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29 Npc1 Trials Near You
Power is an online platform that helps thousands of Npc1 patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Learn More About PowerTrappsol Cyclo for Niemann-Pick Disease
Cincinnati, Ohio
This trial is testing a medicine called Trappsol Cyclo, given through an IV, for patients with Niemann Pick disease type C1. The medicine helps remove harmful fat buildup in cells. The study will also look at safety in younger children. Trappsol Cyclo has shown promising results by mobilizing cholesterol, extending life, and reducing neurological damage in Niemann-Pick disease type C1.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:3+
Key Eligibility Criteria
Disqualifiers:Liver Disease, Kidney Disease, Epilepsy, Others
Must Not Be Taking:Curcumin, Fish Oil, Leucine, Others
94 Participants Needed
Cholesterol-Lowering Drug + Chemotherapy for Pancreatic Cancer
Montreal, Quebec
Cardiovascular diseases and cancers, the two leading causes of death in Canada, require cholesterol to sustain their progression. All cells require cholesterol, but cancer cells have much higher needs to sustain growth, division and metastasis. The availability of new cholesterol-lowering drugs developed to protect patients from heart diseases has resulted in unprecedented low levels of cholesterol. The combination of atorvastatin, ezetimibe and Repatha, which are 3 cholesterol-lowering drugs used in combination, is safe, well tolerated and efficient over years of treatment. Recent reports indicate that abundant cholesterol supplies are required to sustain the progression of pancreatic ductal adenocarcinomas. This proof-of-concept study aims to verify the feasibility, the acceptability and gain preliminary data on adding a cholesterol shortage on top of FOLFIRINOX (standard chemotherapy) in newly diagnosed patients with locally advanced pancreatic adenocarcinomas or metastatic pancreatic adenocarcinomas. It is expected that a drug-induced cholesterol shortage will slow-down or stop the progression of pancreatic adenocarcinomas while increasing the response to chemotherapy.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Early Phase 1
Key Eligibility Criteria
Disqualifiers:Operable Cancer, Other Malignancy, Myalgia, Others
Must Be Taking:Cholesterol-lowering Drugs
3 Participants Needed
This trial tests different combinations of chemotherapy drugs and radiation therapy for advanced nasopharyngeal cancer. It targets patients whose cancer has not spread beyond the local region. The treatment works by killing cancer cells or stopping their growth using powerful drugs and radiation. Previous studies have shown a survival benefit using a combination of these treatments for nasopharyngeal carcinoma.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Key Eligibility Criteria
Disqualifiers:Prior Invasive Malignancy, Hearing Loss, AIDS, Others
685 Participants Needed
Ziftomenib Combinations for Acute Myeloid Leukemia
Columbus, Ohio
This Phase 1 study will assess the safety, tolerability, and preliminary antileukemic activity of ziftomenib in combination with venetoclax and azacitidine (ven/aza), ven, and 7+3 for two different molecularly-defined arms, NPM1-m and KMT2A-r.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Leukostasis, CNS Involvement, Active Infection, Others
Must Not Be Taking:Live Vaccines
212 Participants Needed
Birinapant + IMRRT for Head and Neck Cancer
Columbus, Ohio
This phase I trial studies the side effects and best dose of birinapant when given together with intensity modulated re-irradiation therapy (IMRRT) in treating patients with head and neck squamous cell carcinoma that has come back at or near the same place as the original (primary) tumor (locally recurrent). Birinapant may stop the growth of tumor cells by blocking inhibitor of apoptosis (IAP), a protein needed for tumor cell survival. IMRRT uses thin beams of radiation of different intensities that are aimed at the tumor from many angles. This type of re-irradiation therapy reduces the damage to healthy tissue near the tumor. Giving birinapant with IMRRT may lower the chance of head and neck squamous cell carcinoma growing or spreading.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Not Listed
13 Participants Needed
Nivolumab + Ipilimumab for Rare Cancers
Columbus, Ohio
This phase II trial studies nivolumab and ipilimumab in treating patients with rare tumors. Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
This trial enrolls participants for the following cohorts based on condition:
1. Epithelial tumors of nasal cavity, sinuses, nasopharynx: A) Squamous cell carcinoma with variants of nasal cavity, sinuses, and nasopharynx and trachea (excluding laryngeal, nasopharyngeal cancer \[NPC\], and squamous cell carcinoma of the head and neck \[SCCHN\]) B) Adenocarcinoma and variants of nasal cavity, sinuses, and nasopharynx (closed to accrual 07/27/2018)
2. Epithelial tumors of major salivary glands (closed to accrual 03/20/2018)
3. Salivary gland type tumors of head and neck, lip, esophagus, stomach, trachea and lung, breast and other location (closed to accrual)
4. Undifferentiated carcinoma of gastrointestinal (GI) tract
5. Adenocarcinoma with variants of small intestine (closed to accrual 05/10/2018)
6. Squamous cell carcinoma with variants of GI tract (stomach small intestine, colon, rectum, pancreas) (closed to accrual 10/17/2018)
7. Fibromixoma and low grade mucinous adenocarcinoma (pseudomixoma peritonei) of the appendix and ovary (closed to accrual 03/20/2018)
8. Rare pancreatic tumors including acinar cell carcinoma, mucinous cystadenocarcinoma or serous cystadenocarcinoma. Pancreatic adenocarcinoma is not eligible (closed to accrual)
9. Intrahepatic cholangiocarcinoma (closed to accrual 03/20/2018)
10. Extrahepatic cholangiocarcinoma and bile duct tumors (closed to accrual 03/20/2018)
11. Sarcomatoid carcinoma of lung
12. Bronchoalveolar carcinoma lung. This condition is now also referred to as adenocarcinoma in situ, minimally invasive adenocarcinoma, lepidic predominant adenocarcinoma, or invasive mucinous adenocarcinoma
13. Non-epithelial tumors of the ovary: A) Germ cell tumor of ovary B) Mullerian mixed tumor and adenosarcoma (closed to accrual 03/30/2018)
14. Trophoblastic tumor: A) Choriocarcinoma (closed to accrual)
15. Transitional cell carcinoma other than that of the renal, pelvis, ureter, or bladder (closed to accrual)
16. Cell tumor of the testes and extragonadal germ tumors: A) Seminoma and testicular sex cord cancer B) Non seminomatous tumor C) Teratoma with malignant transformation (closed to accrual)
17. Epithelial tumors of penis - squamous adenocarcinoma cell carcinoma with variants of penis (closed to accrual)
18. Squamous cell carcinoma variants of the genitourinary (GU) system
19. Spindle cell carcinoma of kidney, pelvis, ureter
20. Adenocarcinoma with variants of GU system (excluding prostate cancer) (closed to accrual 07/27/2018)
21. Odontogenic malignant tumors
22. Pancreatic neuroendocrine tumor (PNET) (formerly named: Endocrine carcinoma of pancreas and digestive tract.) (closed to accrual)
23. Neuroendocrine carcinoma including carcinoid of the lung (closed to accrual 12/19/2017)
24. Pheochromocytoma, malignant (closed to accrual)
25. Paraganglioma (closed to accrual 11/29/2018)
26. Carcinomas of pituitary gland, thyroid gland parathyroid gland and adrenal cortex (closed to accrual)
27. Desmoid tumors
28. Peripheral nerve sheath tumors and NF1-related tumors (closed to accrual 09/19/2018)
29. Malignant giant cell tumors
30. Chordoma (closed to accrual 11/29/2018)
31. Adrenal cortical tumors (closed to accrual 06/27/2018)
32. Tumor of unknown primary (Cancer of Unknown Primary; CuP) (closed to accrual 12/22/2017)
33. Not Otherwise Categorized (NOC) Rare Tumors \[To obtain permission to enroll in the NOC cohort, contact: S1609SC@swog.org\] (closed to accrual 03/15/2019)
34. Adenoid cystic carcinoma (closed to accrual 02/06/2018)
35. Vulvar cancer (closed to accrual)
36. MetaPLASTIC carcinoma (of the breast) (closed to accrual)
37. Gastrointestinal stromal tumor (GIST) (closed to accrual 09/26/2018)
38. Perivascular epithelioid cell tumor (PEComa)
39. Apocrine tumors/extramammary Paget's disease (closed to accrual)
40. Peritoneal mesothelioma
41. Basal cell carcinoma (temporarily closed to accrual 04/29/2020)
42. Clear cell cervical cancer
43. Esthenioneuroblastoma (closed to accrual)
44. Endometrial carcinosarcoma (malignant mixed Mullerian tumors) (closed to accrual)
45. Clear cell endometrial cancer
46. Clear cell ovarian cancer (closed to accrual)
47. Gestational trophoblastic disease (GTD)
48. Gallbladder cancer
49. Small cell carcinoma of the ovary, hypercalcemic type
50. PD-L1 amplified tumors
51. Angiosarcoma
52. High-grade neuroendocrine carcinoma (pancreatic neuroendocrine tumor \[PNET\] should be enrolled in Cohort 22; prostatic neuroendocrine carcinomas should be enrolled into Cohort 53). Small cell lung cancer is not eligible (closed to accrual)
53. Treatment-emergent small-cell neuroendocrine prostate cancer (t-SCNC)
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Autoimmune Disease, Hepatitis, Heart Failure, Others
Must Not Be Taking:Steroids, Immunosuppressive Drugs
818 Participants Needed
SNDX-5613 + Chemotherapy for Acute Myeloid Leukemia
Columbus, Ohio
This phase Ib trial tests the safety, side effects, and best dose of SNDX-5613 when given in combination with the standard chemotherapy treatment (daunorubicin and cytarabine) in treating patients with newly diagnosed acute myeloid leukemia that has changes in the NPM1 gene or MLL/KMT2A gene. SNDX-5613 blocks signals passed from one molecule to another inside cancer cells that are needed for cancer cell survival. Drugs used in chemotherapy, such as daunorubicin and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding SNDX-5613 to the standard chemotherapy treatment may be able to shrink or stabilize the cancer for longer than the standard chemotherapy treatment alone.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Pregnancy, CNS Involvement, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers
28 Participants Needed
SNDX-5613 + Gilteritinib for Acute Myeloid Leukemia
Columbus, Ohio
This phase I trial tests the safety, side effects, and best dose of SNDX-5613 and gilteritinib for treating patients with acute myeloid leukemia that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory) and has a mutation in the FLT3 gene along with either a mutation in the NMP1 gene or a type of mutation called a rearrangement in the MLL gene. SNDX-5613 is in a class of medications called menin inhibitors. It works by blocking the action of mutated MLL and NMP1 proteins that signal cancer cells to multiply. Gilteritinib is in a class of medications called tyrosine kinase inhibitors. It works by blocking the action of mutated FLT3 proteins that signal cancer cells to multiply. Giving SNDX-5613 with gilteritinib may be safe, tolerable and/or effective in treating patients with relapsed/refractory FLT3 mutated acute myeloid leukemia.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Acute Promyelocytic Leukemia, CNS Involvement, Others
Must Be Taking:Strong CYP3A4 Inhibitors
30 Participants Needed
DSP-5336 for Leukemia
Columbus, Ohio
This trial is testing a new drug called DSP 5336 to help treat adults with certain types of blood cancer that have come back or didn't respond to previous treatments. The study will first find the best amount to give and then check how safe and helpful it is.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Acute Promyelocytic Leukemia, CNS Leukemia, Others
Must Not Be Taking:Calcineurin Inhibitors, CYP3A4/5 Inhibitors
362 Participants Needed
SNDX-5613 for Acute Leukemia
Columbus, Ohio
Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia.
In Phase 2, participants will be enrolled in 3 indication-specific expansion cohorts to determine the efficacy, short- and long-term safety, and tolerability of revumenib.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:30+
Key Eligibility Criteria
Disqualifiers:Acute Promyelocytic Leukemia, HIV, Hepatitis, Others
Must Be Taking:CYP3A4 Inhibitors
413 Participants Needed
Nivolumab + Cabozantinib for Melanoma
Dayton, Ohio
This trial tests if nivolumab and cabozantinib can help patients with mucosal melanoma. Nivolumab boosts the immune system, while cabozantinib stops cancer cells from growing. The goal is to prevent the cancer from coming back or spreading. Nivolumab and cabozantinib have shown efficacy in treating various cancers, including melanoma and renal cell carcinoma.
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Cardiovascular Disease, Autoimmune Disease, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers
99 Participants Needed
This phase II/III compares the standard therapy (chemotherapy plus cetuximab) versus adding bevacizumab to standard chemotherapy, versus combination of just bevacizumab and atezolizumab in treating patients with head and neck cancer that has spread to other places in the body (metastatic or advanced stage) or has come back after prior treatment (recurrent). Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Bevacizumab is in a class of medications called antiangiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to tumor. This may slow the growth and spread of tumor. Cetuximab is in a class of medications called monoclonal antibodies. It binds to a protein called EGFR, which is found on some types of cancer cells. This may help keep cancer cells from growing. Cisplatin and carboplatin are in a class of chemotherapy medications known as platinum-containing compounds. They work by killing, stopping, or slowing the growth of cancer cells. Docetaxel is in a class of chemotherapy medications called taxanes. It stops cancer cells from growing and dividing and may kill them. The addition of bevacizumab to standard chemotherapy or combination therapy with bevacizumab and atezolizumab may be better than standard chemotherapy plus cetuximab in treating patients with recurrent/metastatic head and neck cancers.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Key Eligibility Criteria
Disqualifiers:Uncontrolled Hypertension, Coagulopathy, Autoimmune, Others
Must Not Be Taking:Antiangiogenics, Aspirin, NSAIDs, Others
430 Participants Needed
Relatlimab + Immunotherapy for Nasopharyngeal Cancer
West Chester, Ohio
This phase II trial tests the addition of BMS-986016 (relatlimab) to the usual immunotherapy after initial treatment for nasopharyngeal cancer that has come back after a period of improvement (recurrent) or that has spread from where it first started (primary site) to other places in the body (metastatic). Relatlimab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. The usual approach of treatment is initial treatment with chemotherapy such as the combination of cisplatin (or carboplatin) and gemcitabine, along with immunotherapy such as nivolumab. After the initial treatment is finished, patients may continue to receive additional immunotherapy. Carboplatin is in a class of medications known as platinum-containing compounds. It works in a way similar to the anticancer drug cisplatin, but may be better tolerated than cisplatin. Carboplatin works by killing, stopping or slowing the growth of tumor cells. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Gemcitabine is a chemotherapy drug that blocks the cells from making deoxyribonucleic acid (DNA) and may kill cancer cells. Giving BMS-986016 in addition to the usual immunotherapy after initial treatment may extend the time without the tumor cells growing or spreading longer than the usual approach in patients with recurrent or metastatic nasopharyngeal cancer.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Autoimmune Diseases, Heart Failure, Others
Must Not Be Taking:Steroids, Immunosuppressants
156 Participants Needed
Immunotherapy +/− Targeted Therapy for Nasopharyngeal Cancer
Parkersburg, West Virginia
This phase II trial tests how well nivolumab and ipilimumab immunotherapy with or without cabozantinib works in treating patients with nasopharyngeal cancer that has come back (after a period of improvement) (recurrent), has spread from where it first started (primary site) to other places in the body (metastatic), or for which no treatment is currently available (incurable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Cabozantinib is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply. This helps slow or stop the spread of cancer cells. Giving immunotherapy with nivolumab and ipilimumab and targeted therapy with cabozantinib may help shrink and stabilize nasopharyngeal cancer.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Autoimmune Diseases, Active Tumor Bleeding, Others
Must Not Be Taking:Anticoagulants, Immunosuppressants
50 Participants Needed
BMX-001 for Head and Neck Cancer
West Chester, Ohio
This phase II trial compares the effectiveness of adding BMX-001 to usual symptom management versus usual symptom management alone for reducing oral mucositis in patients who are receiving chemoradiation for head and neck cancer. Oral mucositis (inflammation and mouth sores) is a common side effect of chemoradiation that can cause pain and difficulty swallowing. Usual management of these side effects typically consists of using mouth rinses and pain medications during treatment and for several weeks after completion of treatment. BMX-001 neutralizes harmful substances in the body, preventing damage to macromolecules such as DNA and minimizes free radical-related toxicity in normal tissues. Adding BMX-001 to usual symptom management may be more effective than usual symptom management alone at reducing oral mucositis in patients receiving chemoradiation for head and neck cancer.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Metastatic Disease, Cardiovascular Disease, Others
Must Be Taking:Cisplatin
98 Participants Needed
Toripalimab + Chemotherapy for Nasopharyngeal Cancer
Cincinnati, Ohio
This study aims to investigate toripalimab with chemotherapy in participants with nasopharyngeal cancer.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:12+
Key Eligibility Criteria
Disqualifiers:Local Therapy, CNS Metastases, Others
Must Not Be Taking:Steroids
100 Participants Needed
Nivolumab + Chemotherapy for Nasopharyngeal Cancer
Cincinnati, Ohio
This phase II trial tests effects of nivolumab in combination with chemotherapy drugs prior to radiation therapy patients with nasopharyngeal carcinoma (NPC). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as gemcitabine and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Researchers want to find out what effects, good and/or bad, adding nivolumab to chemotherapy has on patients with newly diagnosed NPC. In addition, they want to find out if children with NPC may be treated with less radiation therapy and whether this decreases the side effects of therapy.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 21
Key Eligibility Criteria
Disqualifiers:Immunodeficiency, Autoimmune Disease, Others
Must Not Be Taking:Immunosuppressants, Corticosteroids
50 Participants Needed
Ziftomenib + Chemotherapy for Leukemia
Cincinnati, Ohio
The primary objective of the study is to determine the recommended phase 2 dose (RP2D) of ziftomenib in combination with chemotherapy (FLA) in children with relapsed or refractory KMT2A-r, NUP98-r, or NPM1-m acute leukemia based on safety and pharmacokinetics (PK).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:0 - 21
Key Eligibility Criteria
Disqualifiers:Down Syndrome, EMD, APL, JMML, Others
Must Not Be Taking:Proton Pump Inhibitors
20 Participants Needed
PRO1160 for Advanced Cancer
Cleveland, Ohio
This trial tests PRO1160, a targeted cancer treatment, in patients with advanced or hard-to-remove cancers. PRO1160 uses an antibody to find cancer cells and a drug to kill them.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Other Malignancy, Active CNS Metastases, Others
Must Not Be Taking:Strong CYP3A Inhibitors
134 Participants Needed
89Zr-girentuximab PET Imaging for Cancer
Cleveland, Ohio
A prospective, open-label, phase 2 study to explore CAIX expression through 89Zirconium-labelled girentuximab deferoxamine (89Zr-girentuximab) PET/CT imaging in patients with solid tumors.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, Breastfeeding, Renal Insufficiency, Others
100 Participants Needed
Why Other Patients Applied
"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."
AG
Paralysis PatientAge: 50
"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."
WR
Obesity PatientAge: 58
"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."
FF
ADHD PatientAge: 31
"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."
HZ
Arthritis PatientAge: 78
"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."
ID
Pancreatic Cancer PatientAge: 40
SNDX-5613 + Chemotherapy for Acute Myeloid Leukemia
Cleveland, Ohio
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and clinical activity of SNDX-5613 in combination with intensive chemotherapy in participants with newly diagnosed acute myeloid leukemia (AML) harboring alterations in KMT2A, NPM1, or NUP98 genes.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Acute Promyelocytic Leukemia, CNS Leukemia, Others
Must Not Be Taking:Strong CYP3A4 Inducers
76 Participants Needed
BMF-500 for Leukemia
Cleveland, Ohio
This trial is testing BMF-500, a pill that blocks a protein called FLT3, in adults with aggressive types of leukemia. The goal is to stop cancer cells from growing by blocking this protein.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Cardiovascular Disease, High WBC, Pregnancy, Others
Must Be Taking:Azole Antifungals
84 Participants Needed
Penpulimab for Nasopharyngeal Cancer
Ann Arbor, Michigan
This trial is testing a new treatment combining penpulimab and chemotherapy for patients whose nasopharyngeal cancer has returned or spread. Penpulimab helps the immune system better recognize and fight cancer cells.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12 - 75
Key Eligibility Criteria
Disqualifiers:Other Malignancies, Autoimmune Disease, Others
Must Not Be Taking:Immunotherapy, Corticosteroids, Immunosuppressants, Others
296 Participants Needed
Brock String Therapy for Concussion
Pittsburgh, Pennsylvania
The purpose of the proposed study is to evaluate the efficacy of Brock String therapy prescribed in the acute stage of concussion recovery can improve clinical outcomes among patients with receded near point of convergence (NPC).
Aim 1: Determine if participants receiving the Brock String have more significant improvements in NPC measurements at follow up 7-10 days post injury.
Aim 2: Determine if participants receiving the Brock String 1) improve on computerized neurocognitive test scores from initial visit (\<48 hours post injury) to follow up visit (7-10 days post injury) compared to control participants, and 2) have reduced recovery time (i.e., days from injury until return to play) compared to control participants.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:12 - 20
Key Eligibility Criteria
Disqualifiers:Vestibular Disorder, Seizure Disorder, 3+ Concussions, Others
50 Participants Needed
Ziftomenib Combinations for Acute Myeloid Leukemia
Detroit, Michigan
The safety, tolerability, and antileukemic response of ziftomenib in combination with standard of care treatments for patients with relapsed/refractory acute myeloid leukemia will be examined with the following agents: FLAG-IDA, low-dose cytarabine, and gilteritinib.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Acute Promyelocytic Leukemia, CNS Leukemia, Uncontrolled Infection, Others
Must Not Be Taking:Immunosuppressive Drugs
171 Participants Needed
INCB099280 for Cancer
Pittsburgh, Pennsylvania
This trial is testing a new drug called INCB099280 to see if it is safe and effective for patients with certain types of solid tumors. Researchers want to understand how the drug behaves in the body and its early effects on cancer.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
182 Participants Needed
Pembrolizumab + Cetuximab for Head and Neck Cancer
Ann Arbor, Michigan
This trial tests a combination of two drugs, pembrolizumab and cetuximab, for patients with head and neck cancer that has returned or spread. Pembrolizumab helps the immune system fight cancer, and cetuximab attacks the cancer cells directly. Both drugs are used in treating head and neck squamous cell carcinoma.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Active Infection, Autoimmune Disease, Others
Must Not Be Taking:Immunosuppressants, Steroids, Others
78 Participants Needed
KFA115 + Tislelizumab for Advanced Cancers
Pittsburgh, Pennsylvania
This trial tests a new drug, KFA115, alone and with pembrolizumab in patients with advanced cancers. It aims to find the safest dose and see if the drugs can reduce tumors. The focus is on patients whose cancers are hard to treat with current options.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Cardiac Disease, Autoimmune Disease, ILD, Others
Must Not Be Taking:QT-prolonging Agents
180 Participants Needed
Ziftomenib for Relapsed/Refractory Acute Myeloid Leukemia
Ann Arbor, Michigan
This trial is testing ziftomenib, a new drug, in patients with difficult-to-treat acute myeloid leukemia (AML). The drug works by blocking a protein interaction that cancer cells need to grow.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Acute Promyelocytic Leukemia, CNS Leukemia, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers
263 Participants Needed
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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.
Bask GillCEO at Power
Frequently Asked Questions
How much do Npc1 clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Npc1 clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Npc1 trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Npc1 is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Npc1 medical study?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Npc1 clinical trials?
Most recently, we added BMX-001 for Head and Neck Cancer, Ziftomenib + Chemotherapy for Leukemia and Toripalimab + Chemotherapy for Nasopharyngeal Cancer to the Power online platform.
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