Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)

Browse trials for

• alpha 1-Antitrypsin Deficiency 142
• Emphysema 382

Keywords

• Progressive Emphysema
• Chronic Pulmonary Disease

alpha 1-Antitrypsin Deficiency

142 studies on the registry are indexed under alpha 1-Antitrypsin Deficiency; 32 are open to participants now.

This study's planned enrollment of 270 is above the median of 32 across 86 interventional studies indexed under alpha 1-Antitrypsin Deficiency.

Lead sponsor

CSL Behring is the lead sponsor of 145 studies on the registry; 17 are open to participants now.

Of its 24 completed or terminated interventional studies of FDA-regulated products, 17 (71%) have results posted.

First submitted after CSL Behring became part of CSL Limited.

Study arms

• Active comparator
  CE1226 low dose

  CE1226 at low dose.

  Biological: CE1226

• Experimental
  CE1226 medium dose

  CE1226 at medium dose.

  Biological: CE1226

• Experimental
  CE1226 high dose

  CE1226 at high dose.

  Biological: CE1226

Interventions

• BiologicalCE1226

  CE1226 will be administered via intravenous (IV) infusion weekly over 3 years.

  Also known as: Human alpha1-proteinase inhibitor, Respreeza, Zemaira

Primary outcomes

1. Annual rate of change in adjusted lung density

   The annual rate of change (expressed as gram per liter per year \[g/L/year\]) in adjusted lung density (15th percentile lung density \[PD15\]) will be calculated on CT scan assessments of whole lung at total lung capacity (TLC).

   Time frame: From Baseline to Month 36

Secondary outcomes

1. Annual rate of change in forced expiratory volume in 1 second percent predicted (FEV1%)

   The annual rate of change in FEV1% will be assessed by spirometry.

   Time frame: From Baseline to Month 36

2. Annual rate of change in diffusion capacity of carbon monoxide (DLco)

   The annual rate of change in DLco will be assessed by gas transfer using site machinery.

   Time frame: From Baseline to Month 36

3. Number of severe pulmonary exacerbations

   Pulmonary excerbations will be assessed by the investigator using Rodriguez-Roisin criteria, where an exacerbation is defined as: a sustained worsening of the patient's condition, from a stable state and beyond normal day-to-day variations, necessitating a change in regular medication in a patient with underlying chronic obstructive pulmonary disease (COPD). A severe exacerbation is defined as an event requiring the introduction of corticosteroids and / or antibiotics which results in hospitalization or death.

   Time frame: From screening up to Month 36

4. Duration of severe pulmonary exacerbations

   Pulmonary excerbations will be assessed by the investigator using Rodriguez-Roisin criteria, where an exacerbation is defined as: a sustained worsening of the patient's condition, from a stable state and beyond normal day-to-day variations, necessitating a change in regular medication in a patient with underlying COPD. A severe exacerbation is defined as an event requiring the introduction of corticosteroids and / or antibiotics which results in hospitalization or death.

   Time frame: From screening up to Month 36

5. Number of participants experiencing treatment emergent adverse events (TEAEs)

   Time frame: From screening up to Month 36

6. Percentage of participants experiencing TEAEs

   Time frame: From screening up to Month 36

Individual participant data

Plan to share: Yes — CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

Supporting information: Study protocol, Statistical analysis plan

Time frame: Requests for IPD will generally be considered once review by major regulatory authorities (i.e. FDA, EMA) is complete and the primary publication is available.

Access criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.