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174 Down Syndrome Trials
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.
Imlifidase for Goodpasture Syndrome
Columbus, OhioThis 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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
50 Participants Needed
Blinatumomab + Chemotherapy for Leukemia
Columbus, OhioThis 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
Prior Safety Data
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:365 - 31
Sex:All
6720 Participants Needed
Xenon for Out-of-Hospital Cardiac Arrest
Columbus, OhioXePOHCAS: 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
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 3
Age:18 - 80
Sex:All
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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:91 - 3
Sex:All
280 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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Secondary MDS, AML, Stroke, Others
360 Participants Needed
Cedazuridine + Azacitidine for Leukemia
Columbus, OhioThis 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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2, 3
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Others
Must Be Taking:Azacitidine
235 Participants Needed
Imetelstat for Myelodysplastic Syndrome
Columbus, OhioThe 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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:18+
Sex:All
289 Participants Needed
LP352 for Dravet Syndrome
Columbus, OhioThis (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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:2 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Infantile Spasms, Neurodegenerative Disorders, Others
Must Be Taking:Antiseizure Medications
160 Participants Needed
Luspatercept for Myelodysplastic Syndrome
Columbus, OhioThe 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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Anemia, Stem Cell Transplant, AML, Others
100 Participants Needed
Venetoclax + Azacitidine for Myelodysplastic Syndrome
Columbus, OhioThis 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
Age:18+
Sex:All
53 Participants Needed
Pomalidomide + Chemotherapy for Acute Myeloid Leukemia
Columbus, OhioThis 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
Age:18 - 75
Sex:All
50 Participants Needed
Sirolimus for Cowden Syndrome with Colon Polyposis
Columbus, OhioColon 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
Age:18 - 80
Sex:All
10 Participants Needed
Venetoclax + Chemotherapy for Richter's Syndrome
Columbus, OhioThis 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
Age:18+
Sex:All
69 Participants Needed
Pacritinib for VEXAS Syndrome
Columbus, OhioThis 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:Not Yet Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
78 Participants Needed
AK117 + Azacitidine for Myelodysplastic Syndrome
Columbus, OhioThis 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
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Hypertension, Cardiopulmonary Disease, Others
Must Be Taking:Azacitidine
90 Participants Needed
EPX-100 for Dravet Syndrome
Columbus, OhioThe 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
Sex:All
Key Eligibility Criteria
Disqualifiers:Suicidal Thoughts, Cardiac Disease, Others
Must Be Taking:Aeds
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
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Acute Promyelocytic Leukemia, Others
Must Not Be Taking:CLK Inhibitors
74 Participants Needed
RAY121 for Immune System Disorders
Columbus, OhioThis 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
Age:18 - 85
Sex:All
Key Eligibility Criteria
Disqualifiers:Cancer, Hepatitis, HIV, Tuberculosis, Others
Must Not Be Taking:Antivirals, Antibiotics, Antifungals, Others
144 Participants Needed
CC-91633 for Leukemia
Columbus, OhioThis 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:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:CNS Leukemia, Cardiac Diseases, HIV, Others
Must Not Be Taking:Anticoagulants
180 Participants Needed
Safflower Oil + Soy Pretzels for Metabolic Syndrome
Columbus, OhioThis 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
Tamibarotene + Azacitidine for Myelodysplastic Syndrome
Columbus, OhioThis 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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Prior MDS Treatment, Stem Cell Transplant, Others
Must Not Be Taking:Hypomethylating Agents, Lenalidomide
550 Participants Needed
Cochlear Implant Stimulation for Usher Syndrome
Columbus, OhioUsher 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
Trial Phase:Unphased
Age:1 - 85
Sex:All
Key Eligibility Criteria
Disqualifiers:Severe Medical Comorbidities, Electrode Malposition, Others
44 Participants Needed
SPI-62 for Cushing's Syndrome
Columbus, OhioThis 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
Trial Phase:Phase 2
Age:18+
Sex:All
30 Participants Needed
Dilated vs Non-Dilated Eye Exams for Down Syndrome
Columbus, OhioIndividuals 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
Trial Phase:Phase 2
Age:5+
Sex:All
40 Participants Needed
This trial aims to evaluate the safety and tolerability of ABO-102 in patients with MPS IIIA who have already participated in a previous trial. ABO-102 is a gene therapy that delivers a healthy gene to improve their condition. ABO-102 is a gene therapy that has been developed to treat MPS IIIA by delivering a healthy gene to correct the enzyme deficiency.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Age:All
Sex:All
41 Participants Needed
Lyme Disease Vaccine for Healthy Children
Columbus, OhioThis 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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:5 - 17
Sex:All
3235 Participants Needed
VX-147 for Kidney Disease
Columbus, OhioThis 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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2, 3
Age:10 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Transplant, Uncontrolled Hypertension, Diabetes, Others
466 Participants Needed
Chemotherapy for Leukemia and Lymphoma
Columbus, OhioThis partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute lymphoblastic leukemia or B-lineage lymphoblastic lymphoma that is found only in the tissue or organ where it began (localized). Drugs used in chemotherapy 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 more than one drug (combination chemotherapy), giving the drugs in different doses, and giving the drugs in different combinations may kill more cancer cells.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:1 - 30
Sex:All
9350 Participants Needed
Chemotherapy + Surgery for Kidney Cancer (Wilms Tumor)
Columbus, OhioThis phase III trial studies how well combination chemotherapy and surgery work in treating young patients with Wilms tumor. Drugs used in chemotherapy 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. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving it after surgery may kill any tumor cells that remain after surgery.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:< 29
Sex:All
249 Participants Needed
Ecopipam for Tourette Syndrome
Columbus, OhioThe primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to \[\>=\] 6 and less than \[\<\] 12 years of age), adolescents (\>=12 and \<18 years of age), and adults (\>=18 years of age) with Tourette's Syndrome (TS).
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:6+
Sex:All
Key Eligibility Criteria
Disqualifiers:Neurological Conditions, Mood Disorders, Others
Must Be Taking:Ecopipam
150 Participants Needed
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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 Xenon for Out-of-Hospital Cardiac Arrest, Pacritinib for VEXAS Syndrome and Bowel Management Program for Colorectal Cancer to the Power online platform.