AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry

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Brief Title

AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry

Official Title

AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry

Brief Summary

      This is a longitudinal, observational, non-interventional registry study, designed to collect
      both retrospective and prospective real world data, on patients receiving Alpha-1
      augmentation therapy in the home through Coram Specialty Infusion.The data will be collected
      from standard Coram homecare forms, as well as patient reported case forms.
    

Detailed Description

      This is a longitudinal, observational, non-interventional registry study, designed to collect
      both retrospective and prospective data, in order to acquire real-world data on product
      prescribing, product use by patients, and product performance for patients receiving α1-PI
      therapy in a homecare and/or a Coram ambulatory infusion suite. There is no limit on the
      number of subjects that may participate in the registry. The AL1TER Registry will be overseen
      by the Principal Investigator listed on the first page of this protocol. No less than 40
      physicians in the US will participate in this registry; there will be no upper limit to this
      number. All patients ≥18 years old, not members of a vulnerable study population, and who
      require α1-PI therapy may be invited to participate in this study.

      Participation in the AL1TER Registry will continue as long as the subject is:

      Being treated with α1-PI therapy using Coram's infusion services, Using an insurance carrier
      for which Coram is an approved provider, Willing to participate in this registry, and Until
      the Sponsor, Principal Investigator or subject decides to terminate their participation.

      The data collection for this study differs from other registries in that data going into the
      AL1TER Registry is being collected by homecare agency personnel (i.e., pharmacists and
      nurses). The data will be collected from standard Coram homecare forms. AL1TER Registry data
      will be entered into Coram's standard patient database and processed in the same manner as
      for non-registry patients but will be extracted in a systematic fashion for incorporation
      into the AL1TER Registry. Data collected from the St. George Respiratory Questionnaire
      (SGRQ), and the Life Quality index Questionnaire (LQIQ), and subject medical chart review may
      also be entered into the AL1TER Registry database. All data will be de-identified for use
      when reporting results of the database.

      All subjects participating in the AL1TER Registry will be informed about the Registry by the
      submitting investigators, or their designee. Additionally with physician permission, registry
      staff can contact patients via mail and phone to consent, only after patients have been
      accepted on Coram's service and agreed to start therapy. Collection of subject data for
      incorporation into the AL1TER Registry will begin only after the subject signs the informed
      consent form.

      In addition to the standard visit/data collection already carried out by Coram, subjects will
      also be asked to complete two short quality of life questionnaires every six months beginning
      with 30 days after the date subject signs informed consent.

      This registry study will continue until such time as the sponsor, Coram Clinical Trials,
      makes the decision to terminate data collection. At such time all submitting investigators,
      subjects and the IRB will be notified of study termination. Subject participation will begin
      from the date they sign informed consent and will continue until the sponsor, Principal
      Investigator, submitting investigator, or subject decides to terminate their participation or
      the registry itself is terminated. The authorization by a subject for use of their Protected
      Health Information (PHI) for the AL1TER Registry has no expiration date.

      The AL1TER Registry involves compilation of the standard data collected for all patients
      receiving α1-PI therapy within the Coram system; data collected from the SGRQ and LQIQI, and
      relevant items from patient medical history, including pulmonary function tests. Since the
      registry is both retrospective and prospective, forms from the start of the patients care,
      through to the end of their care will be made available for entry into the registry, even
      forms that predate the signing of the ICF. Sample forms of the standard data collected for
      Coram patients are included in the appendices. The information that is captured for each
      α1-PI patient using Coram's standard forms may include:

      Patient Start of Care:

      Patient information (contact, date of birth, height, weight, gender, tobacco/alcohol/drug
      use) Age at time of development of symptoms, and age at time of diagnosis Medical/surgical
      history Alpha-1 Genotype Baseline FEV1 value Therapy and safety review (diagnosis, therapy,
      administration method, PICC length, date of insertion, etc.) Care plan

      Ongoing Patient Assessment:

      Patient information Changes in dosing Infections/illness Antibiotic use Primary care
      physician visits Emergency room visits Hospitalizations Effects from infusions Other changes
      in health Care plan update(s) Clinical monitoring (lab values, weight, nutrition screen, FEV1
      values.)

      Home Visit Assessments:

      Patient information Therapy information Medical history and medications (initial and ongoing)
      Vital signs/weight Patient/home safety Access device for infusion therapy Nursing
      interventions Assessments: respiratory, cardiovascular, gastrointestinal, genitourinary,
      musculoskeletal, neurosensory, integumentary, endocrine, pain Medication administration
      information Teaching and compliance Insurance Information Insurance carriers Insurance
      coverage Reimbursement variances
    


Study Type

Observational [Patient Registry]


Primary Outcome

Change in Pulmonary Function

Secondary Outcome

 Change in Quality of Life

Condition

Alpha-1 Anti-trypsin Deficiency


Study Arms / Comparison Groups

 Adult Alpha-1 Patients
Description:  Adult Alpha-1 patients receiving augmentation therapy in the home through Coram

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

120

Start Date

October 2013

Completion Date

September 6, 2019

Primary Completion Date

September 6, 2019

Eligibility Criteria

        Inclusion Criteria:

          -  Patients must meet the following criteria to participate in this study:

        Signed informed consent Either male or female, ≥18yrs of age requiring α1-PI therapy Agree
        to the use of Coram's infusion services upon entry into AL1TER Registry

        Exclusion Criteria:

          -  Patients will be excluded from participation in the study for any of the following
             reasons:

        Currently using an insurance provider for which Coram's services are not covered/authorized
        Have previously participated in the AL1TER Registry and revoked consent to the use of their
        Protected Health Information in theAL1TER Registry database Under the age of 18, or member
        of a vulnerable population (prisoner, unable to understand the English version of the
        consent, or unable to sign consent themselves)
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Donald Schmechel, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01974830

Organization ID

CT-10-04


Responsible Party

Sponsor

Study Sponsor

Coram Clinical Trials


Study Sponsor

Donald Schmechel, MD, Principal Investigator, Southeast Neurology and Memory Clinic


Verification Date

September 2019