Acute PorphyriasPhase 2NCT02180412
Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria
A Phase 2 study testing Panhematin and Glucose in people with Acute Porphyrias, run by The University of Texas Medical Branch, Galveston at 1 site in United States.
Trial summary
This study aims to provide high quality evidence for the effectiveness and safety of hemin (PanhematinTM , Recordati) for treatment of acute attacks of porphyria. These types of studies have not been done before with either PanhematinTM or the hemin preparation available in Europe (NormosangTM, Orphan Europe). Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Acute Porphyrias
- What is being tested?
- Panhematin and Glucose
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Apr 2014to 3 Feb 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female aged 18 years
- Willing to provide written informed consent
- Acute symptoms (7 days duration or less to time of enrollment) such as abdominal, back and/or limb pain, diagnosed by the investigator as caused by porphyria after initial evaluation has excluded other causes.
- Diagnosis of acute porphyria documented by a substantial increase in urinary or serum porphobilinogen (PBG).
- Type of acute porphyria confirmed by additional testing (in addition to increased PBG), which may be completed before or after treatment begins using pretreatment samples:
- For acute intermittent porphyria (AIP): Normal or only slight increases in plasma and fecal porphyrins. Most (~90 percent) will have deficient activity of erythrocyte porphobilinogen deaminase (PBGD), and almost all (>95 percent) will have a demonstrable disease-causing PBGD mutation.
- For hereditary coproporphyria (HCP): Substantial increases in fecal porphyrins (almost entirely coproporphyrin III). In the absence of skin photosensitivity, most will have normal or only slight increases in plasma porphyrins. Almost all (>95 percent) will have a demonstrable disease-causing coproporphyrinogen oxidase (CPO) mutation.
- For variegate porphyria (VP): Substantial increases in fecal porphyrins (mostly coproporphyrin III and protoporphyrin), increased plasma total porphyrins and a fluorescence emission maximum of diluted plasma at neutral pH near 626 nm. Almost all (~95 percent) will have a demonstrable disease-causing protoporphyrinogen oxidase (PPO) mutation.
Who cannot join
- Symptoms such as abdominal, back or limb pain are explained by another condition, as judged by the investigator
- Therapy with hemin within 7 days prior to enrollment in this study
- Known or suspected allergy to Panhematin™ or related products
- Preexisting coagulation defect or concurrent treatment with an anticoagulant
- Previously documented renal impairment defined as a serum creatinine above 1.7 mg/dL or 150 mmol/L.
- A diagnosis of diabetes mellitus, which might increase the risk of glucose infusion.
- Heart failure, significant chronic anemia or any disease or condition that the investigator judges would lead to an unacceptable risk to the patient or interfere with the successful collection of date for the trial
- Previous randomization in this trial
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas Medical Branch
Galveston, Texas, 77555
About this study
Official title: A Double-blind, Randomized, Placebo-controlled, Parallel Group Trial on the Efficacy and Safety of PanhematinTM in the Treatment of Acute Attacks of Porphyria
This study aims to provide high quality evidence for the effectiveness and safety of hemin (PanhematinTM , Recordati) for treatment of acute attacks of porphyria. These types of studies have not been done before with either PanhematinTM or the hemin preparation available in Europe (NormosangTM, Orphan Europe).
There are two treatment groups in this study. One group will be treated with PanhematinTM plus glucose, and the other group will be treated with glucose plus an inactive salt solution (called a "placebo"). To avoid prejudice, the treatment given to each participant will be blinded (meaning the participants and most of the hospital staff will not know which treatment the participant will receive) and randomized (meaning participants will have an equal chance of receiving either treatment, like the flip of a coin). A placebo-controlled, randomized study is the standard method used to prove treatments are effective and safe. PanhematinTM and glucose will be given in the same manner as is usual for treating an attack of porphyria. For participants who are chosen to receive the placebo, their treatment will be switched to real PanhematinTM at any time if their symptoms do not improve. This is called "rescue" treatment, and assures that they study is safe and patients who need hemin will receive it. Treatment with hemin will be for 4 days, or longer if needed. Since the study treatment is started as soon as possible after symptoms appear, there will be very little delay in providing hemin to those who need it. Funding Source - Office of Orphan Products Development (FDA OOPD)
- Conditions
- Acute Porphyrias
- Being tested
- Panhematin, Glucose
- Keywords
- Acute porphyria, Hemin
- Medical subject headings
- Porphyria, Acute Intermittent
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PanhematinBiological
Also called Glucose
Glucose loading
GlucoseOther
Glucose is administered to both groups as routine care.
| Study group | Role | What they receive |
|---|---|---|
| Panhematin | Experimental | Panhematin plus glucose Receives: Biological: Panhematin; Other: Glucose |
| Placebo | Placebo comparator | Placebo (saline) plus glucose Receives: Other: Glucose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference in NRS Pain Score Between Baseline and 12 Hours
The difference in the pre-infusion NRS pain scores and NRS pain score 12 hours from the infusion start time. To define this variable, all blinded study treatment infusion times were compared with the pain score survey times. The pain score closest to but prior to the infusion time was the pre-infusion pain score. Numeric rating scale for pain (0-10; 0=no pain, 10=most severe pain).
Measured: Baseline and 12 hours
Secondary outcomes (1)
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria
Difference in the panhematin and placebo arm for the change in urinary ALA, (PBG), and total porphyrins between baseline and subsequent time points.
Measured: Day 1 (baseline), Day 2, Day 3 and Day 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2014 (estimated)
- Study start28 Apr 2014
- Primary completion3 Feb 2022
- Study completion3 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute PorphyriasStudies of the same condition, open and under way.
- More trials run by The University of Texas Medical Branch, GalvestonThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Karl E Anderson, MD, Principal investigator, UT, Galveston
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02180412
- Sponsor's protocol ID
- 10-203
- Other IDs
- FD-R-03720
- Status last verified
- Apr 2025
- First submitted
- 25 Jun 2014
- Last updated
- 11 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Storjord E, Anderson KE, Bonkovsky HL, Murti A, Tollali G, Moghe A, Wang B, Emblem A, Brekke OL, Karlsen BO. C-type lectin 10A [CD301] mRNA is upregulated in symptomatic acute intermittent porphyria and decreases after heme treatment. Mol Genet Metab. 2026 Sep 18;149(3):110263. doi: 10.1016/j.ymgme.2026.110263. Online ahead of print.
PubMed 42833135
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Randomized, Placebo-controlled, Parallel Group Trial on the Efficacy and Safety of PanhematinTM in the Treatment of Acute Attacks of Porphyria
- ClinicalTrials.gov ID
- NCT02180412
- Acronym / sponsor ID
- 10-203
- Conditions
- Acute Porphyrias
- Keywords
- Acute porphyria, Hemin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- The University of Texas Medical Branch, GalvestonUniversity, hospital or other
- Study start
- 28 Apr 2014 (actual)
- Primary completion
- 3 Feb 2022 (actual)
- Study completion
- 3 Feb 2022 (actual)
- First posted
- 2 Jul 2014 (estimated)
- Results
- Posted 3 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aims to provide high quality evidence for the effectiveness and safety of hemin (PanhematinTM , Recordati) for treatment of acute attacks of porphyria. These types of studies have not been done before with either PanhematinTM or the hemin preparation available in Europe (NormosangTM, Orphan Europe).
There are two treatment groups in this study. One group will be treated with PanhematinTM plus glucose, and the other group will be treated with glucose plus an inactive salt solution (called a "placebo"). To avoid prejudice, the treatment given to each participant will be blinded (meaning the participants and most of the hospital staff will not know which treatment the participant will receive) and randomized (meaning participants will have an equal chance of receiving either treatment, like the flip of a coin). A placebo-controlled, randomized study is the standard method used to prove treatments are effective and safe. PanhematinTM and glucose will be given in the same manner as is usual for treating an attack of porphyria. For participants who are chosen to receive the placebo, their treatment will be switched to real PanhematinTM at any time if their symptoms do not improve. This is called "rescue" treatment, and assures that they study is safe and patients who need hemin will receive it. Treatment with hemin will be for 4 days, or longer if needed. Since the study treatment is started as soon as possible after symptoms appear, there will be very little delay in providing hemin to those who need it. Funding Source - Office of Orphan Products Development (FDA OOPD)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Panhematin | Experimental | Biological: Panhematin, Other: GlucosePanhematin plus glucose |
| Placebo | Placebo comparator | Other: GlucosePlacebo (saline) plus glucose |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female aged 18 years
- Willing to provide written informed consent
- Acute symptoms (7 days duration or less to time of enrollment) such as abdominal, back and/or limb pain, diagnosed by the investigator as caused by porphyria after initial evaluation has excluded other causes.
- Diagnosis of acute porphyria documented by a substantial increase in urinary or serum porphobilinogen (PBG).
- Type of acute porphyria confirmed by additional testing (in addition to increased PBG), which may be completed before or after treatment begins using pretreatment samples:
Key exclusion criteria (8 listed)
- Symptoms such as abdominal, back or limb pain are explained by another condition, as judged by the investigator
- Therapy with hemin within 7 days prior to enrollment in this study
- Known or suspected allergy to Panhematin™ or related products
- Preexisting coagulation defect or concurrent treatment with an anticoagulant
- Previously documented renal impairment defined as a serum creatinine above 1.7 mg/dL or 150 mmol/L.
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 |
|---|---|---|
| PanhematinAlso: GlucoseGlucose loading | Biological | Panhematin |
| GlucoseGlucose is administered to both groups as routine care. | Other | Panhematin, Placebo |
Primary outcome
Difference in NRS Pain Score Between Baseline and 12 Hours
Time frame: Baseline and 12 hours
The difference in the pre-infusion NRS pain scores and NRS pain score 12 hours from the infusion start time. To define this variable, all blinded study treatment infusion times were compared with the pain score survey times. The pain score closest to but prior to the infusion time was the pre-infusion pain score. Numeric rating scale for pain (0-10; 0=no pain, 10=most severe pain).
1 secondary outcome
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria
Time frame: Day 1 (baseline), Day 2, Day 3 and Day 4
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42833135Storjord E, Anderson KE, Bonkovsky HL, Murti A, Tollali G, Moghe A, Wang B, Emblem A, Brekke OL, Karlsen BO. C-type lectin 10A [CD301] mRNA is upregulated in symptomatic acute intermittent porphyria and decreases after heme treatment. Mol Genet Metab. 2026 Sep 18;149(3):110263. doi: 10.1016/j.ymgme.2026.110263. Online ahead of print.
Related trials for Acute Porphyrias
RecruitingObservational study
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
Acute Hepatic Porphyria
Active, not recruitingObservational study
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
RecruitingObservational study
Identification of Acute Intermittent Porphyria Modifying Genes
Acute Intermittent Porphyria (AIP)
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
Active, not recruitingObservational study
Acute Porphyria Biomarkers for Disease Activity
Acute Intermittent Porphyria, Hereditary Coproporphyria
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.
Karl E Anderson, MD principal investigator
UT, Galveston
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Panhematin and Glucose in people with Acute Porphyrias, run by The University of Texas Medical Branch, Galveston 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 8 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 11 Apr 2025. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Difference in NRS Pain Score Between Baseline and 12 Hours (time frame: Baseline and 12 hours). 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 NCT02180412, 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 11 Apr 2025.