Boston, MANCT06389877Now EnrollingIRB Ready

Alpha 1-Antitrypsin Deficiency Clinical Trial in Boston, MA

Access cutting-edge alpha 1-antitrypsin deficiency treatment through this clinical trial at a research site in Boston. Study-provided care at no cost to qualified participants.

Sponsored by Beam Therapeutics Inc.

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Expert Care in Boston

Access alpha 1-antitrypsin deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related alpha 1-antitrypsin deficiency treatment provided free

Apply for This Boston Location

Check if you qualify for this alpha 1-antitrypsin deficiency clinical trial in Boston, MA

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Boston

    Convenient for MA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Boston site if eligible
  4. 4Begin participation

About This Alpha 1-Antitrypsin Deficiency Study in Boston

This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease and to determine the optimal biological dose (OBD).

Sponsor: Beam Therapeutics Inc.

Who Can Participate

Inclusion Criteria

Males or females 18 - 70 years of age inclusive at the time of consent.
Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
Blood total AAT level \<11 μM or equivalent protein in mg/dL.
Patients receiving augmentation therapy in regions where augmentation is not SoC must be willing to washout augmentation therapy for at least 6 weeks prior to signing the ICF and for the length of the study (unless clinically indicated)
A postbronchodilator FEV1 ≥40% of predicted and an FEV1/FVC \<70% at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)
Evidence of emphysema on a historic CT scan or a DLCO ≤70% of the predicted value (corrected for hemoglobin) at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)

Exclusion Criteria

Body mass index \>30
Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[\>4x/year\]).
Liver disease with any of the following:
FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI \>0.5 can be used as a surrogate exclusion criterion \[Yilmaz, 2011\].
Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.
Have ALT or AST \> upper limit of normal (ULN).
Total bilirubin levels \> ULN; if documented Gilbert's Syndrome, total bilirubin \>2 × ULN.
INR ≥1.2 at screening. If deemed appropriate by the investigator and/or prescribing physician, the patient may stop taking anticoagulants for an appropriate washout period or reversal with vitamin K and if indicated, a repeat INR within \<1.2 would be acceptable.
Seropositive for hepatitis B (positive surface Ag).
Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative. Part B: Inclusion Criteria:
Males or females 18 - 70 years of age inclusive at the time of consent.
Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).
Evidence of METAVIR F1, F2, or F3 liver fibrosis based on a central read of a baseline liver biopsy during the screening period or a histological diagnosis made no more than 6 months before enrollment and stage confirmed by central read.
A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.) Exclusion Criteria:
Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.
Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[\>4x/year\])
Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Boston?

Yes, this clinical trial (NCT06389877) has an active research site in Boston, MA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Alpha 1-Antitrypsin Deficiency Treatment Options in Boston, MA

If you're searching for alpha 1-antitrypsin deficiency treatment options in Boston, MA, this clinical trial (NCT06389877) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Boston research site is actively enrolling participants for this clinical trial. You'll receive care from experienced alpha 1-antitrypsin deficiency specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all alpha 1-antitrypsin deficiency clinical trials near you to find additional studies recruiting in your area.

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