Expanded Access for Hereditary Angioedema (HAE)

February 5, 2024 updated by: Joshua S. Jacobs, MD

Approval for access is solely at CSL Behring's discretion and the following criteria must be met for CSL Behring to consider access to experimental or unlicensed product:

  • The patient has an unmet medical need that is life-threatening, chronic or seriously disabling, as defined by local regulations and applicable to the particular patient. All treatment options available to the patient should have been explored and exhausted by the physician, including participation in clinical trials.
  • The patient and caregiver must be informed of potential risks and benefits of receiving the experimental therapy. The overall potential benefit should outweigh the combined potential risks of the experimental medicine and the outcome of the illness or disease itself. If approved, CSL Behring will provide treating physicians with risk information wording for inclusion in the physician's informed consent form, which will outline the risks of the experimental medicine. Treating physicians must share this information with the patient through the informed consent process. Treating physicians must also agree to participate in CSL Behring's safety monitoring and reporting requirements and follow FDA safety reporting regulations.
  • Only medicines under active clinical development in the USA by CSL Behring will be considered for early access. There must be sufficient clinical trial data on efficacy and safety indicating a potential benefit greater than risks for the intended use and dose.
  • Granting early access should not negatively impact or delay the ongoing development program with the experimental medicine to support approval by FDA and other regulatory authorities.
  • Adequate supply of the experimental medicine must exist to support both ongoing clinical trials and approved expanded access until, and if, the product becomes commercially available.

Only requests received from the licensed physician on behalf of the patient will be considered. CSL Behring will assess the request against the above criteria. If these criteria are met and the IRB/Ethics Committee and FDA approve access to the experimental therapy, early access may be granted. US medicine importation requirements must also be met. The patient's physician is responsible for the patient's ongoing care as well as all relevant US federal and state legislations while the patient is receiving the experimental medicine through early access. If approved the patient/caregiver must provide informed consent and the treating physician must follow safety and monitoring requirements as defined by CSL Behring.

CSL Behring cannot provide a guarantee that expanded access will be available for every experimental medicine. Even if such a program is offered CSL Behring cannot guarantee that an experimental medicine will be available to a particular patient. The early access will cease if clinical trials or regulatory assessment determines the product does not demonstrate a positive risk benefit for patients. Once the experimental medicine is approved and commercially available in the US, early access will no longer be available.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Approved for marketing

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Expanded Access

Expanded Access Type

Expanded Access Type

Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
  • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
  • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
  • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
  • Individual Patients

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

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 in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

-

Exclusion Criteria:

-

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Joshua S. Jacobs, MD

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 30, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 2, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 6, 2017

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 7, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 5, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2024

More Information

Terms related to this study

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CSL830

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on CSL830

Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

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