Emphysema Secondary to Congenital AATDPhase 1NCT05315921
Study of OsrhAAT or Placebo in Healthy Volunteers
A Phase 1 study testing OsrhAAT 1 mg/kg IV, OsrhAAT 3 mg/kg IV and OsrhAAT 10 mg/kg IV in people with Emphysema Secondary to Congenital AATD, run by Healthgen Biotechnology Corp. at 1 site in United States.
Trial summary
A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Immunogenicity of Single Ascending Doses of OsrhAAT in Healthy Volunteers Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Emphysema Secondary to Congenital AATD
- What is being tested?
- OsrhAAT 1 mg/kg IV, OsrhAAT 3 mg/kg IV and OsrhAAT 10 mg/kg IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Mar 2022to 30 Jun 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Subjects must meet all the following criteria to be enrolled in the trial:
- Able to understand and willing to sign the ICF
- Healthy subjects, male or female, non-smokers, 18-55 years of age
- No significant medical history, and in good health as determined by detailed medical history (neurological, endocrinal, cardiovascular, pulmonary, hematological, immunologic, psychiatric, gastrointestinal, renal, hepatic, and metabolic disease), full physical examination, vital signs, 12-lead electrocardiogram (ECG), urinalysis and laboratory tests at screening. For eligibility purposes, abnormal laboratory or vital signs results may be repeated once if abnormal result is observed at the initial reading. Moreover, abnormalities found in the ECG may need to be confirmed by repeated measurements.
- Adequate organ function according to the following laboratory values:
- Bone marrow function (absolute neutrophil count ≥1500/mm3 and platelet count ≥100,000/mm3).
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- Alanine aminotransferase (ALT) 7-56 units per liter of serum ( or institutional equivalent), AST 5-40 units per liter of serum (or institutional equivalent), alkaline phosphatase 20-140 units per liter of serum (or institutional equivalent), total bilirubin 0.1-1.0 mg/dL (or institutional equivalent) and creatinine clearance (Cockcroft-Gault equation) ≥90mL/min.
- Female of non-childbearing potential (i.e., physiologically incapable of becoming pregnant, including any female who is 1 year of post-menopausal with a FSH > 40mIU/mL, or surgically sterile [defined as having a bilateral oophorectomy, hysterectomy or tubal ligation]) or agree to one of the following to prevent pregnancy and, if a woman of childbearing potential, have a negative pregnancy test at screening:
- Practicing abstinence which is the preferred and usual lifestyle of the subject
- If a sexually active woman of childbearing potential (sexually active with a non-sterile male partner) agrees to prevent pregnancy by using double methods of contraception as follow until 180 days after the administration of the investigational product:
- Simultaneous use of intra-uterine contraceptive device, placed at least 4 weeks prior to study drug administration, and condom for the male partner.
- Simultaneous use of hormonal contraceptives, starting at least 4 weeks prior to study drug administration and must agree to use the same hormonal contraceptive throughout the study, and condom for the male partner.
- Simultaneous use of diaphragm with intravaginally applied spermicide and male condom for the male partner, starting at least 21 days prior to study drug administration. Male subjects who are not vasectomized for at least 6 months and who are sexually active with a non-sterile female partner must agree to use double methods of contraception below from the first dose of randomized study drug until 120 days after their dose and must not donate sperm during their study participation period:
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- Simultaneous use of a male condom and, for the female partner, hormonal contraceptives (used since at least 4 weeks) or intra-uterine contraceptive device (placed since at least 4 weeks)
- Simultaneous use of a male condom and, for the female partner, a diaphragm with intravaginally applied spermicide
- Body mass index (BMI) 18.0-32.0 kg/m2 and body weight ≥ 50.0 kg for males and
- 45.0 kg for females
- Blood pressure ≤ 139/89 mm Hg
- Able to follow the study protocol and complete the trial
Who cannot join
- Subjects who meet any of the following criteria cannot be enrolled:
- History of severe infection within 4 weeks prior to administration; signs and symptoms of any active infection regardless of severity within 2 weeks prior to administration.
- History of hypersensitivity to OsrhAAT or any excipient or similar drugs
- Known History of hypersensitivity to rice
- Use of any prescription drugs, herbal supplements, or nonprescription drugs, including oral antihistamines (for seasonal allergies), within 1 month or 5 half-lives (whichever is longer) prior to study drug administration, or dietary supplements within 1 week prior to study drug administration, unless, in the opinion of the Investigator and the Sponsor, the medication will not interfere with the study. Over-the-counter multivitamins will be permitted. If needed, paracetamol/acetaminophen may be used, but must be documented in the Concomitant medications/Significant non-drug therapies page of the source data. Any questions of concomitant medications should be directed to the Sponsor.
- Participation in a clinical research study involving the administration of an investigational or marketed drug or device within 30 days prior to the first dosing, administration of a biological product in the context of a clinical research study within 90 days prior to the first dosing, or concomitant participation in an investigational study involving no drug or device administration.
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- Donation of blood 12 week prior to dosing
- Pregnant, or nursing females
- A history of psychiatric and psychological condition that, in the judgment of the Investigator, may interfere with the planned treatment and follow-up, affect subject compliance or place the subject at high risk from treatment-related complications
- A marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval >450 milliseconds [ms], Bazett Formula: QTc=QT/RR0.5）
- Active hepatitis B or C. HBV carriers without active disease (HBV DNA titer< 1000 cps/mL or 200 IU/mL), or cured Hepatitis C (negative HCV RNA test) may be enrolled, in the judgement of the Investigator.
- Known infection with human immunodeficiency virus (HIV) and a cluster of differentiation 4 (CD4) count that is unknown or documented to be < 350 cells/mm3 within 12 months, or an Acquired Immune Deficiency Syndrome (AIDS)-defining illness
- Known history of severe IgA deficiency
- Immunization with a live or attenuated vaccine is prohibited within 4 weeks prior to study drug administration. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g., FluMist®) are live attenuated vaccines and are not allowed
- Receipt of an immunoglobulin or blood product 90 days prior to dosing
- History of consuming more than 14 units of alcoholic beverages per week or of alcoholism or drug/chemical/substance abuse within past 2 years prior to screening (Note: one unit = 12 ounces of beer, 4 ounces of wine or 1 ounce of spirits). Consumed > 3 (male) or 2 (female) units of alcohol as determined by blood alcohol testing at screening
- History of significant drug abuse within one year prior to screening
- Positive urine drug screen (amphetamines, barbiturates, benzodiazepines, cannabinoids, cocaine, cotinine and opiates)
- Any reason which, in the opinion of the Investigator, would prevent the subject from participating in the study
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Altasciences
Cypress, California, 90630
Yongjun David Kim, MD, 714-252-0700 ext. 1482, YKim@altasciences.com
About this study
Official title: A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Immunogenicity of Single Ascending Doses of OsrhAAT in Healthy Volunteers
A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Immunogenicity of Single Ascending Doses of OsrhAAT in Healthy Volunteers
- Conditions
- Emphysema Secondary to Congenital AATD
- Being tested
- OsrhAAT 1 mg/kg IV, OsrhAAT 3 mg/kg IV, OsrhAAT 10 mg/kg IV, OsrhAAT 20 mg/kg IV, OsrhAAT 40 mg/kg IV
- Keywords
- OsrhAAT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OsrhAAT 1 mg/kg IVDrug
Drug: OsrhAAT 1 mg/kg IV A single dose of OsrhAAT 1 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg.
Placebo: Normal Saline (0.9% Sodium Chloride)
OsrhAAT 3 mg/kg IVDrug
Drug: OsrhAAT 3 mg/kg IV A single dose of OsrhAAT 3 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg.
Placebo: Normal Saline (0.9% Sodium Chloride)
OsrhAAT 10 mg/kg IVDrug
Drug: : OsrhAAT 10 mg/kg IV A single dose of OsrhAAT 10 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg.
Placebo: Normal Saline (0.9% Sodium Chloride)
OsrhAAT 20 mg/kg IVDrug
Drug: OsrhAAT 20 mg/kg IV A single dose of OsrhAAT 20 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg.
Placebo: Normal Saline (0.9% Sodium Chloride)
OsrhAAT 40 mg/kg IVDrug
Drug: OsrhAAT 40 mg/kg IV A single dose of OsrhAAT 40 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg.
Placebo: Normal Saline (0.9% Sodium Chloride)
OsrhAAT 60 mg/kg IVDrug
Drug: OsrhAAT 60 mg/kg IV A single dose of OsrhAAT 60 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg.
Placebo: Normal Saline (0.9% Sodium Chloride)
| Study group | Role | What they receive |
|---|---|---|
| OsrhAAT 1 mg/kg IV | Experimental | Not described Receives: Drug: OsrhAAT 1 mg/kg IV |
| OsrhAAT 3 mg/kg IV | Experimental | Not described Receives: Drug: OsrhAAT 3 mg/kg IV |
| OsrhAAT 10 mg/kg IV | Experimental | Not described Receives: Drug: OsrhAAT 10 mg/kg IV |
| OsrhAAT 20 mg/kg IV | Experimental | Not described Receives: Drug: OsrhAAT 20 mg/kg IV |
| OsrhAAT 40 mg/kg IV | Experimental | Not described Receives: Drug: OsrhAAT 40 mg/kg IV |
| OsrhAAT 60 mg/kg IV | Experimental | Not described Receives: Drug: OsrhAAT 60 mg/kg IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability: Incidence of adverse events (AEs), serious adverse events (SAEs), and infusion site reactions
Measured: Adverse events (AE) will be collected from the time of informed consent until EOS. SAE will be collected by the Investigator from the informed consent through 180 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2022
- Study start22 Mar 2022
- Primary completion31 Mar 2023 (estimated)
- Study completion30 Jun 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Emphysema Secondary to Congenital AATDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- This is a phase 1, single-center, placebo-controlled, double-blind, randomized study to evaluate the safety, tolerability, pharmacokinetics, and immunogenicity of OsrhAAT by a single ascending dose (SAD) design in healthy adults.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Healthgen Biotechnology Corp.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05315921
- Sponsor's protocol ID
- HY1003-2021-P1
- Status last verified
- Apr 2022
- First submitted
- 30 Mar 2022
- Last updated
- 12 Dec 2022
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Immunogenicity of Single Ascending Doses of OsrhAAT in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT05315921
- Acronym / sponsor ID
- HY1003-2021-P1
- Conditions
- Emphysema Secondary to Congenital AATD
- Keywords
- OsrhAAT
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Healthgen Biotechnology Corp.Industry (pharma, biotech, devices)
- Study start
- 22 Mar 2022 (actual)
- Primary completion
- 31 Mar 2023 (estimated)
- Study completion
- 30 Jun 2023 (estimated)
- First posted
- 7 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Immunogenicity of Single Ascending Doses of OsrhAAT in Healthy Volunteers
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 6
- Model description
- This is a phase 1, single-center, placebo-controlled, double-blind, randomized study to evaluate the safety, tolerability, pharmacokinetics, and immunogenicity of OsrhAAT by a single ascending dose (SAD) design in healthy adults.
| Arm | Type | Receives |
|---|---|---|
| OsrhAAT 1 mg/kg IV | Experimental | Drug: OsrhAAT 1 mg/kg IV |
| OsrhAAT 3 mg/kg IV | Experimental | Drug: OsrhAAT 3 mg/kg IV |
| OsrhAAT 10 mg/kg IV | Experimental | Drug: OsrhAAT 10 mg/kg IV |
| OsrhAAT 20 mg/kg IV | Experimental | Drug: OsrhAAT 20 mg/kg IV |
| OsrhAAT 40 mg/kg IV | Experimental | Drug: OsrhAAT 40 mg/kg IV |
| OsrhAAT 60 mg/kg IV | Experimental | Drug: OsrhAAT 60 mg/kg IV |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (20 listed)
- Able to understand and willing to sign the ICF
- Healthy subjects, male or female, non-smokers, 18-55 years of age
- No significant medical history, and in good health as determined by detailed medical history (neurological, endocrinal, cardiovascular, pulmonary, hematological, immunologic, psychiatric, gastrointestinal, renal, hepatic, and metabolic disease), full physical examination, vital signs, 12-lead electrocardiogram (ECG), urinalysis and laboratory tests at screening. For eligibility purposes, abnormal laboratory or vital signs results may be repeated once if abnormal result is observed at the initial reading. Moreover, abnormalities found in the ECG may need to be confirmed by repeated measurements.
- Adequate organ function according to the following laboratory values:
- Female of non-childbearing potential (i.e., physiologically incapable of becoming pregnant, including any female who is 1 year of post-menopausal with a FSH > 40mIU/mL, or surgically sterile [defined as having a bilateral oophorectomy, hysterectomy or tubal ligation]) or agree to one of the following to prevent pregnancy and, if a woman of childbearing potential, have a negative pregnancy test at screening:
Key exclusion criteria (19 listed)
- History of severe infection within 4 weeks prior to administration; signs and symptoms of any active infection regardless of severity within 2 weeks prior to administration.
- History of hypersensitivity to OsrhAAT or any excipient or similar drugs
- Known History of hypersensitivity to rice
- Use of any prescription drugs, herbal supplements, or nonprescription drugs, including oral antihistamines (for seasonal allergies), within 1 month or 5 half-lives (whichever is longer) prior to study drug administration, or dietary supplements within 1 week prior to study drug administration, unless, in the opinion of the Investigator and the Sponsor, the medication will not interfere with the study. Over-the-counter multivitamins will be permitted. If needed, paracetamol/acetaminophen may be used, but must be documented in the Concomitant medications/Significant non-drug therapies page of the source data. Any questions of concomitant medications should be directed to the Sponsor.
- Participation in a clinical research study involving the administration of an investigational or marketed drug or device within 30 days prior to the first dosing, administration of a biological product in the context of a clinical research study within 90 days prior to the first dosing, or concomitant participation in an investigational study involving no drug or device administration.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OsrhAAT 1 mg/kg IVDrug: OsrhAAT 1 mg/kg IV A single dose of OsrhAAT 1 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg. Placebo: Normal Saline (0.9% Sodium Chloride) | Drug | OsrhAAT 1 mg/kg IV |
| OsrhAAT 3 mg/kg IVDrug: OsrhAAT 3 mg/kg IV A single dose of OsrhAAT 3 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg. Placebo: Normal Saline (0.9% Sodium Chloride) | Drug | OsrhAAT 3 mg/kg IV |
| OsrhAAT 10 mg/kg IVDrug: : OsrhAAT 10 mg/kg IV A single dose of OsrhAAT 10 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg. Placebo: Normal Saline (0.9% Sodium Chloride) | Drug | OsrhAAT 10 mg/kg IV |
| OsrhAAT 20 mg/kg IVDrug: OsrhAAT 20 mg/kg IV A single dose of OsrhAAT 20 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg. Placebo: Normal Saline (0.9% Sodium Chloride) | Drug | OsrhAAT 20 mg/kg IV |
| OsrhAAT 40 mg/kg IVDrug: OsrhAAT 40 mg/kg IV A single dose of OsrhAAT 40 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg. Placebo: Normal Saline (0.9% Sodium Chloride) | Drug | OsrhAAT 40 mg/kg IV |
| OsrhAAT 60 mg/kg IVDrug: OsrhAAT 60 mg/kg IV A single dose of OsrhAAT 60 mg/kg IV infusion at a rate approximately 0.04 ml/min/kg. Placebo: Normal Saline (0.9% Sodium Chloride) | Drug | OsrhAAT 60 mg/kg IV |
Primary outcome
Safety and tolerability: Incidence of adverse events (AEs), serious adverse events (SAEs), and infusion site reactions
Time frame: Adverse events (AE) will be collected from the time of informed consent until EOS. SAE will be collected by the Investigator from the informed consent through 180 days.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2022.
- Individual participant dataSharing planned: No
Related trials for Emphysema Secondary to Congenital AATD
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OsrhAAT 1 mg/kg IV, OsrhAAT 3 mg/kg IV and OsrhAAT 10 mg/kg IV in people with Emphysema Secondary to Congenital AATD, run by Healthgen Biotechnology Corp. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yongjun David Kim, MD (714-252-0700 ext. 1482, YKim@altasciences.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability: Incidence of adverse events (AEs), serious adverse events (SAEs), and infusion site reactions (time frame: Adverse events (AE) will be collected from the time of informed consent until EOS. SAE will be collected by the Investigator from the informed consent through 180 days.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2022.