Cgd

Current Location

13 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

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

100 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. This places them at risk of developing infections that may be life-threatening. Stem cell transplant can cure CGD but transplanting stem cells donated by other people can have serious complications. In addition, not everyone has a matched donor. Another approach is a type of gene therapy that involves base-editing to correct the mutation in a person s own stem cells. 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 improve white blood cells' ability to fight against infections 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. Follow-up visits will continue for 15 years.
No Placebo Group

Trial Details

Trial Status:Recruiting
Sex:Male

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

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:Recruiting
Trial Phase:Early Phase 1
Age:4 - 65

38 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

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

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

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+

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

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

20 Participants Needed

Stem Cell Transplant Prep Regimen for Immune Deficiency Disorders

Saint 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

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

30 Participants Needed

Why Other Patients Applied

"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

"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 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 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

"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

Know someone looking for new options? Spread the word

Learn More About Power

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?

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 Therapy for Chronic Granulomatous Disease, Gene Therapy for Chronic Granulomatous Disease and Base Editing for Chronic Granulomatous Disease to the Power online platform.

Popular Searches

By Condition

Depression Clinical Trials

Anxiety Clinical Trials

Schizophrenia Clinical Trials

ADHD Clinical Trials

Bipolar Disorder Clinical Trials

Multiple Sclerosis Clinical Trials

Autism Clinical Trials

Treatment Resistant Depression Clinical Trials

Borderline Personality Disorder Clinical Trials

Social Anxiety Disorder Clinical Trials

Parkinson's Disease Clinical Trials

Alzheimer's Disease Clinical Trials

By Location

Clinical Trials in California

Clinical Trials in Florida

Clinical Trials in Texas

Clinical Trials in New York

Clinical Trials in Ohio

Clinical Trials in Illinois

Clinical Trials in Pennsylvania

Clinical Trials in Michigan

Clinical Trials in North Carolina

Clinical Trials in Massachusetts

Clinical Trials in Missouri

Clinical Trials in Minnesota

Other People Viewed

By Subject

Top Clinical Trials near Farmington, MO

Top Alopecia Areata Clinical Trials

Top Depression Clinical Trials near High Point, NC

20 Alzheimer's Disease Trials near Tulsa, OK

Top Dermatomyositis Clinical Trials

80 Clinical Trials near Hawaii

22 Colorectal Cancer Trials near Las Vegas, NV

Top Infectious Disease Clinical Trials

Top Retinitis Pigmentosa Clinical Trials

Top Clinical Trials near High Point, NC

Top Psoriasis Clinical Trials near Worcester, MA

Top Acl Clinical Trials

By Trial

Alpelisib for Insulin Resistance

Split-belt Treadmill Training for ACL Injury

Therapeutic Estradiol for Breast Cancer

Aspirin Dosing for Obesity

CES for Generalized Anxiety Disorder

Dapagliflozin for Cardio-Renal Syndrome

Empagliflozin for Dialysis Patients with Kidney Failure

Stereotactic Ablative Radiotherapy for Early Stage Breast Cancer

Reproductive Health Care Intervention for Young Female Cancer Survivors

Supported Digital CBT for Depression and Anxiety

Lutetium-177 for Prostate Cancer

Durvalumab +/- Tremelimumab for Bladder Cancer

Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of Service·Privacy Policy·Cookies·Security
Match to a Trial