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Epinephrine

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ENV-101 (taladegib) tablets, 200 mg once-daily in 28-day cycles

ENV-101 (taladegib) tablets, 300 mg once-daily in 28-day cycles

ENV-101 (taladegib)

Paul Frohna, M.D., Ph.D.

This study employs a 2-stage design that aims to evaluate the efficacy and safety of ENV- 101, a potent Hedgehog (Hh) pathway inhibitor, in patients with refractory advanced solid tumors characterized by loss of function (LOF) mutations in the Patched-1 (PTCH1) gene. Stage 1 of this study will enroll approximately 44 patients randomized between two dose levels. As appropriate, Stage 2 of the study will expand enrollment based on the results of Stage 1.

A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations

A Phase 2, Multi-Center Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations

ORR is comprised of Complete Response (CR) and Partial Response (PR), measured by Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST 1.1), as determined by an independent review (confirmed CR or PR will be defined as a repeat assessment performed no less than 28 days after the criteria for response is first met).

Unacceptable toxicities = non-hematologic AEs \>= Grade 3 in severity, not including alopecia and fatigue.

Best overall response of confirmed CR or PR as determined by the treating Investigator using RECIST 1.1.

CBR = the proportion of patients who achieved CR, PR or stable disease as determined by the treating Investigator using RECIST 1.1.

OS = Number of months from initiation of study medication to the date of death due to any cause or last follow up.

DOR = the number of months from the start of CR or PR, whichever response is recorded first and subsequently confirmed, to the first date that recurrent or progressive disease is documented or death.

PFS = number of months from initiation of study medication to the earlier of disease progression or death due to any cause or last follow up.

Percentage change in inhibition of Gli1, a marker for inhibition of the Hedgehog pathway, in skin biopsies.

Measurement of trough concentration of study medication in blood at Baseline and the end of each 28-day treatment cycle

Endeavor Biomedicines, Inc.

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Symptoms and General Pathology

Wilms Tumor

Neoplasms by Histologic Type

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Genetic Diseases, Inborn

Wilms' Tumor

Mahmoud Elzembely, MD

The goal of this cross sectional observational study is to determine the percentage of Wilms tumors among pediatric cancers at Shefa Al-Orman Children Cancer Hospital from june 2020 to june 2024,to study the outcome of patients with Wilms tumor treated according to SIOP Umbrella protocol during the study period and to study the treatment related complications during therapy according to common terminology criteria of adverse events version 5.0 (CTCAE Version 5.0).

Research Design and Methods:

Study design: cross sectional study Study setting: Shefa Al-Orman Children Cancer hospital Study period: from June 2020 to June 2024

Study Objectives:

1. To determine the percentage of Wilms tumors among pediatric cancers.
2. To study the outcome of patients with Wilms tumor treated according to SIOP Umbrella protocol (1).
3. To study the treatment related complications during therapy according to common terminology criteria of adverse events version 5.0 (CTCAE Version 5.0) (2).

Data collection: Each patient is subjected to all the following according to the centre policy:

I. History and physical examination at presentation:

1. Demographic data as: Age, sex, residence and date of diagnosis.
2. Clinical history and Physical examination including

   * Initial signs and symptoms of pediatric renal tumors including palpable abdominal mass, abdominal pain, hypertension, hematuria, weight loss, constipation, diarrhea, urinary tract infection, previous trauma....etc.
   * Associated congenital anomalies including. Aniridia, genitourinary anomalies, hemihypertrophy
   * Family history of cancer

II. Laboratory studies done for diagnosis of renal tumors at presentation:

- Complete blood count: presence/absence of polycythemia, anemia, thrombocytopenia

* Serum chemistries: blood urea nitrogen, creatinine, uric acid, Alanin transaminases, aspartate aminotransferase, serum bilirubin.
* Assessment of coagulation factors: prothrombin time, partial thromboplastin time

III. Imaging Studies:

1. Abdominal ultrasound
2. Abdominal CT scan or MRI with special attention to

   \* Presence and function of the opposite kidney

   \*Evidence of bilateral lesions

   \*Evidence of involvement of renal vein or inferior vena cava with tumor

   \*Lymph node involvement

   \*Liver metastasis
3. Chest computed tomography scan.
4. Echocardiography: To

   * Assess myocardial contractility before starting cardiotoxic chemotherapy.
   * Detect the presence of tumor in the inferior vena cava or right atrium

IV. Pathology:

1. Ultrasound or CT guided True cut needle biopsy is indicated in the following conditions:

   A- Unusual clinical presentations:

   - Age \> 5-6 years or less than 6 months

   B- Unusual findings by imaging:

   - Calcification

   - Voluminous adenopathies

   - Renal parenchyma not visible

   - Almost totally extrarenal process

   C- Contraindications for the use of Needle Biopsy:
   * Suspicion of rupture or hemorrhage
   * Needle biopsy is unlikely to be of benefit in pure cystic structures with no solid component. Immediate surgery to establish the diagnosis is recommended in such cases.

   V. Reporting of treament related complications include:

   Chemotherapy toxicity will be determined according to WHO Common Terminology Criteria for Adverse Events (CTCAE Version 5.0, 2017) (2).

   VI. Treatment: Patients are treated according to SIOP Umbrella protocol (1)

Study of Wilms Tumors Among Patients of Shefa Al-Orman Children Cancer Hospital

will be calculated from date of diagnosis to last date of follow up or date of death for any cause

will be measured from the date of Wilms tumor diagnosis to the first treatment failure or date of last follow-up. Treatment failure included abandonment of treatment, death, and progressive or relapsed disease.

Shefa Al-orman Children Cancer Hospital

Assiut University

South Egypt Cancer Institute

Lecturer of Pediatric Hematology Oncology

Carcinoma, Renal Cell

Renal Cell Carcinoma

Cohort study using one arm, no comparator group is possible or feasible

Will the use of ICG delineate the margin between normal kidney parenchyma and renal tumour in children at a macroscopic level and can this be replicated at a microscopic level

Indocyanine Green (ICG)

max pachl

Sarah Hadfield

Max Pachl

Indocyanine Green (ICG) is a dye which fluoresces under near-infrared (NIR) light. It has been used for several applications in adult surgery. The CI is pioneering its use in children's kidney cancer surgery for lymph node identification and removal. This study concentrates on its use for procedures where only the part of the kidney containing tumour is removed. It is known that kidney tumours in both adults and children do not take up ICG at all. This absence of uptake can be used to define the border between normal and abnormal renal tissue giving a real-time picture of the area of tumour. This then delivers surgeons an intra-operative roadmap for removing only the cancerous part of the kidney. At present the international society of paediatric oncology - renal tumour study group (SIOP-RTSG) protocol, which is followed in the UK, advises consideration of partial nephrectomy for children with bilateral renal tumours and in children with unilateral tumours who have a renal tumour predisposition syndrome. There is ongoing debate about partial nephrectomy in unilateral renal tumour surgery in children who do not have a predisposition syndrome.

This study aims to provide the evidence that paediatric renal tumours do not take up ICG at a naked-eye level and confirm this at a cell level. ICG will be infused into kidneys containing tumour once they have been removed from the patient, The kidney and tumour will be observed under NIR light to show where the areas of fluorescence are. Then, a pathologist will prepare the specimen in theatre, in the same way they would do in the lab. The specimen would be bivalved and reviewed under NIR. Microscopy specimens of the border between normal and abnormal tissue would then be reviewed with an NIR capable microscope. The standard histopathological assessment would then take place.

This study will ascertain if paediatric renal tumours are ICG avid or not. This is critical for determining what the value of using ICG/NIRF will be during nephron sparing surgery (NSS)/ partial nephrectomy in children who have renal tumours that are suitable for this type of surgery. If the normal renal parenchyma is ICG avid but the tumour is not (as has been shown for RCC in adults) then we can conclude that if a fluorescent margin left in-situ on the tumour, then it would be completely resected. Early work at St. Jude has suggested this technique shows promise, but it has not been validated prospectively at a macro or microscopic level.

This is important because in children with unilateral tumours, total nephrectomy has been performed routinely due to concern over leaving behind tumour tissue. If tumour tissue is left behind the patient is upstaged and requires radiotherapy with the consequent sequelae that confers. Radiotherapy to this area invariably damages the kidney so much that it is essentially non-functioning and avoiding this exposure would clearly be ideal. In children with bilateral tumours, retaining as much renal tissue as possible whilst facilitating a complete resection is the goal of surgery. If ICG/NIRF can help with this then it will change the game for this population of patients In addition, this technique will aid in retaining as much normal parenchyma as possible giving the patient better long-term outcomes for renal function. As a further advantage, the study may ascertain if ICG can differentiate between non-cancerous nephrogenic rests (NRs), normal renal parenchyma, and tumour. The difference between these types of tissue is critical when doing NSS because currently only formal histopathological assessment can reliably differentiate NRs from normal tissue or tumour. This is because the definition hinges on whether the lesions have a capsule or not. This is not identifiable on imaging or on core biopsy. The vital feature is that NR's do not require resection, whereas tumour obviously does.

Avidity for ICG may be different in different types of tumours and this will be recorded using the intensity mapping feature of the Storz Rubina Opal1 system. At a cellular level it will be important to investigate why ICG does not cross into tumour and whether this is due to the tumour capsule preventing or limiting vascular flow, or some other factor.

ICG does not affect the histopathological assessment and this research study will validate whether ICG survives the fixing process as this has not been assessed with an NIR capable microscope.

Single centre cohort study using consecutive patients. No comparison group.

Nephrogreen. Appearances of Exvivo Renal Tumours Under Near-infrared

An Open Label Cohort Study Assessing the Near Infrared Fluoroscopic Macro and Microscopic Appearances of Paediatric Renal Parenchyma and Tumours Following Ex-vivo Injection of Indocyanine Green

all IPD that underlie results in a publication

Macroscopic comparison assessment of ICG avidity using Storz Rubina NIR between normal renal parenchyma and tumour.

Measurement of the quantifiable level of avidity using intensity mapping on a Karl Storz Endoskope™ Opal 1 Rubina system.

Microscopic comparison of ICG avidity between tumour, non-tumour and nephrogenic rests using a nearinfrared microscope.

Children's Cancer and Leukaemia Group

Birmingham Women's and Children's NHS Foundation Trust

Neoplasm Metastasis

Sarcoma, Ewing

Neoplasms, Connective and Soft Tissue

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Myosarcoma

Ewing Sarcoma

Injection of Indocyanine Green at doses detailed on summary of product characteristics for each age range

Intravenous or intraparenchymal injection

Indocyanine green

Indo-cyanine green (ICG) is a dye that has been used for a variety of adult and paediatric uses since 1956. Over the past few years, near infrared (NIRF) technology has been developed which allow is use as a fluorescence agent during surgery. It has been used increasingly in the field of adult oncology surgery and has been shown to increase the efficacy of this surgery.

The aim of this study is to evaluate the use of NIRF and ICG during specific minimally invasive surgery (MIS) procedures within paediatric oncology surgery. Their use will complement existing surgical techniques rather than replace them.

Given the published advantages in adults this study aims to provide evidence of feasibility in the paediatric patients with cancer.

The purpose of this study is to show the feasibility of using ICG in combination with NIRF and MIS in paediatric oncology surgery. It has been used increasingly over the past few years for a range of adult oncology surgical indications including tumour margin identification, lymph node identification and pulmonary metastectomy. It will be investigated in three groups of patients in our study:

1. Tumour margin identification-Identifying the margins of a tumour intra-operatively is crucial for many paediatric cancers. Incomplete resections or resections with involved margins often require upstaging of treatment with more intensive chemotherapy or radiotherapy. ICG will be injected intravenously and then NIRF will be used intermittently until fluorescence is achieved and an evaluation of the tumour and its margins will be performed.
2. Lymph node identification - It is standard of care for most oncology resections to remove lymph nodes that are suspected to be involved with tumour. Both SIOP and COG recommend the removal of 7 or more lymph nodes during a nephrectomy for Wilm's tumour although this is often not achieved. IGC will be injected directly into the tumour (or the adjacent tissue in 2-4 places) and then NIRF will be used intermittently until fluorescence is achieved. An evaluation of the tumour and any fluorescent lymph nodes will then take place and any fluorescent lymph nodes will be removed.
3. Pulmonary metastectomy - Many paediatric cancers including Wilm's tumour have a propensity to metastasise to the lungs. Removal of all lung metastases is important as it has the potential to down-stage tumours, obviating the need for pulmonary radiotherapy.

Modern radiology techniques can identify lesions as small as 1mm which are often not palpable at the time of surgery. Relying on finger palpation requires patients to have a thoracotomy with all of the associated complications. ICG will be injected intravenously and then NIRF will be combined with MIS to identify any fluorescent lesions. The location of any lesions will be compared with those seen on pre-surgical imaging (current standard practice).

For all three groups of patients, the use of ICG and NIRF will complement rather than replace existing surgical technique in patients who require surgery already. Data will be collected on the number of lesions which do/do not fluoresce as well as the histology of lesions removed. They will be separated into lesions which do not fluoresce, lesions which were removed before fluorescence (they will be checked for ex-vivo fluorescence) and fluorescing lesions so that the histological characteristics of each group can be compared.

open label, single centre, single arm, prospective feasibility study

Indo-cyanine Green (ICG) in Paediatric Oncology MIS

An Open Label, Single Centre, Single Arm, Prospective Feasibility Study Evaluating the Effectiveness of Near-infrared Fluorescence (NIRF) Using Indo-cyanine Green (ICG) in Minimally Invasive Paediatric Oncology Surgery (MIS)

Number of fluorescent nodes resected per patient

Subjective surgeon analysis of intra-operative conditions on a Likert scale per patient

Comparison of number of lesions seen on CT with those seen with fluoroscopy per patient

Number of fluorescent nodes and metastases containing viable tumour per patient

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations	NCT05199584	Solid Tumors Wi...	ENV-101 (talade...	18 Years -	Endeavor Biomedicines, Inc.
Study of Wilms Tumors Among Patients of Shefa Al-Orman Children Cancer Hospital	NCT06288802	Wilms Tumor		- 18 Years	South Egypt Cancer Institute
Nephrogreen. Appearances of Exvivo Renal Tumours Under Near-infrared	NCT05735977	Wilms Tumor Renal Cancer	Indocyanine Gre...	1 Day - 15 Years	Birmingham Women's and Children's NHS Foundation Trust
Indo-cyanine Green (ICG) in Paediatric Oncology MIS	NCT04854018	Pediatric Renal... Metastatic Oste... Metastatic Ewin... Pulmonary Metas... Rhabdomyosarcom... Non-Rhabdo. Sof...	Indocyanine gre...	1 Day - 15 Years	Birmingham Women's and Children's NHS Foundation Trust

Spots Global Cancer Trial Database for wilms

Every month we try and update this database with for wilms 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 wilms

Every month we try and update this database with for wilms 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 wilms 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.