ThalassemiaPhase 1NCT03165864
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
A Phase 1 study testing IONIS TMPRSS6-Lrx in people with Thalassemia, run by Ionis Pharmaceuticals, Inc. at 1 site in Australia.
Trial summary
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Thalassemia
- What is being tested?
- IONIS TMPRSS6-Lrx
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 1 siteAustralia
- Study dates
- 9 May 2017to 25 Sep 2018
- 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 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Must have given written informed consent and be able to comply with all study requirements
- Healthy males or females aged 18-65 inclusive at the time of Informed Consent
- Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal
- Males must be surgically sterile, abstinent or using an acceptable contraceptive method
- BMI < 32 kg/m2
Who cannot join
- Clinically significant abnormalities in medical history or physical examination
- Clinically significant lab abnormalities that would render a subject unsuitable for inclusion
- Known history or positive test for HIV, HCV, or HBV
- Treatment with another Study Drug, biological agent, or device within one-month of Screening or 5 half-lives of investigational agent, whichever is longer
- Smoking > 10 cigarettes per day
- Regular excessive use of alcohol within 6 months of screening
- Current use of concomitant medications other than occasional acetaminophen (paracetamol) or ibuprofen unless approved by Sponsor Medical Monitor
- Considered unsuitable for inclusion by the Investigator or Sponsor
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers
- Conditions
- Thalassemia
- Being tested
- IONIS TMPRSS6-Lrx, Placebo
- Keywords
- Thalassaemia, IONIS TMPRSS6-Lrx
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IONIS TMPRSS6-LrxDrug
Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
PlaceboOther
Saline .9%
| Study group | Role | What they receive |
|---|---|---|
| IONIS TMPRSS6-Lrx | Experimental | Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously Receives: Drug: IONIS TMPRSS6-Lrx |
| Placebo | Placebo comparator | Saline .9% Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx
The safety and tolerability of single and multiple doses of IONIS TMPRSS6-Lrx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS TMPRSS6-Lrx
Measured: Up to 148 Days
Secondary outcomes (5)
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax)
The plasma pharmacokinetics (maximum observed drug concentration or Cmax) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Measured: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax)
The plasma pharmacokinetics (time taken to reach maximal concentration or Tmax) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Measured: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz)
The plasma pharmacokinetics (Plasma terminal elimination half-life (t1/2λz) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Measured: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the subcutaneous administration (AUCt)
The plasma pharmacokinetics (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the subcutaneous administration (AUCt) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Measured: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Percentage of the administered dose excreted in urine (% Dose Excreted)
The plasma pharmacokinetics (Percentage of the administered dose excreted in urine (% Dose Excreted) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Measured: Up to 148 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2017
- Study start9 May 2017
- Primary completion25 Sep 2018
- Study completion25 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ThalassemiaStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03165864
- Sponsor's protocol ID
- ISIS 702843-CS1
- Status last verified
- Jan 2019
- First submitted
- 18 May 2017
- Last updated
- 22 Jan 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ganz T, Nemeth E, Rivella S, Goldberg P, Dibble AR, McCaleb ML, Guo S, Monia BP, Barrett TD. TMPRSS6 as a Therapeutic Target for Disorders of Erythropoiesis and Iron Homeostasis. Adv Ther. 2023 Apr;40(4):1317-1333. doi: 10.1007/s12325-022-02421-w. Epub 2023 Jan 23.
PubMed 36690839
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
- ClinicalTrials.gov ID
- NCT03165864
- Acronym / sponsor ID
- ISIS 702843-CS1
- Conditions
- Thalassemia
- Keywords
- Thalassaemia, IONIS TMPRSS6-Lrx
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 9 May 2017 (actual)
- Primary completion
- 25 Sep 2018 (actual)
- Study completion
- 25 Sep 2018 (actual)
- First posted
- 24 May 2017 (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.
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IONIS TMPRSS6-Lrx | Experimental | Drug: IONIS TMPRSS6-LrxAscending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously |
| Placebo | Placebo comparator | Other: PlaceboSaline .9% |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Must have given written informed consent and be able to comply with all study requirements
- Healthy males or females aged 18-65 inclusive at the time of Informed Consent
- Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal
- Males must be surgically sterile, abstinent or using an acceptable contraceptive method
- BMI < 32 kg/m2
Key exclusion criteria (8 listed)
- Clinically significant abnormalities in medical history or physical examination
- Clinically significant lab abnormalities that would render a subject unsuitable for inclusion
- Known history or positive test for HIV, HCV, or HBV
- Treatment with another Study Drug, biological agent, or device within one-month of Screening or 5 half-lives of investigational agent, whichever is longer
- Smoking > 10 cigarettes per day
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 |
|---|---|---|
| IONIS TMPRSS6-LrxAscending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously | Drug | IONIS TMPRSS6-Lrx |
| PlaceboSaline .9% | Other | Placebo |
Primary outcome
Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx
Time frame: Up to 148 Days
The safety and tolerability of single and multiple doses of IONIS TMPRSS6-Lrx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS TMPRSS6-Lrx
5 secondary outcomes
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax)
Time frame: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax)
Time frame: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz)
Time frame: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the subcutaneous administration (AUCt)
Time frame: Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Percentage of the administered dose excreted in urine (% Dose Excreted)
Time frame: Up to 148 Days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2019.
- Derived from this study: PubMed 36690839Ganz T, Nemeth E, Rivella S, Goldberg P, Dibble AR, McCaleb ML, Guo S, Monia BP, Barrett TD. TMPRSS6 as a Therapeutic Target for Disorders of Erythropoiesis and Iron Homeostasis. Adv Ther. 2023 Apr;40(4):1317-1333. doi: 10.1007/s12325-022-02421-w. Epub 2023 Jan 23.
Related trials for Thalassemia
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
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.
The record lists no contact. Use the official record to reach the sponsor.
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 IONIS TMPRSS6-Lrx in people with Thalassemia, run by Ionis Pharmaceuticals, Inc. at 1 site in Australia.
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 5 inclusion and 8 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: finished, as of the sponsor's last update on 22 Jan 2019. 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 Australia. 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. Some participants in this study may receive a placebo. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx (time frame: Up to 148 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03165864, which also lists 1 linked publication.
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 22 Jan 2019.