Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 1NCT01335165
Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 1 study testing ALXN1102 and ALXN1103 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Alexion Pharmaceuticals, Inc. at 5 sites in 5 countries.
Trial summary
The purpose of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients with PNH. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- ALXN1102 and ALXN1103
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 5 sitesin 5 countries
- Study dates
- Apr 2011to Mar 2014
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Individuals at least 18 years of age with a diagnosis of PNH \& vaccination against meningococcus.
Who cannot join
- Abnormal renal or liver function
- History of meningococcal disease
- History of Guillain-Barre syndrome
- Known infection with HIV or Hepatitis B or C
- History of thrombotic events
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
United States1 site
USC/Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Czechia1 site
Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze
U Nemocnice 1, Prague 2, 128 08
United Kingdom1 site
King's College Hospital NHS Foundation Trust
Denmark Hill, London, SE5 9RS UK
About this study
Official title: A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients with PNH.
Read the detailed description
This is an open-label, single-dose, dose-escalation study to assess the safety, tolerability, PK, PD, and immunogenicity of TT30 given as an IV infusion and as a SC injection in subjects with PNH or evidence of circulating PNH cells.
Eligible subjects with PNH will be vaccinated with meningococcal vaccine at least two weeks prior to dosing (if not previously vaccinated or if revaccination is required).
On study day 1, the subject will receive the single dose of TT30 as an IV infusion over 60 minutes or as a SC injection, and be followed with additional study evaluations over the next 59 days.
- Being tested
- ALXN1102, ALXN1103
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN1102Drug
Single Ascending Dose IV
ALXN1103Drug
Single Ascending Dose IV or SC
| Study group | Role | What they receive |
|---|---|---|
| TT30 (ALXN1102 Formulation) | Experimental | IV: 0.1, 0.3, and 1.0 mg/kg Receives: Drug: ALXN1102 |
| TT30 (ALXN1103 Formulation) | Experimental | IV: 3.0, 6.0, and 10.0 mg/kg
SC: 1.0 and 3.0 mg/kg Receives: Drug: ALXN1103 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the safety and tolerability of a single dose of TT30.
Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.
Measured: 60 days
Secondary outcomes (1)
Characterize PK, PD and immunogenicity of a single dose of TT30.
Immunogenicity will be will be assessed using standard measures for these parameters. PK and PD will be assessed by using standard measures, including reticulocyte count and lactate dehydrogenase (LDH) levels.
Measured: 60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2011 (estimated)
- Study startApr 2011
- Primary completionMar 2014
- Study completionMar 2014
Why it stopped: Inability to reach enrollment as planned
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
- More trials run by Alexion 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bill Lundberg, MD, Study director, Alexion Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01335165
- Sponsor's protocol ID
- TT30-PNH-002
- Status last verified
- May 2014
- First submitted
- 12 Apr 2011
- Last updated
- 22 May 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT01335165
- Acronym / sponsor ID
- TT30-PNH-002
- Keywords
- PNH
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Inability to reach enrollment as planned
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2011
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 14 Apr 2011 (estimated)
- 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 of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients with PNH.
Detailed description
This is an open-label, single-dose, dose-escalation study to assess the safety, tolerability, PK, PD, and immunogenicity of TT30 given as an IV infusion and as a SC injection in subjects with PNH or evidence of circulating PNH cells.
Eligible subjects with PNH will be vaccinated with meningococcal vaccine at least two weeks prior to dosing (if not previously vaccinated or if revaccination is required).
On study day 1, the subject will receive the single dose of TT30 as an IV infusion over 60 minutes or as a SC injection, and be followed with additional study evaluations over the next 59 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| TT30 (ALXN1102 Formulation) | Experimental | Drug: ALXN1102IV: 0.1, 0.3, and 1.0 mg/kg |
| TT30 (ALXN1103 Formulation) | Experimental | Drug: ALXN1103IV: 3.0, 6.0, and 10.0 mg/kg SC: 1.0 and 3.0 mg/kg |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Individuals at least 18 years of age with a diagnosis of PNH \& vaccination against meningococcus.
Key exclusion criteria (5 listed)
- Abnormal renal or liver function
- History of meningococcal disease
- History of Guillain-Barre syndrome
- Known infection with HIV or Hepatitis B or C
- History of thrombotic events
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 |
|---|---|---|
| ALXN1102Single Ascending Dose IV | Drug | TT30 (ALXN1102 Formulation) |
| ALXN1103Single Ascending Dose IV or SC | Drug | TT30 (ALXN1103 Formulation) |
Primary outcome
Assess the safety and tolerability of a single dose of TT30.
Time frame: 60 days
Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.
1 secondary outcome
Characterize PK, PD and immunogenicity of a single dose of TT30.
Time frame: 60 days
Results and safety
The study was stopped early (reason given: Inability to reach enrollment as planned). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2014.
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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.
Bill Lundberg, MD study director
Alexion Pharmaceuticals, Inc.
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALXN1102 and ALXN1103 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Alexion Pharmaceuticals, Inc. at 5 sites in 5 countries.
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 1 inclusion and 5 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: stopped early, as of the sponsor's last update on 22 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Czechia, Italy, Poland, United Kingdom. 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?
The study was stopped early (reason given: Inability to reach enrollment as planned). Results are not available in the source record.
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?
Assess the safety and tolerability of a single dose of TT30. (time frame: 60 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 22 May 2014.