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Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
Phase 1, PHASE2 trial testing ADVM-043 in Alpha 1-Antitrypsin Deficiency in 6 participants. Completed in 29 August 2019.
| Lead sponsor | Adverum Biotechnologies, Inc. |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | sequential |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 6 |
| Start date | 28 November 2017 |
| Primary completion | 29 August 2019 |
| Estimated completion | 29 August 2019 |
| Sites | 2 locations across United States |
Adverum Biotechnologies, Inc. — full company profile →
18 and older, any sex, with Alpha 1-Antitrypsin Deficiency. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number and proportion of subjects experiencing treatment-related adverse events related to ADVM-043
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 0 | |
| Part A: Dose 2 | 1 | |
| Part A: Dose 3 | 2 |
Number of participants with ≥1 abnormal shift from Baseline in neutrophil count, hemoglobin, important serum chemistry parameters
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 2 | |
| Part A: Dose 2 | 1 | |
| Part A: Dose 3 | 2 | |
| Total | 5 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 0 | |
| Part A: Dose 2 | 0 | |
| Part A: Dose 3 | 0 | |
| Total | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 0 | |
| Part A: Dose 2 | 1 | |
| Part A: Dose 3 | 1 | |
| Total | 2 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 0 | |
| Part A: Dose 2 | 0 | |
| Part A: Dose 3 | 0 | |
| Total | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 1 | |
| Part A: Dose 2 | 1 | |
| Part A: Dose 3 | 1 | |
| Total | 3 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 0 | |
| Part A: Dose 2 | 1 | |
| Part A: Dose 3 | 1 | |
| Total | 2 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 0 | |
| Part A: Dose 2 | 0 | |
| Part A: Dose 3 | 0 | |
| Total | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 0 | |
| Part A: Dose 2 | 1 | |
| Part A: Dose 3 | 1 | |
| Total | 2 |
Change from baseline at Week 52 of plasma concentration of M-specific A1AT for subjects who did not receive PAT post-dose Note: 1. Two subjects in Dose 1 Arm/Group, had results available at Week 52; the remaining 4 subjects in Dose 2 Arm/Group and Dose 3 Arm/Group had resumed PAT therapy after Week 24, and their results were censored from the Week 52 timepoint. 2. While data on the Total Plasma Concentrations of A1AT up to 52 Weeks were collected for 1 study participant in Part A: Dose 3, no data were collected on the Change in Plasma Concentrations of M-specific A1AT due to the initiation o
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 88.050 | ± 19.955 |
| Total: All Participants | 88.050 | ± 19.955 |
Change from baseline at Week 24 and Week 52 of total A1At plasma concentration for subjects who did not receive PAT post -dose
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 1.230 | -0.08 – 2.54 |
| Part A: Dose 2 | 1.870 | 0.14 – 3.60 |
| Part A: Dose 3 | 1.145 | 0.20 – 2.09 |
| Total: All Participants | 1.415 | -0.08 – 3.60 |
| Group | Value | 95% CI |
|---|---|---|
| Part A: Dose 1 | 1.220 | -0.44 – 2.88 |
| Part A: Dose 3 | 0.640 | 0.640 – 0.640 |
| Total: All Participants | 1.027 | -0.44 – 2.88 |
Time frame: From ADVM-043 infusion through End-of-Study visit at 52 weeks. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Part A: Dose 1 | Part A: Dose 2 | Part A: Dose 3 |
|---|---|---|---|---|
| Clostridium difficile colitis | Infections and infestations | — | — | — |
| Influenza | Infections and infestations | — | — | — |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Meningioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — | — | — |
| Reaction | System | Part A: Dose 1 | Part A: Dose 2 | Part A: Dose 3 |
|---|---|---|---|---|
| Transaminases increased | Investigations | — | — | — |
| Bronchitis | Infections and infestations | — | — | — |
| Pneumonia | Infections and infestations | — | — | — |
| Chronic sinusitis | Infections and infestations | — | — | — |
| Tooth abscess | Infections and infestations | — | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — | — |
| Viral upper respiratory tract infection | Infections and infestations | — | — | — |
| Diarrhea | Gastrointestinal disorders | — | — | — |
| Dysphagia | Gastrointestinal disorders | — | — | — |
| Nausea | Gastrointestinal disorders | — | — | — |
| Toothache | Gastrointestinal disorders | — | — | — |
| Cortisol decreased | Investigations | — | — | — |
| Crystal urine present | Investigations | — | — | — |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Face oedema | General disorders | — | — | — |
| Oedema peripheral | General disorders | — | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — | — |
| Bursitis | Musculoskeletal and connective tissue disorders | — | — | — |
| Anxiety disorder | Psychiatric disorders | — | — | — |
| Insomnia | Psychiatric disorders | — | — | — |
| Benign prostatic hyperplasia | Reproductive system and breast disorders | — | — | — |
| Hypertension | Vascular disorders | — | — | — |
Most-reported serious reactions: Clostridium difficile colitis, Influenza, Chronic obstructive pulmonary disease, Pulmonary embolism, Meningioma.
Data from ClinicalTrials.gov NCT02168686 adverse events section.
The ADVANCE study is being conducted by Adverum Biotechnologies, Inc. as an open-label, multicenter, dose-escalation study in order to assess the safety and protein expression of ADVM-043 following a single intravenous or intrapleural administration.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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