Clinical Trials

Zagociguat in Participants With Melas (Prizm)

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  2 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  1 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Nicotinamide Riboside (NR) for Mitochondrial Myopathy Disorder Treatment

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  2 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  1/2 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Health TAPESTRY: Use of Genomic Sequencing in Clinical Practice

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

A Study to Evaluate Food on Gastrointestinal Toxicity and The Effect of Selumetinib in Adolescents with Neurofibromatosis Type 1

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  1 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  3 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Adults With PMM2-CDG

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  2 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

A Phase I study of Epalrestat Therapy in a Single Patient with Phosphomannomutase Deficiency (PMM2-CDG)

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  1 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2

• Study type:

  Interventional Show What is this?

  Describes the nature of a clinical study. Types include:

   

  • Observational study — observes people and measures outcomes without affecting results.
  • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
  • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.

• Study phase:

  2 Show What is this?
  During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

About this study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.