Alpha-1-antitrypsin deficiency (A1AD/AATD)

Clinics: Greater Manchester, South Yorkshire, Teesside and West Yorkshire

Therapy Area: Alpha-1 antitrypsin deficiency

Gender and Age: Males & Females aged 18 – 72

Reimbursement: £2600 plus reasonable travel expenses or transport provided.

Alpha-1 antitriptysin deficiency

About The Research...

The purpose of this clinical trial is to test a new medicine for treating alpha-1-antitrypsin deficiency (A1AD/AATD).

A1AD/AATD is an inherited condition, most often caused by a gene (DNA) mutation called the Z mutation – that mutation affects how the body makes a protein called alpha-1-antitrypsin (A1AT).

There’s currently no effective treatment for A1AD/AATD and the condition causes lung disease (such as emphysema or chronic obstructive pulmonary disease [COPD]). People who have A1AD/AATD may also have problems with their liver.

The clinical trial aims to assess the side effects and blood levels of the study medicine, and the effect of the study medicine on blood levels of A1AT. We hope the study medicine will work by changing how the Z mutant of A1AT is made in the body, thereby increasing its levels in the bloodstream.

Following a successful medical screen and upon review of medical history, a minimum of 19 volunteers who have at least 1 copy of the Z mutation will take oral solutions of the study medicine twice daily and attend a total of 13 outpatient visits over 8 weeks.

There is no obligation for you to take part in this research and there will be no direct benefit to you, but if you do decide to participate you are contributing to the scientific knowledge which may lead to a treatment option for people living with A1AD/AATD.

A full description of the study will be given before you decide to take part, both over the phone and during an appointment at a MAC clinic. This will include you receiving the full patient information sheet (PIS) and the opportunity to discuss the study in depth with a doctor. Your GP will also be notified of your participation.

If you contact us it does not mean you have to decide at that point whether to participate. If you do decide to participate you are free to withdraw at any time.


£2600 plus reasonable travel expenses or transport provided.


Key Inclusion Criteria:

  • Male or Females aged 18 to 72
  • Diagnosis Alpha-1-antitrypsin (A1AD/AATD) genotype PiXZ
  • BMI 18 – 35 kg/m2


Key Exclusion Criteria:

  • Inability to stop smoking for 30 days before start of study and withhold for duration of trial
  • History of cancer in the last 5 years


Other eligibility criteria will apply. Please contact MAC for more information.

If you are eligible you will receive a comprehensive health screen. Your GP will be fully informed of your participation and provided with any information relevant to your healthcare.

Key Inclusion Criteria


Key Exclusion Criteria


MAC Clinical Research is currently conducting a new clinical research study for people with Alpha-1-antitrypsin deficiency (A1AD/AATD).

If you would like more information, please fill in the form below and we will be in touch…

Alternatively, please call our recruitment team on freephone 0800 633 5507.

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