Abbreviation25

AAV

Adeno-associated virus, e.g. AAV2, AAV5, AAV2/5 indicates virus containing the genome of serotype 2 packaged in the capsid from serotype 5

ABE

Adenine base editor

Ad

Adenovirus

Cas9

CRISPR associated protein 9

CBE

Cytosine base editor

CDISC

Clinical Data Interchange Standards Consortium

CRISPR

Clustered regularly interspaced short palindromic repeats

DOID

Disease Ontology Identifier

Gc

Genome copies

HIV

Human immunodeficiency virus

HSV

Herpes simplex virus

LNP

Lipid nanoparticle

LV

Lentivirus

MRNA

Messenger ribonucleic acid

NCTID

National Clinical Trial identifier

ODD

Orphan Drug Designation

PFU

Plaque forming units

RMAT

Regenerative Medicine Advanced Therapy

RNP

Ribonucleoprotein

RPDD

Rare Pediatric Disease Designation

RV

Retrovirus

START

Support for Clinical Trials Advancing Rare Disease Therapeutics

TU

Transducing units

Vg

Vector genomes

VSV-G

Vesicular stomatitis virus G

Drug Product Type6

Encapsulated mRNA

Drug product consists of an mRNA-based therapeutic product, enclosed within a protective layer (such as liposomes or nanoparticles)

Encapsulated plasmid

Drug product consists of a plasmid enclosed within a protective layer (such as liposomes or nanoparticles)

Encapsulated RNP

Drug product consists of an editing protein bound to a guide RNA and enclosed within a protective layer (such as liposomes or nanoparticles)

Naked plasmid

Drug product consists of a purified plasmid DNA administered without a viral vector or encapsulating delivery vehicle

Undisclosed

Sponsor has not disclosed information on the drug product type

Viral vector

Drug product consists of any virus used to deliver therapeutic genetic material or genome-editing components

FDA Designation8

Accelerated Approval

These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint

Breakthrough Therapy

A process designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy.

Fast Track

Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need

Orphan Drug

This designation is for drugs intended to treat rare diseases or conditions that affect a small number of people, offering incentives like tax credits and market exclusivity

Priority Review

A Priority Review designation means FDA’s goal is to take action on an application within 6 months.

Rare Pediatric Disease

The rare pediatric disease PRV program aims to incentivize drug development for rare pediatric diseases.

Regenerative Medicine Advanced Therapy

Facilitates the development and expedites the review of regenerative medicine therapies, including cell therapies, therapeutic tissue engineering products, and human cell and tissue products, for serious or life-threatening conditions.

Support for Clinical Trials Advancing Rare Disease Therapeutics

(START) Pilot Program is a program designed to accelerate the development of novel drug and biological products for rare diseases by providing sponsors with enhanced communication and guidance from FDA staff.

Funder Type3

Industry

All for-profit entities

NIH

U.S. National Institutes of Health

Other Non-Profit

Includes individuals, universities, community-based organizations

Gene Delivery System Type5

Electroporation

A physical method introducing drug product into cells using an electrical pulse to briefly open pores in the cell membrane

Endocytosis

A cellular uptake mechanism where the cell membrane engulfs the drug product, allowing its therapeutic contents to be released inside target cells

Not Applicable

A designation used when the drug product lacks a specific delivery system type, such as naked DNA directly injected into tissue

Undisclosed

A designation used when the sponsor has not disclosed information regarding the delivery system used to introduce the drug product to cells

Viral transduction

A biological delivery process utilizing a modified viral vector to infect target cells and transfer genetic material

Mechanism of Action9

Exon skipping/splice editor

Gene therapy product is intended to modify pre-mRNA splicing to restore or improve expression of a functional protein

Functional gene replacement

Gene therapy product is intended to introduce a functional copy of a gene to restore normal gene function

Gene excision

Gene therapy product is intended to excise a disease-causing gene or pathogenic genomic sequence

Gene inactivation

Gene therapy product is intended to permanently disrupt or inactivate a disease-causing gene to reduce or eliminate its function

Genetic delivery of therapeutic protein

Gene therapy product is intended to enable endogenous expression of a therapeutic protein, such as an antibody, enzyme or cytokine

MiRNA knockdown of mutant/aberrant gene

Gene therapy product is intended to express miRNAs that reduce expression of a disease-causing gene or allele

Mutation correction

Gene therapy product is intended to directly correct a disease-causing genetic variant

Overexpression of protective allele/gene

Gene therapy product is intended to increase expression of a protective gene or allele to mitigate disease pathology

Undisclosed

Sponsor has not disclosed the drug product's mechanism of action

Non-Viral Vector Type3

Liposome

A synthetic lipid vesicle consisting of one or more bilayers that encapsulates active cargo into both hydrophobic and hydrophilic compartments

LNP

A non-viral delivery vehicle formed primarily of lipids that encapsulates one or more active cargo into a particle with nanoscale dimensions

Transposon

A non-viral integration system formed of terminal inverted repeats and a transposase enzyme that inserts genetic cargo into a host chromosome

Phases6

Early Phase I

Describes exploratory trials conducted before traditional Phase I trials to investigate how or whether a drug affects the body, have no therapeutic or diagnostic goals

Phase I

Describes clinical trials that focus on the safety of a drug

Phase II

Describes clinical trials that gather preliminary data on effectiveness, continue to monitor safety

Phase I/II

Combination Phase I/Phase II clinical trial

Phase III

Pivotal experiments to gather data on safety and effectiveness

Phase II/III

Combination Phase II/Phase III clinical trial

Route Of Administration25

Cutaneous

Administration to the skin

Intra-arterial

Administration within an artery or arteries

Intra-articular

Administration within a joint

Intrabronchial

Administration within a bronchus

Intracardiac

Administration within the heart

Intracerebral

Administration within the cerebrum

Intracisternal

Administration within the cisterna magna cerebellomedularis

Intracochlear

Administration within the cochlea

Intracoronary

Administration within the coronary arteries

Intraglandular

Administration within a gland

Intramedullary

Administration within the marrow cavity of a bone

Intramuscular

Administration within a muscle

Intraneural

Administration of a substance directly into a nerve or nervous tissue

Intraocular

Administration within the eye

Intraparenchymal

Administration within or into the parenchyma of a targeted organ

Intrathalamic

Administration within the thalamus

Intrathecal

Administration within the cerebrospinal fluid at any level of the cerebrospinal axis, including injection into the cerebral ventricles

Intravenous

Administration within or into a vein or veins

Intraventricular

Administration within a ventricle, also called intracerebroventricular

Intravitreal

Administration within the vitreous body of the eye

Respiratory (Inhalation)

Administration within the respiratory tract by inhaling orally or nasally for local or systemic effect

Subretinal

Administration beneath the retina

Suprachoroidal

Administration above the choroid

Topical

Administration to a particular spot on the outer surface of the body

Undisclosed

Sponsor has not disclosed information about the route of administration

Study Status7

Active, Not Recruiting

Study has ongoing, but is not enrolling new participants

Completed

Study has concluded normally

Not Yet Recruiting

Study has not started enrollment

Recruiting

Study is actively looking for participants

Suspended

Study halted prematurely but has the potential to resume

Terminated

Study was halted prematurely and will not resume, participants are no longer recieving intervention

Unknown

Study has passed its completion date, but last known status was not listed as Completed, Terminated or Withdrawn. Status has not been verified within the past 2 years. Studies with an unknown status are considered closed studies

Table Column Header56

Actual Study Start Date (m/d/y)

The actual date on which the first participant was enrolled in a clinical study

Adult/Pediatric/Both

Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both

Ages Eligible for Study

More specific age ranges eligible for the study

Clinical Publications

URL connections to clinical data on human subjects

Completion Date

The anticipated date that the primary outcome measure data will be complete (last participant data collected)

Compound Name

The interventional compound given to the study subjects

Countries

List of countries that contain at least 1 clinical trial site

Current Phase

The current phase of the clinical trial, determined based on the studies' objective

Delivery System

Describes the nature of the drug substance or process that is used to deliver the editor or corrected gene

Description

Text summitted by the sponsor to Clinicaltrials.gov, outlining the purpose of the study

Development Status

Indicates whether or not the clinical development program is ongoing, or if the drug is approved

Disease Ontology Term

A standardized, unique Disease Ontology Identifier (DOID) applied to the disease indication/s of the study

Dose levels

List of doses given in the indicated clinical trial, may be expressed in specific units, or a range of possible doses

Drug Product Alias

Additional names the investigational product has gone by during its clinical development

Drug Product Description

Short text description of the drug product that should include information on the therapy type, drug product type, vector type and contents, editor (if any), transgene, target locus, and mechanism of action

Drug Product Name

Primary name of the investigational product given to the study subjects

Drug Product Type

Broadly classifies advanced genetic therapies according to how the drug product is packaged and delivered into a patient's body

Editor Type

For gene editing type therapies, the protein that will perform the gene correction

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate) and exclusions criteria (which prevent a person from participating).

Eligible Age

More specific age ranges eligible for the study

Enrollment Count

Number of study subjects planned or actually enrolled in the study

Estimated Primary Completion Date (m/d/y)

The anticipated date that the primary outcome measure data will be complete (last participant data collected)

FDA Designations

The formal status granted by the US Food and Drug Administration that confers defined procedural, development, review, or incentive-related consequences for a regulated medical product. Each drug product may have multiple designations

Funder Type

Describes the organization that provides support for the clinical study

Gene Delivery System Type

Describes the nature of the process that is used to deliver the drug product, such as an editor or corrected gene (see Drop Down Menu)

Has US IND?

Indication that a clinical study is studying a drug product subject to section 505 of the Federal Food, Drug, and Cosmetic Act or to section 351 of the Public Health Service Act.

Indication

The disease, disorder, syndrome, illness, or injury that is being studied

Last Update

The most recent date on which changes to a study record were made available on ClinicalTrials.gov

Locations

List of countries that contain at least 1 clinical trial site

Mechanism of Action

Simplified description of the means by which the drug product achieves an intended therapeutic effect or action

NCT Number

Unique identification code given to each clinical study upon registration at ClinicalTrials.gov

News and Press Releases

URL connections to press releases generated by drug product Sponsor

Nonclinical Publications

URL connections to nonclinical data (in vitro, animal data)

Non-Viral Vector Type

Free text column to specify which non-viral vector is used in the drug product

No. of Trial Sites

Number of clinical sites where the clinical trial is conducted

Phases

The stage of the clinical trial, determined based on the studies' objective

Protocols

URL connections to clinical trial protocols, study design papers

Recent Updates

News, updates on recent or anticipated regulatory milestones

Recruitment Status

Indicates the current recruitment status or the expanded access status

Regulatory Element

Text description of a regulatory element included in the vector expression cassette (might be a promotor or enhancer)

Related NCTID

URL connections to other clinicaltrials.gov records where the same (or related) study compound was tested, or observational studies conducted by the Sponsor relevant to the studied indication

Results Posted

Indicates if summary results are posted to the clinical trial record

Route of Administration

The path by which a drug product is introduced to the body, using CDISC SDTM Terminology

Sexes Eligible for Study

A type of eligibility criteria that indicates the sex of people who may participate in a clinical study (all, female, male)

Sponsor

The organization or person who initiates the study and who has authority and control over the study

Sponsor Class

Describes the organization that provides support for the clinical study

Standard Ages

Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both

Study Title

A short title submitted to ClinicalTrials.gov by the study sponsor that describes what the study is about

Submit Date

The date on which the study sponsor or investigator submitted a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria

Target Gene or Variant

Novel gene or genetic material, i.e. DNA or RNA that is artificially introduced into an organism to correct disease, or the endogenous locus (gene symbol or genomic coordinates) where a genome editor is directed to correct disease.

Target Tissue or Cell

Lists target cells if the therapy is directed at a particular cell type (either by ex-vivo enrichment, or tissue-specific regulatory elements)

Therapeutic Modality

Indicates whether drug product achieves its intended effect by gene transfer or gene editing

Therapy Route

Describes whether the gene therapy is introduced to cells in-vivo or ex-vivo

Trial Enrollment

Number of study subjects planned or actually enrolled in the study

Viral Vector Subtype

Free text column to give additional specifics (if known) about the viral vector used in the drug product (e.g. AAV serotype and capsid)

Viral Vector Type

Describes the base virus family the viral drug product belongs to

Therapeutic Modality3

Gene editing

A gene therapy where disease-causing variant is corrected via a gene editor

Gene transfer

A gene therapy where a corrected gene is administered to relevant cells/tissues via a delivery system

Undisclosed

Sponsor has not disclosed whether the therapy works by gene editing or gene transfer

Therapy Route3

Ex vivo

Autologous cells are modified outside the body to create the drug product, before reintroduction to the intended recipient

In vivo

The drug product is given directly to the intended recipient, and the drug product's mechanism of action takes place inside the body of the intended recipient

Undisclosed

Sponsor has not disclosed whether the drug product is introduced to cells in vivo or ex vivo

Viral Vector Type7

Adeno-associated virus

A small-capacity single-stranded DNA virus that can infect dividing and non-dividing cells and does not require genome integration to express its cargo

Adenovirus

A medium to large capacity double-stranded DNA virus that can infect dividing or non-dividing cells and does not require genome integration to express its cargo

Herpesvirus

A very large-capacity double-stranded DNA virus that can infect dividing or non-dividing cells and does not require genome integration to express its cargo

Lentivirus

A medium-capacity single-stranded RNA virus that can infect dividing or non-dividing cells and integrates into the host genome to express its cargo

Other

Viral drug product is based on a viral type that is not AAV, Ad, HSV, LV, RV

Retrovirus

A medium-capacity single-stranded RNA virus that infects dividing cells and integrates into the host genome to express its cargo

Undisclosed

Sponsor has disclosed that the drug product is a viral vector, but has not disclosed any details about the type of virus used

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