediate risk group with MRD-positive (PCR) after HR1 block.
* The allocation of children in the first year of life without the rearranged KMT2A gene into a separate group seems to be logical, since the prognosis in this group is better than in children with the rearranged KMT2A gene. In this protocol, non-intensive therapy with consolidations and maintenance therapy remains for those who achieve a low MRD level (less than 0.1%) after a course of induction. The rest of the patients move into a high-risk group: they receive blinatumomab and HSCT.
* The concept of therapy for patients at intermediate risk is based on the rate at which MRD-negativity is achieved: standard consolidation and maintenance therapy for those who became MRD-negative at the end of induction, "block" chemotherapy for those who were positive at the end of induction, but achieved negativity after HR1 block, blinatumomab with HSCT for those who have preserved the MRD after the HR1 block.
* For high-risk patients, a combination of immunotherapy (blinatumomab - a bispecific CD3 / CD19 T-cell activator) and HSCT in the first remission was chosen.

* Standard risk group:
* Induction of remission: 36 days of dexamethasone, 5 weekly injections of vincristine, 2 injections of daunorubicin on days 8 and 22, a single injection of pegelated asparaginase on days 5-7 and 6 weekly intrathecal injections of three drugs (methotrexate, cyamethosar and dexamethason ).
* Further therapy in this therapeutic group depends on the status of remission, the level of MRD on the 36th day of therapy.
* MRD-negative patients receive consolidation therapy in the amount of 3 consolidations (6-mercaptopurine, methotrexate, peg-asparaginase, daunorubicin) with re-induction courses (dexamethasone, vincristine) and maintenance therapy (6-mercaptopurine, methotrexate).
* MRD-positive patients receive a course of blinatumomab and HSCT.
* Intermediate risk group:
* Induction of remission: 36 days of dexamethasone, 5 weekly injections of vincristine, 2 injections of daunorubicin on days 8 and 22, a single injection of pegelated asparaginase on days 5-7, 6 weekly intrathecal injections of three drugs (methotrexate, cyamethosar, dexamethasone).
* Further therapy in this therapeutic group depends on the status of remission on the 36th day of therapy.
* Patients who have achieved molecular remission receive consolidation therapy in the amount of 3 consolidations with re-induction courses and maintenance therapy.
* Patients who have not achieved molecular remission receive HR1 block. Further therapy depends on the remission status after HR1 block. Patients who have not achieved molecular remission receive a course of blinatumomab and HSCT, patients who have achieved molecular remission, two more blocks HR2 and HR3, protocol II and maintenance therapy.
* High risk group:
* Induction of remission: 36 days of dexamethasone, 5 weekly injections of vincristine, 2 injections of daunorubicin on days 8 and 22, a single injection of pegelated asparaginase on days 5-7, 6 weekly intrathecal injections of three drugs (methotrexate, cyamethosar, dexamethasone).
* Further therapy in this therapeutic group does not depend on the status of remission on the 36th day of therapy.
* All patients receive HR1 block, blinatumomab course and HSCT (subject to morphological remission).

Combined Immuno-chemotherapy for Patients With B-linear Acute Lymphoblastic Leukemia Diagnosed From 0 to 365 Days of Life (ALL-Baby-2021)

Prospective Single Group Study Combined Immuno-hemotherapy for Patients With B-linear Acute Lymphoblastic Leukemia Diagnosed From 0 to 365 Days of Life (ALL-Baby-2021)

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Brain Neoplasms

Central Nervous System Neoplasms

Nervous System Neoplasms

Reduced-dose Craniospinal Radiotherapy Followed by High-dose Chemotherapy and Autologous Stem Cell Rescue in Children with Newly Diagnosed High-risk Brain Tumor

High-dose Chemotherapy and Autologous Stem Cell Rescue in Infants and Young Children with Newly Diagnosed High-risk Brain Tumor To Avoid or Reduce Craniospinal Radiation

High-dose Chemotherapy and Autologous Stem Cell Rescue in Children with Recurrent Brain Tumor or Non-germinomatous Germ Cell Tumor with Inadequate Response to Conventional Treatment

Surgery -\> enroll Pre-RT chemotherapy (2 cycles: A1, B1) PBSC collection during first cycle (A1) (2nd look surgery if necessary) RT (Reduced dose CSI) Post-RT chemotherapy (4 cycles: A2, B2, A3, B3) (if relapse or progression prior to HDCT, off study -\> enroll S-083 protocol) HDCT1 (CTE) HDCT2 (CM)

high-dose chemotherapy and autologous stem cell rescue

Surgery pre-HDCT chemotherapy (6 cycles: A1, B1, A2, B2, A3, B3) PBSC collection during first cycle of chemotherapy (A1) (2nd look surgery if necessary) (if relapse or progression prior to HDCT, off study -\> enroll S-083 protocol) HDCT1 (CTE) HDCT2 (CM) (RT if necessary) Observe

(Surgery if possible) Chemotherapy (4 cycles) PBSC collection during first cycle of chemotherapy during 4th cycle of chemotherapy (if BM involvement) (RT, if possible, after PBSC collection) (if less than PR prior to HDCT, off study) HDCT1 (CTE) HDCT2 (CM)

Ki Woong Sung

* Study No.: KSPNO-S-081 Reduced-dose Craniospinal Radiotherapy Followed by High-dose Chemotherapy and Autologous Stem Cell Rescue in Children with Newly Diagnosed High-risk Brain Tumor
* Study No.: KSPNO-S-082 High-dose Chemotherapy and Autologous Stem Cell Rescue in Infants and Young Children with Newly Diagnosed High-risk Brain Tumor To Avoid or Reduce Craniospinal Radiation
* Study No.: KSPNO-S-083 High-dose Chemotherapy and Autologous Stem Cell Rescue in Children with Recurrent Brain Tumor or Non-germinomatous Germ Cell Tumor with Inadequate Response to Conventional Treatment

Although significant progress has been made in the treatment of brain tumors, the prognosis remains dismal in patients with relapsed tumor. The outlook for infants and young children is also poor, primarily because of the limited use of radiotherapy, although a recent report suggested that vigorous combination chemotherapy alone improved the survival of young children without macroscopic metastases at diagnosis. The prognosis is also not satisfactory when a large residual tumor remains after surgery or when leptomeningeal seeding is present at diagnosis. Given the above situation, we plan to explore the possible efficacy of high-dose chemotherapy and autologous stem cell rescue in patients with high-risk embryonal tumors, relapsed brain tumors and in infants and young children with brain tumors.High-dose chemotherapy and autologous stem cell rescue has improved the survival of children with high-risk solid tumors. this treatment strategy is based on the hypothesis that a dose escalation might improve the survival of children with high-risk solid tumors.Many investigators demonstrated that further dose escalation using sequential high-dose chemotherapy and autologous stem cell rescue might result in additional improvements in the survival of patients with high-risk tumors. As embryonal brain tumors are a chemosensitive tumors, a strategy using high-dose chemotherapy might be effective in the treatment of high-risk embryonal brain tumors and relapsed brain tumors. In addition, it might defer or eliminate irradiation in infants and young children with brain tumors

High-dose Chemotherapy With Autologous Stem Cell Rescue in Pediatric High-risk Brain Tumors

Multicenter Clinical Trial to Evaluate Efficacy of High-dose Chemotherapy With Autologous Stem Cell Rescue for Children With High-risk Brain Tumors

KSPNO stem cell transpantation committee

Samsung Medical Center

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study	NCT05104983	Tuberous Sclero... Epilepsy	Sirolimus Placebo	1 Day - 6 Months	Children's Hospital Medical Center, Cincinnati
Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas	NCT01147601	Hemangioma Infant	topical 0.5% Ti... Control (placeb...	1 Month - 8 Months	Oregon Health and Science University
Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas	NCT01147601	Hemangioma Infant	topical 0.5% Ti... Control (placeb...	1 Month - 8 Months	Oregon Health and Science University
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study	NCT05104983	Tuberous Sclero... Epilepsy	Sirolimus Placebo	1 Day - 6 Months	Children's Hospital Medical Center, Cincinnati
Combined Immuno-chemotherapy for Patients With B-linear Acute Lymphoblastic Leukemia Diagnosed From 0 to 365 Days of Life (ALL-Baby-2021)	NCT05029531	Acute Lymphobla... ALL, Infants	the risk-adapte...	1 Day - 365 Days	Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas	NCT01147601	Hemangioma Infant	topical 0.5% Ti... Control (placeb...	1 Month - 8 Months	Oregon Health and Science University
High-dose Chemotherapy With Autologous Stem Cell Rescue in Pediatric High-risk Brain Tumors	NCT00798811	Brain Tumor	high-dose chemo... high-dose chemo... high-dose chemo...	-	Samsung Medical Center

Spots Global Cancer Trial Database for infant

Every month we try and update this database with for infant cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for infant

Every month we try and update this database with for infant cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for infant cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.