Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||3 Years - 25 Years|
- - Patients must have histologically confirmed progressive medulloblastoma, malignant glioma or any other recurrent/progressive malignant brain tumor, for which curative measures do not exist.
- - Patients with DIPG and DMG with H3K27M who are post-radiation but have not exhibited tumor progression are also eligible.
- - Patients must have previously undergone standard-of-care treatment including surgery, radiation, and/or first line adjuvant chemotherapy prior to the experimental treatment (WP1066).
- - Patients must have recovered from the acute treatment related toxicities (defined as < grade 1 if not defined in eligibility criteria) of all prior chemotherapy, immunotherapy or radiotherapy prior to entering this study.
- - Age > 3 to 25 years.
- - Karnofsky or Lansky Performance Scale score > 60%.
- - Patients must have normal organ and marrow function as defined below: - Absolute neutrophil count> 1,000/mcL.
- - Platelets> 100,000/mcL.
- - Total bilirubin within normal institutional limits.
- - AST(SGOT)/ALT(SGPT)< 5 x (<10 x if taking steroids) institutional upper limit of normal.
- - creatinine within normal institutional limits for age OR creatinine clearance> 60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal.
- - PT/PTT< 1.5 x normal institutional standard.
- - Patients with stable seizures (e.g., no seizures for ≥ 14 days and not requiring escalation or addition of anti-epileptic drugs) will be eligible.
- - Signed informed written consent obtained from patient if 18 years of age or older, or from guardian/legal representative if patient is less than 18 years of age.
- - Patients must have received their last dose of known myelosuppressive anticancer therapy at least three (3) weeks prior to study enrollment or at least six (6) weeks if prior nitrosourea.
- - Biologic or investigational agent (anti-neoplastic): Patient must have received their last dose of the investigational or biologic agent ≥ 7 days prior to study enrollment.
- - Immunotherapy: Patient must have completed immunotherapy (e.g. tumor vaccines, oncolytic viruses.
- - Radiation: Patients must have had their last fraction of: - Craniospinal irradiation ≥ 3 months prior to enrollment.
- - Other substantial bone marrow irradiation ≥ 6 weeks prior to enrollment.
- - Local palliative XRT (small port) ≥2 weeks.
- - Stem Cell Transplant: Patient must be ≥ 12 weeks since autologous bone marrow/stem cell transplant prior to enrollment.
- - Surgery Patients must be fully recovered from all acute effects of prior surgical intervention.
- - History of allergic reactions attributed to compounds of similar chemical or biologic composition to WP1066.
- - The enzymatic metabolism profile of WP1066 is unknown.
- - Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- - No single lesion can be larger than 5 cm in maximal diameter.
- - The effects of WP1066 on the developing human fetus are unknown.
- - HIV-positive patients receiving combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with WP1066.
- - The potential for further hemorrhaging with the use of WP1066 is unknown.
- - Patients requiring escalation of the corticosteroid dose will be excluded, but patients receiving a stable or decreasing dose for at least one week prior to registration will be eligible.
- - The cardiac toxicities of WP1066 are unknown.
- - Patients with uncontrolled seizures or seizure requiring escalation or addition of anti-epileptic drugs will be excluded.
- - The use of medical cannabis and CBD oil is prohibited during the first 2 cycles of this protocol.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Tobey MacDonald, MD|
|Principal Investigator Affiliation||Emory University|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Brain Tumor, Medulloblastoma, Brain Metastases|
The goal of this clinical research study is to find the highest tolerable dose of WP1066 that can be given to pediatric patients with recurrent (has returned after treatment) cancerous brain tumors or melanoma that has gotten worse and spread to the brain. The safety of this drug will also be studied. WP1066 is designed to target the STAT3 pathway in cancer cells, which makes these cells divide, increases new blood vessels to the tumor, causes the cancer cells to move throughout the body and brain, and avoids them being detected by the immune system. Targeting this pathway may cause the immune system to kill the cancer cells. The investigators will administer five escalating doses of WP1066, starting at lowest dose currently found to be safe and tolerable in adults. WP1066 is not FDA approved or commercially available. It is currently being used for research purposes only. Up to 36 participants will be enrolled in this study. All will take part at Children's Healthcare of Atlanta (CHOA).
There will be 5 groups based on the enrollment timing. The first group of participants will receive the lowest dose level of WP1066. Each subject within a group will receive an assigned dose of the investigational drug. The dose levels are 4, 6, 8 and 16 mg/kg of the investigational drug given twice a day. The first group will receive the lowest dose level, 4mg/kg twice a day, and subsequent groups will escalate to the next higher dose level. All groups will be treated identically, except for the dose of drug administered, with the liquid formulation of the drug.
Drug: - WP1066
WP1066 is an analogue of caffeic acid that is a potent inhibitor of p-STAT3. It will be taken by mouth 2 times per day on Monday, Wednesday, and Friday of Weeks 1 and 2 of each 28-day cycle.
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.