DM1 Clinical Trial - Induction of Pluripotent Stem Cells From Human

See also
Status	Clinical Trial	Phase
Completed	`NCT05027269` - Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients	Phase 1/Phase 2
Recruiting	`NCT06411288` - Global Study of Del-desiran for the Treatment of DM1	Phase 3
Active, not recruiting	`NCT05479981` - Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients	Phase 2

See also

Status

Clinical Trial

Phase

Completed

NCT05027269 - Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Phase 1/Phase 2

Recruiting

NCT06411288 - Global Study of Del-desiran for the Treatment of DM1

Phase 3

Active, not recruiting

NCT05479981 - Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Phase 2

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT02084407
Study type	Interventional
Source	Institut de Myologie, France
Contact
Status	Completed
Phase	N/A
Start date	November 2013
Completion date	August 2014

Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms