What side effects are associated with this type of intervention?

Alpha-1 Antitrypsin Replacement Therapy, such as Alpha-1 MP, commonly causes infusion-related reactions including headache, dizziness, fever, and chills. Other side effects may include fatigue, nausea, and localized pain or swelling at the infusion site. Serious adverse effects are rare but can include allergic reactions and an increased risk of infections.

What prior FDA approvals exist?

Alpha-1 Antitrypsin Replacement Therapy has been approved by the FDA for the treatment of Alpha-1 Antitrypsin Deficiency. This therapy involves the use of purified human alpha-1 antitrypsin protein to augment the deficient levels in affected individuals. Prior FDA-approved drugs for this condition include Prolastin, Aralast, Zemaira, and Glassia. These therapies aim to slow the progression of emphysema by maintaining adequate levels of alpha-1 antitrypsin in the blood and lungs.

What other types of research exist?

Promising, novel alternatives to Alpha-1 MP for Alpha-1 Antitrypsin Deficiency patients may include gene therapy, novel protein replacement therapies, and small molecule drugs aimed at enhancing Alpha-1 Antitrypsin function or expression. Ongoing research and clinical trials are likely to provide more options in the near future.

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Proteinase Inhibitor

Alpha-1 MP for Emphysema Due to AATD (SPARTA Trial)

Q: What is the mechanism of action of this treatment?

Alpha-1 Antitrypsin Replacement Therapy, such as the one studied in the Alpha-1 MP trial, involves regular intravenous infusions of alpha-1 antitrypsin derived from human plasma. This therapy aims to increase the levels of AAT in the blood and lungs, thereby protecting lung tissue from inflammation and damage. For patients with Alpha-1 Antitrypsin Deficiency, this treatment is essential as it helps to slow the progression of emphysema, improve lung function, and manage chronic lung damage associated with the condition.

Phase 3

Waitlist Available

Research Sponsored by Grifols Therapeutics LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume in 1 second (FEV1) ≥ 30% and < 80% of predicted and FEV1/forced vital capacity (FVC) < 70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III)

Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or 'at-risk' alleles

Must not have

Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 5 weeks prior to the Screening Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase

On the waiting list for lung surgery, including lung transplant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up weeks 26, 52, 78, 104, 130 and 156

Awards & highlights

Pivotal Trial

Summary

This trial tests if Alpha-1 MP, given through an IV drip regularly, can improve lung health in patients with lung conditions. The effectiveness will be monitored using CT scans over a long period. Alpha-1 MP is related to a therapy that has been used to reduce severe lung issues in patients.

Who is the study for?

This trial is for individuals with a specific lung condition called Alpha-1 Antitrypsin Deficiency (AATD) and related emphysema. Participants must have certain levels of lung function, documented AATD genetic variants, and evidence of emphysema. Smokers or those who've had recent alpha1-PI therapy, lung surgery, severe allergic reactions to blood products, or exacerbations of COPD are excluded.

What is being tested?

The study tests two doses (60 mg/kg and 120 mg/kg) of a drug called Alpha-1 MP against a saline placebo. Given weekly through IV for three years, the effectiveness is measured by CT scans of the lungs. The trial randomly assigns participants to receive either dose or placebo without them knowing which one they're getting.

What are the potential side effects?

While not specified here, potential side effects may include reactions at the infusion site such as pain or swelling, flu-like symptoms including fever and chills, nausea or headaches. Severe allergic reactions could occur but are less common.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My lung function test shows moderate to severe COPD.

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I have a specific genetic form of AATD.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't taken more than a low dose of steroids in the last 5 weeks.

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I am on the waiting list for lung surgery or a lung transplant.

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I have had a lung or liver transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ weeks 26, 52, 78, 104, 130 and 156

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~weeks 26, 52, 78, 104, 130 and 156

This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 26, 52, 78, 104, 130 and 156 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from Baseline in Whole lung PD15 (15th percentile point)

Secondary study objectives

Adverse Events (AEs)

Change from Baseline in PD15 of the basal lung region

Change from baseline in Saint George's Respiratory Questionnaire: Minimum value = 0, maximum value = 100, higher scores indicate worse condition

+6 more

Side effects data

From 2007 Phase 3 trial • 24 Patients • NCT00295061

Upper respiratory tract infection

Headache

100%

80%

60%

40%

20%

Study treatment Arm

Alpha-1 MP

Prolastin

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Alpha-1 MP 60 mg/kgExperimental Treatment1 Intervention

Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks

Group II: Alpha-1 MP 120 mg/kgExperimental Treatment1 Intervention

Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks

Group III: PlaceboPlacebo Group1 Intervention

0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Alpha-1 MP

2016

Completed Phase 3

~40

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Alpha-1 Antitrypsin Replacement Therapy, such as the one studied in the Alpha-1 MP trial, involves regular intravenous infusions of alpha-1 antitrypsin derived from human plasma. This therapy aims to increase the levels of AAT in the blood and lungs, thereby protecting lung tissue from inflammation and damage. For patients with Alpha-1 Antitrypsin Deficiency, this treatment is essential as it helps to slow the progression of emphysema, improve lung function, and manage chronic lung damage associated with the condition.

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