Down Syndrome

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183 Down Syndrome Trials Near You

Power is an online platform that helps thousands of Down Syndrome 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.

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No Placebo
Highly Paid
Stay on Current Meds
Pivotal Trials (Near Approval)
Breakthrough Medication
This trial tests imlifidase, a drug that breaks down harmful antibodies, in patients with severe anti-GBM disease. The goal is to see if it improves kidney function. Imlifidase is conditionally approved in the EU for use in certain kidney transplant patients.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3

50 Participants Needed

This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in the body's immune system and may interfere with the ability of cancer cells to grow and spread. Chemotherapy drugs, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, 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. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better than combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:365 - 31

6720 Participants Needed

XePOHCAS: Prospective, randomized, multicenter interventional trial in adult subjects with out-of-hospital cardiac arrest comparing treatment with standard-of-care post-cardiac arrest intensive care (which is targeted temperature management \[TTM\]) to xenon by inhalation plus standard-of-care post-cardiac arrest intensive care (including TTM).
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Not Yet Recruiting
Trial Phase:Phase 3

1436 Participants Needed

This trial studies a chemotherapy treatment that adjusts based on how well patients respond initially. It targets younger patients with Down syndrome who have certain types of blood cancer. The treatment aims to effectively kill cancer cells while reducing side effects.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:91 - 3

280 Participants Needed

This study will enroll participants with myelodysplastic syndromes (MDS) with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have not received treatment with a hypomethylating agent previously. Participants will be randomized to receive either ivosidenib (IVO) alone or azacitidine (AZA) alone. IVO will be administered daily throughout the 28-day treatment cycle and AZA will be administered for the first 7 days of each 28-day cycle. Study visits will be conducted every week during Cycle 1 (Days 1, 8, 15, and 22), and Day 1 of each cycle thereafter. After the last dose of treatment, participants will attend an safety follow-up visit and participants will be followed to assess overall survival. Study visits may include a bone marrow aspirate, physical exam, echocardiogram (ECHO), electrocardiogram (ECG), blood and urine analysis, and questionnaires.
No Placebo Group
Prior Safety Data
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3

48 Participants Needed

This trial is testing two treatments, Luspatercept and epoetin alfa, to see which is better for treating anemia in adults with certain types of myelodysplastic syndromes (MDS). The participants have not used similar treatments before and do not need regular blood transfusions. Luspatercept helps red blood cells mature, while epoetin alfa increases their production.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3

360 Participants Needed

This trial tests a new pill form of two drugs, cedazuridine and azacitidine, for patients needing azacitidine treatment. The goal is to see if the pill is as effective as the injection. Cedazuridine helps azacitidine work better by preventing its breakdown, and azacitidine stops cancer cells from growing.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2, 3

235 Participants Needed

The purpose of this study is to evaluate the efficacy and safety of imetelstat sodium in transfusion-dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment in Phase 2 study and to compare the efficacy, in terms of red blood cell (RBC) transfusion independence (TI), of imetelstat sodium to placebo in transfusion-dependent participants with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment in Phase 3 study. A separate Ventricular Repolarization Substudy (QTc Substudy) will evaluate the effect of imetelstat sodium on ventricular repolarization. An Extension Phase has been included to allow continued treatment for those participants who are benefitting from imetelstat sodium and to continue to evaluate the long-term safety, overall survival (OS), and disease progression, including progression to acute myeloid leukemia (AML) in transfusion-dependent participants with low or immediate-1 risk MDS that is relapsed/refractory to ESA treatment.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3

289 Participants Needed

The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 17

150 Participants Needed

This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS. The study consists of 3 main phases: Screening, Titration period, and Maintenance period, followed by a Taper period and Follow-Up. Participants will be randomized to LP352 or placebo. The total duration of the study will be approximately 24 months.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 65

160 Participants Needed

The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3

100 Participants Needed

This phase II trial studies the effect of venetoclax and azacitidine in treating patients with therapy related or secondary myelodysplastic syndrome. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Chemotherapy drugs, such as azacitidine, 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. Giving venetoclax in combination with azacitidine may work better in treating patients with therapy related or secondary myelodysplastic syndrome.
No Placebo Group
Prior Safety Data

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

53 Participants Needed

This trial is testing if adding pomalidomide to standard chemotherapy can improve treatment for patients with a specific type of newly diagnosed leukemia. Pomalidomide works by cutting off the blood supply to cancer, boosting the immune system, and killing cancer cells. The chemotherapy drugs attack cancer cells in multiple ways. Pomalidomide is related to thalidomide and has shown remarkable activity in patients who did not respond to other treatments.
No Placebo Group
Prior Safety Data

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2

50 Participants Needed

This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:< 22

960 Participants Needed

Colon polyposis (the presence of multiple colon polyps) is very common with Cowden syndrome, as over 60% of patients have 50 or more polyps. In a previous clinical trial, some participants had reduction in the number of colon polyps with the use of the medication sirolimus for a very short time period. This study is investigating sirolimus and its effect on the number of colon polyps in patients with Cowden syndrome and polyposis over a 1 year period.
No Placebo Group
Prior Safety Data

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2

10 Participants Needed

This research study is evaluating the combination of a study drug, venetoclax, and a standard chemotherapy regimen, R-EPOCH or R-CHOP, as a possible treatment for Richter's Syndrome. The drugs involved in this study are: * Venetoclax * R-EPOCH: * Rituximab * Etoposide * Prednisone * Vincristine Sulfate (Oncovin) * Cyclophosphamide * Doxorubicin Hydrochloride (Hydroxydaunomycin) * R-CHOP: * Rituximab * Cyclophosphamide Vincristine * Doxorubicin Hydrochloride (Hydroxydaunomycin) * Sulfate (Oncovin) * Prednisone
No Placebo Group
Prior Safety Data

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2

69 Participants Needed

This study is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. 78 patients will be enrolled, randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. Randomization will be stratified by prescribed GC dose on the day of randomization.

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

78 Participants Needed

This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study evaluating the efficacy and safety of AK117 or placebo, combined with azacitidine in patients with newly diagnosed higher-risk myelodysplastic syndromes (HR-MDS).

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2

90 Participants Needed

The purpose of this study is to evaluate the safety and efficacy of EPX-100 as adjunctive therapy in participants with Dravet syndrome.
Stay on current meds

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 80

100 Participants Needed

Study BH-30236-01 is a first-in-human (FIH), Phase 1/1b, open-label, dose escalation and expansion study in participants with relapsed/refractory acute myelogenous leukemia (R/R AML) or higher-risk myelodysplastic syndrome (HR-MDS). Phase 1 (Dose Escalation) will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of BH-30236 administered orally. Approximately 50 participants may be enrolled in Phase 1 of the study. Phase 1b (Dose Expansion) will follow Phase 1 to further understand the relationships among dose, exposure, toxicity, tolerability, and clinical activity. Up to 24 participants may be enrolled in Phase 1b of the study. The dose expansion part (Phase 1b) will be followed to understand the relationships among dose, exposure, toxicity, tolerability and clinical activity. Up to 24 participants may be enrolled in Phase 1b of the study.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1

74 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

"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

"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 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
This trial is testing a new drug called RAY121, which aims to help people with certain immune system diseases by blocking a part of the immune system that causes inflammation. The trial will check if the drug is safe and effective for patients with conditions like APS, BP, BS, DM, IMNM, and ITP.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 1

144 Participants Needed

CC-91633 for Leukemia

Columbus, Ohio
This trial tests a new drug, CC-91633, for patients with certain blood cancers that haven't responded to other treatments. Researchers will find the safest dose by increasing it over time and checking for side effects and effectiveness.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1

180 Participants Needed

This trial is testing a new drug called JNJ-74856665, alone or with other drugs, in patients with specific blood cancers like AML, MDS, and CMML. The goal is to see if it can safely stop cancer cells from growing and make them die.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 1

153 Participants Needed

This research study is being done to help researchers develop new dietary options for menopausal women to maintain a healthy weight by developing more nutritious snacks that have health benefits. From this study, the researchers hope to gain understanding on how menopausal women with metabolic syndrome digest and absorb foods with safflower oil on its own and when combined with soy. The research team hypothesize that the two different types of pretzels may be processed by your body differently and that components in the pretzel snacks may be helpful for preventing diseases like obesity and cancer. Safflower oil and soybeans contain many natural chemicals that may benefit human health. However, this relationship is not well understood. This study will look at the impact of the pretzel snacks on your blood fat and glucose levels as well as a group of chemicals found in soy called "isoflavones". Isoflavones are natural chemicals found commonly in soybeans or foods made from them. Participants will be screened to determine if they qualify in meeting the study requirements. Participants cannot have a known allergy to dairy, soy, safflower oil, or wheat protein. Also, participants will be asked to stop eating legumes (beans, peas, soy protein, sprouts and peanuts) and to document the oils they eat for the entire 14 weeks of this study. The study will require five visits to the Ohio State University Clinical Research Center (CRC) which part of the Ohio State University's Center for Clinical and Translational Sciences. Once the investigators have determined that you qualify for this study and you decide to participate, you will be consuming three different pretzels each for one month, starting with a control pretzel. After the control pretzel treatment period, you will be randomly assigned (like the "flip of a coin") to start with one of the two pretzel groups (wheat or soy pretzel with safflower oil) for your first treatment period and then switch to the other safflower oil pretzel at your second treatment period.

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:45 - 70
Sex:Female

20 Participants Needed

This study compares the efficacy of Tamibarotene in combination with azacitidine to azacitidine in combination with placebo in participants who are Retinoic Acid Receptor Alpha (RARA) positive, and newly diagnosed with higher-risk myelodysplastic syndrome (HR-MDS), and who have not received treatment for this diagnosis. The primary goal of the study is to compare the complete remission rate between the two treatment arms.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3

550 Participants Needed

Usher syndrome (USH) causes extensive degeneration in the cochlear nerve (CN), especially in CN fibers innervating the base of the cochlea. As the first step toward developing evidence-based practice for managing implant patients with USH, this study evaluates local neural health, as well as the neural encoding of temporal and spectral cues at the CN in implanted patients with USH. Aim 1 will determine local CN health in patients with USH by assessing the sensitivity of the electrically evoked compound action potential to changes in interphase gap and pulse polarity. Aim 2 will determine group differences in neural encoding of temporal and spectral cues at the CN between patients with USH and patients with idiopathic hearing loss. Aim 3 will use supervised machine learning techniques to develop an objective tool for assessing the electrode-neuron interface at individual electrode locations.
No Placebo Group

Trial Details

Trial Status:Recruiting
Age:1 - 85

44 Participants Needed

This trial tests SPI-62, a medication for people with high cortisol due to a benign adrenal tumor. It aims to lower cortisol levels to improve health issues like diabetes, high cholesterol, high blood pressure, and weak bones.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting

30 Participants Needed

Individuals with Down syndrome (DS) live with visual deficits due, in part, to elevated levels of higher-order optical aberrations (HOA). HOAs are distortions/abnormalities in the structure of the refractive components of the eye (i.e. the cornea and the lens) that, if present, can result in poor quality focus on the retina, thus negatively impacting vision. HOAs in the general population are overall low, and thus not ordinarily considered during the eye examination and determination of refractive correction. However, for some populations, such as individuals with DS, HOAs are elevated, and thus the commonly used clinical techniques to determine refractive corrections may fall short. The most common clinical technique for refractive correction determination is subjective refraction whereby a clinician asks the patient to compare different lens options and select the lens that provides the best visual outcome. Given the cognitive demands of the standard subjective refraction technique, clinicians rely on objective clinical techniques to prescribe optical corrections for individuals with DS. This is problematic, because it may result in errors for eyes with elevated HOA given that these techniques do not include measurement of the HOAs. The proposed research evaluates the use of objective wavefront measurements that quantify the HOAs of the eye as a basis for refractive correction determination for patients with DS. The specific aim is to determine whether dilation of the eyes is needed prior to objective wavefront measurements. Dilation of the eyes increases the ability to measure the optical quality of the eye and paralyzes accommodation (the natural focusing mechanism of the eye), which could be beneficial in determining refractions. However, the use of dilation lengthens the process for determining prescriptions and may be less desirable for patients.
No Placebo Group

Trial Details

Trial Status:Recruiting
Age:5+

40 Participants Needed

This study is to understand if the study vaccine (called VLA15) is safe in healthy children. We are looking for children who: * are healthy * are age 5 through 17 * have not been diagnosed with any form of Lyme disease in the past * have not received any vaccines for Lyme disease in the past Lyme disease happens most often in children of this age. The study vaccine may be used potentially to help prevent Lyme disease. The goal of this study is to get more information about the safety of the study vaccine in this age group. Participants will be in this study for about 2 years. During that time, they will receive VLA15 or placebo (sterile saltwater solution) by a "shot" in the arm. We will compare experience of children receiving VLA15 to those receiving the placebo. Participants will not know whether they get VLA15 or placebo. Everyone participating in this study will: * get the shots in a clinic or in a hospital office * receive a total of 4 shots * receive the first 3 shots within 6 months * receive the last shot about 1 year afterwards * need to come to the trial site for 6 planned visits; 4 of these are vaccination visits and 2 are follow-up visits. We will contact you by phone 1 time every year during the study to monitor your experience. You may have extra visits if you experience a severe reaction after a vaccine dose.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:5 - 17

3235 Participants Needed

This trial is testing VX-147, a new drug, in adults and children with a genetic form of kidney disease. The drug aims to reduce the harmful effects of a specific protein in the kidneys.
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2, 3
Age:10 - 65

466 Participants Needed

12...7

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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
Learn More About Trials

Frequently Asked Questions

How much do Down Syndrome 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 Down Syndrome 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 Down Syndrome 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 Down Syndrome 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 Down Syndrome 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 Down Syndrome clinical trials?

Most recently, we added Gene Therapy for Charcot-Marie-Tooth Disease, Xenon for Out-of-Hospital Cardiac Arrest and Bowel Management Program for Colorectal Cancer to the Power online platform.

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