Acute Intermittent PorphyriaPhase 1NCT02082860
Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria
A Phase 1 study testing rAAV2/5-PBGD vector dosage 1, rAAV2/5-PBGD vector dosage 2 and rAAV2/5-PBGD vector dosage 3 in people with Acute Intermittent Porphyria, run by Digna Biotech S.L. at 2 sites in Spain.
Trial summary
This is a Phase I trial aimed to determine the safety of the investigational gene therapy product (rAAV2/5-PBGD) for the treatment of Acute Intermittent Porphyria (AIP). Up to eight patients fulfilling the eligibility criteria will participate in this multicentre, open label, single dose, dose-ranging Phase I clinical… Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Acute Intermittent Porphyria
- What is being tested?
- rAAV2/5-PBGD vector dosage 1, rAAV2/5-PBGD vector dosage 2 and rAAV2/5-PBGD vector dosage 3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 2 sitesSpain
- Study dates
- Nov 2012to Nov 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 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patient's written Informed Consent
- Age between 18 and 64 years, inclusively.
- Patients with confirmed diagnosis of Acute Intermittent Porphyria(AIP), as confirmed by clinical, biochemical data and genetic confirmation of porphobilinogen deaminase (PBGD) gene mutation. The patient must have a severe AIP condition, with at least two hospitalizations during the previous year due to acute attacks (clinical manifestations of acute porphyria), or at least four hospitalizations during the previous year due to the requirement of hospital treatment administration (including day-hospital and home hospital program)
- Previous participation in the "Observational study of acute intermittent porphyria patients" for at least six months.
- Ability to follow instructions and cooperate during the study conduct
Who cannot join
- Pregnant women, as confirmed by a positive urine pregnancy test, or with intention of becoming pregnant
- Female subjects of childbearing potential who are not using barrier methods of contraception, at least during the study.
- Male subjects with partners of child bearing potential who are not using barrier contraceptive methods, at least during the study
- Acute or chronic liver disease of viral, autoimmune or metabolic causes
- History of acute or chronic severe gastrointestinal dysfunction (different than those typical gastrointestinal symptoms associated with an acute attack of AIP), in the opinion of the principal investigator
- Kidney disorder (renal impairment defined as plasma creatinine > 2 mg/dl (150 µmol/l)), severe respiratory disease, severe autoimmune disease or severe acute active infection
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- Evidence of active Hepatitis B virus (HBV) or Hepatitis C virus (HCV) infection as reflected by HBs antigen or HCV-antibodies positivity (in case of HCV-antibodies positivity a HCV-RNA test should be performed in order to confirm active viral replication)
- Positive human immunodeficiency virus (HIV) serological test
- History of drug use (cannabis, cocaine, amphetamines, barbiturates) or alcohol abuse or addiction, during the three months preceding the selection visit
- Presence of neutralizing antibodies against adeno-associated serotype 5 (AAV5)
- Current or previous (within the previous 12 months) participation in a gene therapy trial.
- Previous participation (at any time) in a gene therapy trial using AAV vectors
- Any other disease or condition that, in the opinion of the principal investigator, contraindicates the participation in the study because it can expose the patient to a risk or because it disqualifies the patient to complete the schedule of the study.
Trial locations and contacts
2 sites in 1 country.
Spain2 sites
12 Octubre Hospital
Madrid, Madrid, 28041
Clinica Universidad de Navarra
Pamplona, Navarre, 31008
About this study
Official title: Phase I, Multicentre, Open Label, Single Dose, Dose-ranging Clinical Trial to Investigate the Safety and Tolerability of a Gene Therapy rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria
This is a Phase I trial aimed to determine the safety of the investigational gene therapy product (rAAV2/5-PBGD) for the treatment of Acute Intermittent Porphyria (AIP).
Up to eight patients fulfilling the eligibility criteria will participate in this multicentre, open label, single dose, dose-ranging Phase I clinical trial.
Read more
The enrolled patients will be followed up to assess the safety profile of the investigational gene therapy product and to establish the maximum therapeutic safe dose to be administered in future confirmatory/pivotal clinical trial(s). In addition, the biological and clinical response to the treatment with rAAV2/5-PBGD in AIP patients will be assessed.
A complete evaluation of the clinical (symptoms and quality of life assessment) and laboratory (blood and urine) data will be performed.
Read the detailed description
Acute Intermittent Porphyria (AIP) is inherited as an autosomal dominant disorder of the heme biosynthesis pathway. AIP is caused by a genetic defect in porphobilinogen deaminase (PBGD), a key enzyme for heme synthesis.
AIP is characterized by acute episodes and asymptomatic periods. Neuropathic symptoms are predominantly in these attacks, which may be related to the toxic effect produced by the precursors delta-aminolevulinic acid (ALA) and porphobilinogen (PBG), accumulated because the enzyme deficiency. It occurs with very low prevalence (1 in 50,000), but figures for prevalence based on clinical manifestations (i.e., acute attacks) greatly underestimate the number of patients with latent AIP.
Abdominal pain is the most common symptom, sometimes with constipation. Paresthesia and paralysis also occur, and death may result from respiratory paralysis. Other symptoms, including seizures, psychotic episodes, and hypertension, develop during acute attacks. They may be precipitated by porphyrogenic drugs such as barbiturates, progestogens and sulfonamides, some of which are known to induce the first rate-controlling step in heme synthesis, ALA synthesis. Other known precipitants are alcohol, infection, starvation, and hormonal changes; attacks are more common in women. Acute attacks rarely occur before puberty.
This is a Phase I clinical trial mainly aimed to evaluate the safety of a recombinant adeno associated vector with a liver-specific promoter for the PBGD expression (rAAV2/5-PBGD), for the treatment of Acute Intermittent Porphyria.
The patients will be enrolled in an adaptive dose-escalation, multicentre trial to assess safety profile, and to establish the maximum therapeutic safe dose to be administrated to patients in further confirmatory or pivotal clinical trial.
This clinical trial is preceded by an "Observational study of acute intermittent porphyria patients" (DIG-API-2011-01). In this observational study, severe AIP patients have been followed for 6 to up to a maximum 24 months. During this time, the clinical and laboratory (blood and urine biochemistry) conditions of the patients were evaluated, in order establish clinical and biological baseline and history to compare the future results of this clinical trial.
During this clinical trial, the safety will be evaluated by the Adverse Events (AEs) and Serious Adverse Events (SAEs) assessment. A complete evaluation of the clinical and laboratory (blood and urine) data will be collected. The study will also investigate as secondary endpoints the effect of this treatment to modify other aspects of the patient condition.
Due to the heterogeneity of genetic mutations and inter-individual variation, clinical symptomatology and ALA/PBG levels in AIP subjects showed an evident variability in urine samples both during acute attacks and during remission; each subject will be its own control, so this study will be an intra-individually controlled clinical trial. At the end of the clinical trial the efficacy evaluation will be performed based on the clinical and biochemical changes compared to the baseline established in the previous observational study.
- Conditions
- Acute Intermittent Porphyria
- Being tested
- rAAV2/5-PBGD vector dosage 1, rAAV2/5-PBGD vector dosage 2, rAAV2/5-PBGD vector dosage 3, rAAV2/5-PBGD vector dosage 4
- Keywords
- Porphyria, Gene therapy, AAV, Liver gene transfer
- Medical subject headings
- Porphyria, Acute Intermittent, Porphyrias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rAAV2/5-PBGD vector dosage 1Genetic
Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.
rAAV2/5-PBGD vector dosage 2Genetic
Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.
rAAV2/5-PBGD vector dosage 3Genetic
Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.
rAAV2/5-PBGD vector dosage 4Genetic
Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Experimental | rAAV2/5-PBGD vector dosage 1 Receives: Genetic: rAAV2/5-PBGD vector dosage 1 |
| Cohort B | Experimental | rAAV2/5-PBGD vector dosage 2 Receives: Genetic: rAAV2/5-PBGD vector dosage 2 |
| Cohort C | Experimental | rAAV2/5-PBGD vector dosage 3 Receives: Genetic: rAAV2/5-PBGD vector dosage 3 |
| Cohort D | Experimental | rAAV2/5-PBGD vector dosage 4 Receives: Genetic: rAAV2/5-PBGD vector dosage 4 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with Adverse Events and Serious Adverse Events
To assess the safety and determine the maximum therapeutic safe dose of the investigational gene therapy (GT) product rAAV2/5-PBGD for the treatment of AIP, registering and evaluating the occurrence of Adverse Events and/or Serious Adverse Events at the dose identified will be performed
Measured: Up to 48 weeks
Secondary outcomes (7)
Effect of the treatment on porphobilinogen (PBG) and delta-aminolevulinic acid (ALA) urinary level.
Measured: Up to 48 weeks
Clinical evolution of acute intermittent porphyria. Frequency of hospitalizations
Measured: Up to 48 weeks
Frequency of treatments for AIP symptoms
The information regarding the requirement of specific treatments for symptoms control (analgesics, hemin and glucose endovenous solutions) will be collected.
Measured: Up to 48 weeks
Psychological evaluation of AIP patients
Anxiety and depression will be assessed by using the Beck Anxiety Inventory (BAI) and Beck Depression Inventory (BDI) rating scales.
Measured: Up to 48 weeks
Health related quality of life of AIP patients
Health-related quality of life will be assessed through the SF-36v2 questionnaire
Measured: Up to 48 weeks
Frequency of AIP symptoms
Measured: Up to 48 weeks
Pharmacokinetic parameters
Measured: Selection visit, Days 1, 2 and 3, week 1, week 2, week 3 and week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2014 (estimated)
- Study startNov 2012
- Primary completionJul 2014
- Study completionNov 2014
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 Acute Intermittent PorphyriaStudies 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Digna Biotech S.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Porphyria Centre Sweden, University of Navarra, UniQure N.V., Nationales Centrum für Tumorerkrankungen
- Responsible party
- Sponsor
- Investigators
- Juan Ruiz, MD, Study chair, Digna Biotech S.L.Jesus Prieto, MD, Principal investigator, Clinica Universidad de NavarraRafael Enriquez de Salamanca, MD, Principal investigator, Hospital 12 de Octubre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02082860
- Sponsor's protocol ID
- AAVPBGD-AIP-001
- Other IDs
- 2011-005590-23
- Status last verified
- Dec 2014
- First submitted
- 28 Feb 2014
- Last updated
- 18 Dec 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
D'Avola D, Lopez-Franco E, Sangro B, Paneda A, Grossios N, Gil-Farina I, Benito A, Twisk J, Paz M, Ruiz J, Schmidt M, Petry H, Harper P, de Salamanca RE, Fontanellas A, Prieto J, Gonzalez-Aseguinolaza G. Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria. J Hepatol. 2016 Oct;65(4):776-783. doi: 10.1016/j.jhep.2016.05.012. Epub 2016 May 17.
PubMed 27212246
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I, Multicentre, Open Label, Single Dose, Dose-ranging Clinical Trial to Investigate the Safety and Tolerability of a Gene Therapy rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria
- ClinicalTrials.gov ID
- NCT02082860
- Acronym / sponsor ID
- AAVPBGD-AIP-001
- Conditions
- Acute Intermittent Porphyria
- Keywords
- Porphyria, Gene therapy, AAV, Liver gene transfer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Digna Biotech S.L.Industry (pharma, biotech, devices)
- Collaborators
- Porphyria Centre Sweden, University of Navarra, UniQure N.V., Nationales Centrum für Tumorerkrankungen
- Study start
- Nov 2012
- Primary completion
- Jul 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 10 Mar 2014 (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.
This is a Phase I trial aimed to determine the safety of the investigational gene therapy product (rAAV2/5-PBGD) for the treatment of Acute Intermittent Porphyria (AIP).
Up to eight patients fulfilling the eligibility criteria will participate in this multicentre, open label, single dose, dose-ranging Phase I clinical trial.
The enrolled patients will be followed up to assess the safety profile of the investigational gene therapy product and to establish the maximum therapeutic safe dose to be administered in future confirmatory/pivotal clinical trial(s). In addition, the biological and clinical response to the treatment with rAAV2/5-PBGD in AIP patients will be assessed.
A complete evaluation of the clinical (symptoms and quality of life assessment) and laboratory (blood and urine) data will be performed.
Detailed description
Acute Intermittent Porphyria (AIP) is inherited as an autosomal dominant disorder of the heme biosynthesis pathway. AIP is caused by a genetic defect in porphobilinogen deaminase (PBGD), a key enzyme for heme synthesis.
AIP is characterized by acute episodes and asymptomatic periods. Neuropathic symptoms are predominantly in these attacks, which may be related to the toxic effect produced by the precursors delta-aminolevulinic acid (ALA) and porphobilinogen (PBG), accumulated because the enzyme deficiency. It occurs with very low prevalence (1 in 50,000), but figures for prevalence based on clinical manifestations (i.e., acute attacks) greatly underestimate the number of patients with latent AIP.
Abdominal pain is the most common symptom, sometimes with constipation. Paresthesia and paralysis also occur, and death may result from respiratory paralysis. Other symptoms, including seizures, psychotic episodes, and hypertension, develop during acute attacks. They may be precipitated by porphyrogenic drugs such as barbiturates, progestogens and sulfonamides, some of which are known to induce the first rate-controlling step in heme synthesis, ALA synthesis. Other known precipitants are alcohol, infection, starvation, and hormonal changes; attacks are more common in women. Acute attacks rarely occur before puberty.
This is a Phase I clinical trial mainly aimed to evaluate the safety of a recombinant adeno associated vector with a liver-specific promoter for the PBGD expression (rAAV2/5-PBGD), for the treatment of Acute Intermittent Porphyria.
The patients will be enrolled in an adaptive dose-escalation, multicentre trial to assess safety profile, and to establish the maximum therapeutic safe dose to be administrated to patients in further confirmatory or pivotal clinical trial.
This clinical trial is preceded by an "Observational study of acute intermittent porphyria patients" (DIG-API-2011-01). In this observational study, severe AIP patients have been followed for 6 to up to a maximum 24 months. During this time, the clinical and laboratory (blood and urine biochemistry) conditions of the patients were evaluated, in order establish clinical and biological baseline and history to compare the future results of this clinical trial.
During this clinical trial, the safety will be evaluated by the Adverse Events (AEs) and Serious Adverse Events (SAEs) assessment. A complete evaluation of the clinical and laboratory (blood and urine) data will be collected. The study will also investigate as secondary endpoints the effect of this treatment to modify other aspects of the patient condition.
Due to the heterogeneity of genetic mutations and inter-individual variation, clinical symptomatology and ALA/PBG levels in AIP subjects showed an evident variability in urine samples both during acute attacks and during remission; each subject will be its own control, so this study will be an intra-individually controlled clinical trial. At the end of the clinical trial the efficacy evaluation will be performed based on the clinical and biochemical changes compared to the baseline established in the previous observational study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort A | Experimental | Genetic: rAAV2/5-PBGD vector dosage 1rAAV2/5-PBGD vector dosage 1 |
| Cohort B | Experimental | Genetic: rAAV2/5-PBGD vector dosage 2rAAV2/5-PBGD vector dosage 2 |
| Cohort C | Experimental | Genetic: rAAV2/5-PBGD vector dosage 3rAAV2/5-PBGD vector dosage 3 |
| Cohort D | Experimental | Genetic: rAAV2/5-PBGD vector dosage 4rAAV2/5-PBGD vector dosage 4 |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Patient's written Informed Consent
- Age between 18 and 64 years, inclusively.
- Patients with confirmed diagnosis of Acute Intermittent Porphyria(AIP), as confirmed by clinical, biochemical data and genetic confirmation of porphobilinogen deaminase (PBGD) gene mutation. The patient must have a severe AIP condition, with at least two hospitalizations during the previous year due to acute attacks (clinical manifestations of acute porphyria), or at least four hospitalizations during the previous year due to the requirement of hospital treatment administration (including day-hospital and home hospital program)
- Previous participation in the "Observational study of acute intermittent porphyria patients" for at least six months.
- Ability to follow instructions and cooperate during the study conduct
Key exclusion criteria (13 listed)
- Pregnant women, as confirmed by a positive urine pregnancy test, or with intention of becoming pregnant
- Female subjects of childbearing potential who are not using barrier methods of contraception, at least during the study.
- Male subjects with partners of child bearing potential who are not using barrier contraceptive methods, at least during the study
- Acute or chronic liver disease of viral, autoimmune or metabolic causes
- History of acute or chronic severe gastrointestinal dysfunction (different than those typical gastrointestinal symptoms associated with an acute attack of AIP), in the opinion of the principal investigator
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 |
|---|---|---|
| rAAV2/5-PBGD vector dosage 1Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. | Genetic | Cohort A |
| rAAV2/5-PBGD vector dosage 2Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. | Genetic | Cohort B |
| rAAV2/5-PBGD vector dosage 3Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. | Genetic | Cohort C |
| rAAV2/5-PBGD vector dosage 4Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. | Genetic | Cohort D |
Primary outcome
Number of patients with Adverse Events and Serious Adverse Events
Time frame: Up to 48 weeks
To assess the safety and determine the maximum therapeutic safe dose of the investigational gene therapy (GT) product rAAV2/5-PBGD for the treatment of AIP, registering and evaluating the occurrence of Adverse Events and/or Serious Adverse Events at the dose identified will be performed
7 secondary outcomes
Effect of the treatment on porphobilinogen (PBG) and delta-aminolevulinic acid (ALA) urinary level.
Time frame: Up to 48 weeks
Clinical evolution of acute intermittent porphyria. Frequency of hospitalizations
Time frame: Up to 48 weeks
Frequency of treatments for AIP symptoms
Time frame: Up to 48 weeks
Psychological evaluation of AIP patients
Time frame: Up to 48 weeks
Health related quality of life of AIP patients
Time frame: Up to 48 weeks
Frequency of AIP symptoms
Time frame: Up to 48 weeks
Pharmacokinetic parameters
Time frame: Selection visit, Days 1, 2 and 3, week 1, week 2, week 3 and week 4
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 18 Dec 2014.
- Derived from this study: PubMed 27212246D'Avola D, Lopez-Franco E, Sangro B, Paneda A, Grossios N, Gil-Farina I, Benito A, Twisk J, Paz M, Ruiz J, Schmidt M, Petry H, Harper P, de Salamanca RE, Fontanellas A, Prieto J, Gonzalez-Aseguinolaza G. Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria. J Hepatol. 2016 Oct;65(4):776-783. doi: 10.1016/j.jhep.2016.05.012. Epub 2016 May 17.
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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.
Juan Ruiz, MD study chair
Digna Biotech S.L.
Jesus Prieto, MD principal investigator
Clinica Universidad de Navarra
Rafael Enriquez de Salamanca, MD principal investigator
Hospital 12 de Octubre
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing rAAV2/5-PBGD vector dosage 1, rAAV2/5-PBGD vector dosage 2 and rAAV2/5-PBGD vector dosage 3 in people with Acute Intermittent Porphyria, run by Digna Biotech S.L. at 2 sites in Spain.
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 13 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 18 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Spain. 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 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?
Number of patients with Adverse Events and Serious Adverse Events (time frame: Up to 48 weeks). 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 NCT02082860, 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 18 Dec 2014.