Ended Phase I and Phase II (Integrated)- First administration to humans Pulmonary emphysema

A Phase 1/2 Study of NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease

EU CTIS ID: 2023-508138-33-00

What this study is testing

To evaluate the safety and tolerability of NTLA-3001 following a single treatment in adult participants with AATD-associated lung disease

Phase I and Phase II (Integrated)- First administration to humans

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1. Male or female participants, 18 to 75 years of age inclusive, at the time of signing informed consent.
10. Participants must provide written informed consent before any protocol-specified assessment is performed.
11. Participants must agree to the alcohol consumption restrictions.
12. Participants must have a negative cotinine test and agree to the smoking and nicotine restrictions.
2. Diagnosis of AATD-associated lung disease meeting specific criteria.
3. 3. Per Investigator assessment, the participant has either maximized or refused available standard of care. For participants on AAT augmentation therapy, the Investigator determines that it is clinically acceptable to hold augmentation therapy for at least 4 weeks prior to dosing and refrain from initiating augmentation therapy for at least 12 weeks postdosing. The Investigator will review the protocol with the participant, including temporary withholding of AAT augmentation therapy, and potential consequences.

You likely can't join if

1. Impaired liver function.
10. History of active malignancy within 5 years prior to Screening or during the Screening period, except curatively resected basal cell or squamous cell carcinoma of skin.
11. Prior liver, heart, or other solid organ transplant; lung volume reduction surgery (LVRS); bone marrow transplant; or anticipated transplant or LVRS within 1 year of Screening. Note: Prior history of or planned corneal transplant is not exclusionary.
12. Prior receipt of any gene therapy.
13. Receiving an investigational intervention or participating in another clinical study within 30 days or within 5 half-lives of the drug prior to Screening. Note: Observational, non-interventional registry trials (studies with no procedural assessments) are acceptable.
14. Uncontrolled hypertension (systolic BP > 160 mmHg, diastolic BP > 100 mmHg) despite maximal medical treatment.

See the full eligibility criteria

Who can join

1. Male or female participants, 18 to 75 years of age inclusive, at the time of signing informed consent.
10. Participants must provide written informed consent before any protocol-specified assessment is performed.
11. Participants must agree to the alcohol consumption restrictions.
12. Participants must have a negative cotinine test and agree to the smoking and nicotine restrictions.
2. Diagnosis of AATD-associated lung disease meeting specific criteria.
3. 3. Per Investigator assessment, the participant has either maximized or refused available standard of care. For participants on AAT augmentation therapy, the Investigator determines that it is clinically acceptable to hold augmentation therapy for at least 4 weeks prior to dosing and refrain from initiating augmentation therapy for at least 12 weeks postdosing. The Investigator will review the protocol with the participant, including temporary withholding of AAT augmentation therapy, and potential consequences.
4. No prior diagnosis of protein-losing enteropathy or nephropathy or history of hypoalbuminemia.
5. AAV TAb below the laboratory assay cut-off titer.
6. Participants must meet specific laboratory criteria.
7. aPTT, INR, fibrinogen and D-dimer within the reference range or clinically nonsignificant per Investigator assessment.
8. Male participants must agree to the contraceptive requirements and sperm donation restrictions.
9. Female participants must not be pregnant or breastfeeding and must agree to the contraceptive requirements and egg (ova, oocyte) restrictions.

What rules you out

1. Impaired liver function.
10. History of active malignancy within 5 years prior to Screening or during the Screening period, except curatively resected basal cell or squamous cell carcinoma of skin.
11. Prior liver, heart, or other solid organ transplant; lung volume reduction surgery (LVRS); bone marrow transplant; or anticipated transplant or LVRS within 1 year of Screening. Note: Prior history of or planned corneal transplant is not exclusionary.
12. Prior receipt of any gene therapy.
13. Receiving an investigational intervention or participating in another clinical study within 30 days or within 5 half-lives of the drug prior to Screening. Note: Observational, non-interventional registry trials (studies with no procedural assessments) are acceptable.
14. Uncontrolled hypertension (systolic BP > 160 mmHg, diastolic BP > 100 mmHg) despite maximal medical treatment.
15. Participants who have known hypersensitivity to any LNP component (or its excipients) or formulation buffer used to suspend the viral vector, or contraindication to high-dose steroids.
16. Unable or unwilling to take the required pretreatment medication regimen or postinfusion corticosteroid regimen.
17. Participant is not considered suitable for study inclusion in the opinion of the Investigator for other reasons.
2. Any of the following within 12 months prior to Screening: (a) Myocardial infarction; (b) Transient ischemic attack; (c) Cerebrovascular accident; (d) Pulmonary embolism; (e) New York Health Association Class III or IV heart failure.
3. History of hepatitis B or C infection or positive hepatitis B surface antigen (HbsAg) or hepatitis C virus antibody (HCV Ab) test. Laboratory results confirmed at Day -1.
4. History of positive HIV status or positive HIV status at Screening. Laboratory results confirmed at Day -1.
5. Known or suspected systemic parasitic, fungal, or viral infection, including COVID-19, or received antibiotics for bacterial infection; vaccines within 14 days prior to Screening; live vaccines within 30 days prior to Screening. Status confirmed at Day -1.
6. Use of corticosteroids above 5 mg/day of prednisone (or equivalent) or other immunosuppressive medications within 4 weeks prior to Screening.
7. Have had a serious COPD exacerbation (as determined by the Investigator) or used antibiotics for a COPD exacerbation or respiratory infection within 4 weeks prior to Screening.
8. History of anaphylaxis or severe systemic reaction to AAT augmentation therapy, or immune response to AAT augmentation therapy as indicated by clinical history of an adverse immune response to infusion with decreased therapeutic effect in combination with documentation of serum anti-AAT antibodies.
9. History of alcohol or drug abuse within 3 years prior to Screening.

The study team makes the final eligibility decision.

Where it's taking place

New Zealand
United Kingdom

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include New Zealand; United Kingdom. Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.