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Why We Started Power

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
Bask GillCEO at Power
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      Clear All
      Why We Started Power

      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
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      15 Cgd Trials Near You

      Power is an online platform that helps thousands of Cgd 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 Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      Stem Cell Transplant for Immunodeficiency

      Pittsburgh, Pennsylvania
      This study is evaluating whether a lung transplant followed by a stem cell transplant is safe and effective for people with primary immunodeficiency.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 1, 2
      Age:5 - 45

      Key Eligibility Criteria

      Disqualifiers:Malignant Conditions, HIV, Uncontrolled Infections, Others
      Must Not Be Taking:Live Vaccines

      16 Participants Needed

      Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders

      Pittsburgh, Pennsylvania
      The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:2 - 55

      Key Eligibility Criteria

      Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others

      100 Participants Needed

      Gene Transfer for CGD

      Bethesda, Maryland
      Background: X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a gene mutation that makes the immune system to not work properly. Researchers want to see if a lentiviral gene transfer treatment will have the ability to make the patient s immune system more normal, in particular reduce the risk of CGD related infections. The gene transfer takes a person s own stem cells, cultures them to put the normal gene in, then gives the cells back to the person. Objective: To test a gene transfer treatment for X-CGD. Eligibility: Participants aged 3-60 with X-CGD Design: Participants will be screened under protocol 05-I-0123. They will undergo: Medical history Physical exam Heart tests Imaging tests, as needed Blood tests Lung function tests, as needed Dental and audiology exams, if needed Quality of life questionnaire Bone marrow aspiration. A needle will be inserted into the hip bone or breastbone to collect bone marrow. Some screening tests will be repeated during the study. Participants will have an apheresis procedure under protocol 94-I-0073. Stem cells will be collected. Participants will get a series of drugs to prepare them for the gene transfer. Participants will stay at the NIH Clinical Center for a little over a month. They will get a central line. It is a large intravenous (IV) catheter that is placed into a vein of the neck, chest, or arm. They will get chemotherapy and their corrected stem cells through their IV line. Participants will have 12 follow-up outpatient visits in the 2 years after their gene transfer, as well as visits with their local doctor. Then they will enroll in another study for long-term follow-up visits that will last for 13 years.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 1, 2
      Age:3 - 60

      Key Eligibility Criteria

      Disqualifiers:HIV, Hepatitis B, Hepatitis C, Others
      Must Not Be Taking:Gamma-interferon

      10 Participants Needed

      Base Editing for Chronic Granulomatous Disease

      Bethesda, Maryland
      Background: Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly and are at greater risk of getting infections. Gene therapy using lentivector has helped people with CGD. Researchers want to know if the base-edited stem cells can improve the white cells' functioning and result in fewer CGD-related infections. Objective: To learn if base-edited stem cells will correct the white blood cells in people with CGD. Eligibility: Males aged 18 years and older with X-linked CGD. Design: This is a non-randomized study. Participants with the specific mutation under study will be screened during the initial phase. During the development phase, participants will undergo apheresis to collect stem cells for base-editing correction of the mutation. During the treatment phase, participants will receive the base-edited cells after chemotherapy with busulfan. Participants will remain in the hospital until their immunity recovers. Participants will be maintained on sirolimus to prevent an immune response to the new protein expressed by the base-edited cells. Follow-up visits will continue for 15 years.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Hematologic Malignancy, Chronic Infections, Cardiac Abnormalities, Others

      10 Participants Needed

      Stem Cell Transplant + Drug Therapy for Chronic Granulomatous Disease

      Bethesda, Maryland
      Background: Chronic granulomatous disease (CGD) affects the immune system. People with CGD are more likely to get infections. Drugs can help control infections, but these treatments can cause side effects including kidney failure and deafness. Stem cell transplants can cure CGD, but these don t always work. Objective: To find out if a different drug treatment can improve the success rates of stem cell transplants in people with CGD. Eligibility: People aged 4-65 years with CGD. Design: Participants will undergo screening. They will have a physical exam. They will have blood and urine tests and tests of their heart function and breathing. They will have imaging scans. They will have a bone marrow biopsy; a needle will be inserted into their hip to draw a sample of tissue from the bone. A tube called a catheter will be placed into a vein in the participant s chest. This catheter will remain in place for the transplant and recovery period. Blood for tests can be drawn from the catheter, and medications and the stem cells can be administered through it. Participants will be in the hospital for either 10 or 21 days to receive 3 or 4 drugs before the transplant. They will get 2 doses of total body radiation on the same day. Participants will receive donor stem cells through the catheter. They will remain in the hospital for 6 weeks afterward. Participants will visit the clinic 2 to 3 times per week for 3 months after discharge. Follow-up visits will continue for 5 years.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:4 - 65

      Key Eligibility Criteria

      Disqualifiers:Heart Failure, Lung Disease, Psychiatric Disorder, Others

      50 Participants Needed

      JSP191 for Chronic Granulomatous Disease

      Bethesda, Maryland
      Background: Chronic granulomatous disease (CGD) is a rare immune disorder that can cause serious infections throughout the body. The only cure for CGD is a stem cell transplant. Transplants from a sibling are best, but many people must get transplants from unrelated donors. However, these transplants can cause serious complications in people with CGD. Objective: To see if a study drug (JSP191) can help improve the success rates of stem cell transplants for people with CGD from an unrelated donor. Eligibility: People aged 4 to 65 years with CGD who require a transplant. Design: Participants will be screened. Part of the screening will help to identify the best match to a transplant donor. Participants will have a physical exam, including dental and eye exams. They will have blood and urine tests. They will have tests of their breathing and heart function. A bone marrow sample will be taken. They will have their stem cells collected. Participants will have a catheter inserted into a vein in their chest. It will remain in place for the entire period of transplant and recovery. Participants will be in the hospital 40 to 50 days for the transplant. This will include a conditioning phase, to prepare their body for the procedure, as well as the transplant and recovery phases. As part of the conditioning phase, participants will receive JSP191 through a vein for 1 hour. After discharge, participants will have follow-up visits 2 times a week for 100 days. Additional follow-up visits will continue for 5 years....
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Early Phase 1
      Age:4 - 65

      Key Eligibility Criteria

      Disqualifiers:Pregnant, HIV Positive, Uncontrolled Seizures, Others

      7 Participants Needed

      Haploidentical Transplant for Chronic Granulomatous Disease

      Bethesda, Maryland
      Background: CGD causes infections and inflammation. The only cure currently is a bone marrow transplant. Most often a perfectly matched bone marrow donor is used. Researchers want to see if they can lower the risks of using a mismatched donor. Objectives: To see if it is safe to use a related bone marrow donor who is only a partial match to a person with CGD. To see how well drugs given to a person before and after transplant help the body accept the transplant. Eligibility: People ages 4-65 with CGD for whom stem cell transplant may be a cure and who do not have a perfectly matched donor, related or unrelated. Design: Participants will be screened with: Medical history Physical exam Blood tests Participants will be admitted to the hospital about 2 weeks before the transplant. They will have blood, urine, breathing, and heart tests. They may have CT and/or MRI scans. They will have a needle inserted into their hipbone to remove marrow. They will have dental, neurologic, and psychologic tests. They will have a central catheter placed: A line will be placed into a vein in their upper chest. They will get drugs, chemotherapy, and radiation to prepare for the transplant. Participants will receive the donated cells through their catheter. The cells will be from one of their relatives. Participants will stay in the hospital about 6 weeks after the transplant. After they leave the hospital, participants will have to stay in the area with visits about 2 times a week for approximately 100 days post transplant. Then visits will be every 3 to 6 months for 2 years. Then visits will be once a year.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Early Phase 1
      Age:4 - 65

      Key Eligibility Criteria

      Disqualifiers:Severe Colitis, Heart Failure, Psychiatric Disorders, Others
      Must Not Be Taking:Anti-TNF, Anti-interleukin, Anti-integrin

      4 Participants Needed

      gp91 Grans for Chronic Granulomatous Disease

      Bethesda, Maryland
      Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections. Researchers want to see if a new procedure can help a person s cells kill germs for a short time. It uses messenger RNA (mRNA) to deliver correct instructions for the gene mutation to the cells. Objective: To test a procedure in which mRNA is added to a person s blood cells. Eligibility: Males aged 18-75 with CGD with a mutation in the gene that makes the protein gp91phox. Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Swab to test for strep throat Some screening tests will be repeated during the study. Participants will be admitted to the NIH Clinical Center hospital for at least 7 days. They will have apheresis. For this, a medicine is injected under their skin to prepare their white blood cells for collection. An IV line is placed into an arm vein. Blood goes through the IV line into a machine that divides whole blood into red blood cells, plasma, and white blood cells. The white blood cells are removed, and the rest of the blood is returned to the participant through an IV line in their other arm. The next day, they will get their mRNA-corrected cells via IV. They will be monitored for 3 more days. After discharge, participants will keep a symptom diary. They will be contacted weekly for one month, and then once a month. They will have a follow-up visit 3 months after the infusion.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Systemic Infections, Kidney Disease, Diabetes, Heart Failure, Others
      Must Not Be Taking:High-dose Steroids

      25 Participants Needed

      Elemental Diet for Inflammatory Bowel Disease

      Bethesda, Maryland
      Background: Bacteria that live inside the stomach and intestines are important for health. Chronic granulomatous disease (CGD) and inflammatory bowel disease (IBD) can make people have unhealthy bacteria. This can lead to gastrointestinal (GI) problems. Researchers want to see if people with CGD and IBD feel better when they change the bacteria in the stomach by following a special liquid diet. Objective: To see if an elemental diet can change the bacteria in the stomach and intestines of people with CGD and IBD. Also, to see if this helps GI symptoms. Eligibility: People ages 8-65 years with CGD, CGD-associated colitis, and IBD. Design: Participants will first be screened with: Upper GI endoscopy and/or colonoscopy. A long, thin tube with a tiny camera at the end will be passed into the participant s body through the mouth or anus. Tissue will be collected. Participants will be sedated for the procedure. They will be sedated using a special mask or small plastic tube placed in an arm vein using a needle. Participants will be put on the special diet for up to 4 weeks. They will stay in the hospital for up to 2 weeks. They will have check-ups. They will have blood, urine, and stool samples collected. They will keep a symptom diary to record how they feel and any GI symptoms. Participants will have 2 follow-up visits. The first will be right after they finish the diet. The second will be 4 weeks later. They will have blood, urine, and stool samples collected. They will learn about re-introducing other foods into their diet.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 2
      Age:8 - 65

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Active Infection, Others
      Must Not Be Taking:Antibiotics

      10 Participants Needed

      Gene Therapy for Chronic Granulomatous Disease

      Bethesda, Maryland
      This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with autosomal recessive Chronic Granulomatous Disease (CGD) caused by mutations in the NCF1 (Neutrophil Cytosolic Factor 1) gene.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:6+

      Key Eligibility Criteria

      Disqualifiers:HIV, Hepatitis B, Hepatitis C, Others

      12 Participants Needed

      Gene Therapy for Chronic Granulomatous Disease

      Bethesda, Maryland
      Background: Chronic granulomatous disease (CGD) is a genetic disorder. People with CGD are missing a gene that affects their white blood cells. White cells are part of the immune system, and people with GCD are vulnerable to many infections. Researchers want to test a new treatment to replace the missing gene that may be safer than the current treatment for CGD. Objective: To test a new type of gene therapy in people with CGD. Eligibility: People aged 3 years or older with CGD. Design: Participants will undergo apheresis: Blood will be collected through a tube attached to a needle inserted in a vein; the blood will run through a machine that separates certain cells (stem cells); the remaining blood will be returned to the body through a second needle. The participant s stem cells will be modified in a laboratory to add the gene they are missing. Participants will stay in the hospital for about 40 days. For the first 10 days, they will undergo many exams, including imaging scans and tests of their heart and lung function. They will receive drugs to prepare their bodies for the gene therapy. They will receive a "central line": A hollow tube will be inserted into a vein in the chest, with a port opening above the skin. This port will be used to draw blood and administer drugs without the need for new needle sticks. For the gene therapy, each participant s own modified stem cells will be put into their body through the port. Participants will have 8 follow-up visits over 3 years.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 1, 2
      Age:3 - 65

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, HIV, Hepatitis, Anemia, Others
      Must Not Be Taking:Gamma-interferon

      10 Participants Needed

      Tofacitinib for Chronic Granulomatous Disease

      Bethesda, Maryland
      This trial is testing tofacitinib, a pill that reduces inflammation, in adults with chronic granulomatous disease (CGD) who haven't responded to other treatments. The drug aims to calm the overactive immune system in these patients. Tofacitinib has been approved for the treatment of rheumatoid arthritis, psoriatic arthritis, and ulcerative colitis.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Tuberculosis, Hepatitis B/C, HIV, Others
      Must Not Be Taking:NSAIDs, Strong CYP3A4 Inducers

      20 Participants Needed

      EN-374 Gene Therapy for Chronic Granulomatous Disease

      Durham, North Carolina
      The goal of this clinical trial is to evaluate the safety and potential efficacy of the EN-374 treatment regimen and identify a dose level for further evaluation in participants with x-linked chronic granulomatous disease. The main questions it aims to answer are: * safety of the EN-374 treatment regimen * effect of the EN-374 treatment regimen on the production of functional neutrophils with NADPH oxidase activity
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:3+
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:HIV, Hepatitis B, Hepatitis C, Others

      15 Participants Needed

      Stem Cell Transplant Prep Regimen for Immune Deficiency Disorders

      St Louis, Missouri
      This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 21

      Key Eligibility Criteria

      Disqualifiers:HIV, Pregnancy, Infections, Others

      20 Participants Needed

      Stem Cell Transplant for Primary Immunodeficiency

      Minneapolis, Minnesota
      This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:< 50

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, HIV, Hepatitis, Others

      30 Participants Needed

      Why Other Patients Applied

      "As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."

      IZ
      Healthy Volunteer PatientAge: 38

      "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

      "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
      Match to a Cgd Trial

      Know someone looking for new options?
      Spread the word

      Why We Started Power

      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
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?
      Match to a Trial
      Match to a Trial

      Frequently Asked Questions

      How much do Cgd 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 Cgd 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 Cgd 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 Cgd 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 Cgd 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 Cgd clinical trials?

      Most recently, we added Gene Transfer for CGD, EN-374 Gene Therapy for Chronic Granulomatous Disease and Gene Therapy for Chronic Granulomatous Disease to the Power online platform.