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Metabolic & Lysosomal

Acute Hepatic Porphyria (AHP) Clinical Trials and Treatments

Also called AHP, Acute Intermittent Porphyria, AIP, Hereditary Coproporphyria, HCP, Variegate Porphyria, ALAD Deficiency Porphyria, ALAD Porphyria, Acute Porphyria, Swedish Porphyria

Heme is the iron-containing part of hemoglobin and of many liver enzymes, and the liver builds it in 8 steps. In each acute hepatic porphyria one step runs slow: step 3 in AIP (the HMBS enzyme), step 6 in HCP (CPOX), step 7 in VP (PPOX) and step 2 in ADP (ALAD).

FDA-approved medicines

2

Givlaari, Panhematin

Active trials

4

4 recruiting now

Next FDA decision

None set

FDA calendar

Medicines are those whose current FDA label names Acute Hepatic Porphyria, checked October 14, 2026. Corticosteroids and immune globulins count once each. Download the data

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About Acute Hepatic Porphyria

Heme is the iron-containing part of hemoglobin and of many liver enzymes, and the liver builds it in 8 steps. In each acute hepatic porphyria one step runs slow: step 3 in AIP (the HMBS enzyme), step 6 in HCP (CPOX), step 7 in VP (PPOX) and step 2 in ADP (ALAD). Most of the time the liver copes. When something pushes it to make more heme, the first enzyme in the chain, ALAS1, speeds up and the early building blocks aminolevulinic acid (ALA) and porphobilinogen (PBG) pile up. Those 2 chemicals are toxic to nerves, and the result is a porphyria attack. Anything that revs up ALAS1 can be a trigger, which is why hundreds of common drugs, fasting, alcohol and the hormone swings of the menstrual cycle matter so much.

Diagnosis depends on a single test done at the right moment: PBG (and usually ALA) in a spot urine sample collected during symptoms and kept away from light. A clearly raised PBG confirms an acute porphyria; stool and blood porphyrin tests then tell AIP, HCP and VP apart, and gene testing identifies the family variant so relatives can be checked. Because attacks mimic appendicitis, kidney stones and psychiatric illness, many people are diagnosed years late or after an unsafe drug has made an attack worse.

An attack is treated in the hospital with pain and nausea control, IV fluids with glucose, correction of low sodium and removal of the trigger. Panhematin (hemin), the first drug approved under the 1983 Orphan Drug Act, is infused into a vein for 3 to 14 days and shuts ALAS1 down. Givlaari (givosiran), approved in 2019, is a monthly injection that silences the ALAS1 gene with small interfering RNA and cut attacks by 70% in its pivotal trial; it is for adults with recurrent attacks. Liver transplant corrects the disease and is reserved for the most severe cases. Everyone with AHP should check new medicines against the NAPOS drug safety database linked by the American Porphyria Foundation, carry an emergency letter, avoid fasting, and have regular checks of blood pressure and kidney function and, after age 50, liver imaging for hepatocellular carcinoma.

Common Symptoms of Acute Hepatic Porphyria

Recognizing the signs of Acute Hepatic Porphyria early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Severe, steady abdominal pain that builds over hours to days, often with pain in the back, chest, arms or legs
  • Nausea, vomiting, constipation and bloating, sometimes with trouble passing urine
  • Dark, red or brown urine, especially after it sits in the light
  • Fast heartbeat, high blood pressure and sweating during an attack
  • Anxiety, confusion, insomnia, hallucinations or other sudden changes in mood or thinking
  • Low blood sodium (hyponatremia), which can bring on seizures
  • Numbness, tingling, burning pain or weakness in the arms and legs that can progress to paralysis and, rarely, trouble breathing
  • Blistering, fragile skin on the backs of the hands and face after sun exposure (variegate porphyria and hereditary coproporphyria only)
  • Between attacks, some people have ongoing pain, fatigue, nausea or nerve symptoms
  • Over many years: high blood pressure, chronic kidney disease and a higher risk of liver cancer

Who Acute Hepatic Porphyria Affects

Attacks almost always begin after puberty, most often in the 20s and 30s, and women have them far more often than men; Orphanet puts women at 80% of symptomatic AIP cases, and many women notice attacks in the days before a period, when progesterone is high. Onset before puberty is extremely rare except in ALAD deficiency porphyria, which can start in childhood and has been reported only in males.

AIP, HCP and VP are autosomal dominant: 1 changed copy of the HMBS, CPOX or PPOX gene is enough to inherit the condition, and each child of a carrier has a 50% chance of inheriting it. Penetrance is very low, so most people who carry the variant never have an attack and may not know they are carriers. Symptoms usually need a trigger on top of the gene: certain prescription or recreational drugs, crash dieting or fasting, alcohol, infections, surgery, stress or hormonal changes. ALAD deficiency porphyria is autosomal recessive and needs a changed copy from each parent.

AIP occurs in every ethnic group but is most common in Sweden because of a founder variant, and NORD notes it may be reported less often in African American individuals. About 3% to 5% of people with symptomatic AIP, mostly women, go on to have recurrent attacks, defined as more than 4 a year (NORD; Elder et al., 2013). Expert groups advise testing all first-degree relatives of someone with a confirmed diagnosis so that carriers can learn which drugs to avoid before an attack ever happens.

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Find trials I might fit Answer a few questions and see which open Acute Hepatic Porphyria trials fit your age, location and treatment history.

FDA-Approved Treatments for Acute Hepatic Porphyria

There are currently 2 FDA-approved medications for Acute Hepatic Porphyria. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

givosiran
Alnylam Pharmaceuticals
Search
hemin for injection
Recordati Rare Diseases
Search

Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Help Paying for Acute Hepatic Porphyria Treatment

Charity funds and drugmaker programs for Acute Hepatic Porphyria, checked at the source.

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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Acute Hepatic Porphyria Resources

Reputable organizations and medical references for learning more about Acute Hepatic Porphyria, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Acute Hepatic Porphyria

Use this Acute Hepatic Porphyria clinical trial finder to see the 4 studies recruiting patients in the United States and worldwide, with eligibility criteria in plain English. Of these, 1 tests a treatment and 3 are observational studies or registries; each card says which. These studies play a critical role in advancing care for metabolic & lysosomal conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

New to trials? What Placebos and Randomization Actually Mean for You (In Plain English) · 4 min readWorried about cost? The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them) · 4 min read
TrialsSite mapPipeline timelineMedication checker

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

4 active trials worldwide
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RECRUITINGObservational study, no treatment givenRecently updatedNCT04883905

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

Sponsor: Alnylam Pharmaceuticals

This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and other approved AHP therapies.

Ages 12 Years+30 locations
Started Apr 2021Updated 1 week agoEst. Dec 2030 (~4y 2m)
RECRUITINGObservational study, no treatment givenRecently updatedNCT05502133

Identification of Acute Intermittent Porphyria Modifying Genes

Sponsor: Icahn School of Medicine at Mount Sinai

This study proposes to identify the predisposing/protective modifying genes that underlie the acute attacks in symptomatic patients with Acute Intermittent Porphyria (AIP), an autosomal dominant inborn error of heme biosynthesis.

Ages 12 Years+1 location
Started Sep 2022Updated 1 month agoEst. Jun 2029 (~2y 8m)
RECRUITINGObservational study, no treatment givenUpdated a few months agoNCT01561157

Longitudinal Study of the Porphyrias

Sponsor: The American Porphyrias Expert Collaborative

The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders.

Ages 1 Minute+16 locations
Started Nov 2010Updated 5 months agoEst. Jun 2030 (~3y 8m)
RECRUITINGTreatment trialNo updates in a whileNCT06273644

Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment

Intervention: Carbohydrates

Sponsor: Nordlandssykehuset HF

The main aim of this clinical trial is to learn about the effect of carbohydrate-rich diet as a treatment for AIP (acute intermittent porphyria).

Ages 18 Years+3 locations
Started Jan 2024Updated 6 months agoEst. Oct 2027 (~1 year)
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Active trial locations19 cities in the US
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Trial Pipeline

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Can I Join an Acute Hepatic Porphyria Study While Taking My Current Medications?

This medication checker does 2 jobs for people with acute hepatic porphyria (AHP) and their caregivers. It screens the active AHP studies and registries for eligibility conflicts with the medicines you take, and it reminds you of the bigger question in this disease: whether a medicine is safe for you at all, since hundreds of common drugs can trigger an attack.

AHP is unusual among rare diseases in that the medicine list matters more for safety than for trial eligibility. The studies open today are observational (Alnylam's ELEVATE registry, the Porphyrias Consortium's Longitudinal Study of the Porphyrias, Mount Sinai's AIP modifier gene study, a Norwegian diet study and a Texas biomarker study), and their published criteria ask about a confirmed diagnosis, age and whether you are in another drug trial, not about your prescriptions. The safety question is answered by the drug safety database maintained by NAPOS and linked by the American Porphyria Foundation and the United Porphyrias Association, which rates each drug by its potential to trigger an attack in AIP, VP, HCP and ADP. The checker below reads the published criteria of every active AHP study and flags the ones that mention your medicine; the cards explain what each class means for you.

Attack-Triggering Drugs
Barbiturates, sulfonamide antibiotics, rifampin, several anti-seizure drugs, progestins and estrogens, many others — the classes the American Porphyria Foundation names as the worst offenders, because they push the liver's ALAS1 enzyme harder and set off the ALA and PBG build-up behind an attack. The drug safety database rates each medicine by name; check it before any new prescription, anesthesia or over-the-counter product, and ask your porphyria specialist about anything rated unsafe, probably unsafe or not classified. No trial will enroll you while an attack is being triggered, and these drugs are the most common cause.
Preventive Treatment
Givlaari (givosiran) — the monthly siRNA injection approved for adults with recurrent attacks. Observational studies take people on or off Givlaari: ELEVATE enrolls anyone aged 12 and older with a physician-documented AHP diagnosis and excludes only people in an investigational drug trial, and the Longitudinal Study of the Porphyrias enrolls anyone with biochemical or genetic proof of porphyria. If a new interventional trial opens, expect it to ask how long you have been on Givlaari and your liver and kidney results.
Attack Treatment
Panhematin (hemin for injection), IV glucose — the hospital treatments for an attack in progress. Hemin use is recorded in every registry as a measure of disease activity, and the University of Texas Medical Branch biomarker study (NCT02935400) follows adults with a documented AIP, HCP or VP diagnosis around hemin treatment. Needing hemin never excludes you from the observational studies; it is part of what they measure.
Pain and Nausea Medicines
Opioids such as morphine, anti-nausea medicines, chlorpromazine — the medicines used during attacks and, for some people, between them. NORD notes attack pain usually needs opioids, and chlorpromazine labels still carry a decades-old indication for acute intermittent porphyria. These rarely affect study eligibility, but a registry will record them and a study measuring daily pain scores may ask for a stable dose. Confirm each one in the drug safety database; a few anti-nausea and sedative drugs are rated unsafe.
Hormonal Treatments
Birth control pills, progestin-only methods, hormone replacement, GnRH analogs (leuprolide) — progesterone is a well-known attack trigger and the Panhematin label tells patients to avoid estrogens such as oral contraceptives. Some women with attacks tied to their cycle are treated with a GnRH analog to stop ovulation under specialist care. Studies do not exclude people on these medicines, but you should tell any study team and your porphyria doctor about every hormonal product, including implants and IUDs.
Seizure, Mood and Infection Medicines
Anti-seizure drugs, antidepressants, antipsychotics, antibiotics and antifungals — the classes with the widest spread of ratings in the drug safety database: some anti-seizure drugs and antibiotics are among the most dangerous triggers while others in the same class are rated safe. Seizures during an attack are often caused by low sodium, which is treated with saline, so an attack is not by itself a reason to start a long-term anti-seizure drug. When the checker flags one of these at low confidence it usually means a study excludes an unrelated condition that could confuse its results, not the drug itself.
Check every new prescription before you fill it
The American Porphyria Foundation says prevention and avoidance of unsafe medications is essential to good management, and attacks set off by an unsafe drug given to someone whose diagnosis was not known are among the most dangerous. Search the NAPOS drug safety database (drugs-porphyria.org) by brand name, keep a wallet card or emergency letter that names your diagnosis and safe drugs, and tell every prescriber, dentist and anesthesiologist that you have an acute porphyria. Report any reaction to the APF at 866-APF-3635.
On Givlaari? The registries want you, and so does the safety record
Givlaari was approved in 2019 on a 6-month trial of 94 people, so long-term data on liver enzymes, kidney function, homocysteine and pancreatitis still come largely from registries and reports. ELEVATE (NCT04883905) follows people on and off Givlaari through 2030, and the Longitudinal Study of the Porphyrias collects the same information at Consortium centers. Enrolling adds nothing to your treatment and gives the next patient better answers.
Carriers who have never had an attack can join too
Most people with an HMBS, CPOX or PPOX variant never have an attack, and researchers want to know why. Mount Sinai's modifier gene study (NCT05502133) enrolls symptomatic people with AIP and their parents and relatives out to third-degree, and the Longitudinal Study of the Porphyrias makes provision for relatives with biochemical or genetic documentation but no symptoms. If you are a carrier, these are your studies, and the drug safety rules apply to you as well.
Don't see your medication listed?
The checker covers the classes that appear most often in AHP study criteria and on the drug safety lists: attack-triggering drugs, Givlaari, hemin, pain and nausea medicines, hormonal treatments and seizure, mood and infection medicines. If yours is missing, it has not come up in current AHP criteria, but look it up in the drug safety database anyway and tell the study team about everything you take, including herbal products and supplements.
How the medication conflict checker works: This free tool helps people with acute hepatic porphyria learn whether their current medications could affect clinical study eligibility. It scans the published eligibility criteria of every active AHP study and registry and flags the ones that may exclude your treatment. Matches are sorted by confidence: high confidence means the study names your exact drug, medium confidence means it names your drug class, and low confidence means it uses broad language that may or may not apply. It does not rate whether a drug is safe for you; that is what the NAPOS and APF drug safety database is for. Always confirm eligibility with the study team and drug safety with a porphyria specialist.

Across 1,853 open rare disease treatment trials, a third exclude people over a medication they commonly take. See which medications and diseases, in our September 2026 analysis.

Sources: Eligibility details above come from ClinicalTrials.gov records NCT04883905 (ELEVATE), NCT01561157 (Longitudinal Study of the Porphyrias), NCT05502133 (Mount Sinai modifier genes), NCT06273644 (carbohydrate-rich diet) and NCT02935400 (UTMB biomarkers), read October 18, 2026; drug safety statements come from the American Porphyria Foundation's AHP Drug Safety Database page and its AIP and HCP pages, the NAPOS database (drugs-porphyria.org), NORD's AIP report and the Panhematin prescribing information.

Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Acute Hepatic Porphyria patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Acute Hepatic Porphyria treatments, clinical trial participation, and day-to-day disease management.

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American Porphyria Foundation
The US foundation for all porphyrias since the 1980s: patient portal, Porphyria Experts list, diet and nutrition guidance, member stories, podcast and a support line at 866-APF-3635 (866-273-3635)
APF Drug Safety Database (NAPOS)
The expert-rated safe and unsafe drug list for AIP, VP, HCP and ADP, which links to the NAPOS database at drugs-porphyria.org; check every new prescription here and report bad reactions to the APF
APF Emergency Room Guidelines for Acute Porphyrias
What to hand an emergency team during an attack: testing, safe medicines, glucose and hemin
United Porphyrias Association
Patient organization in Bethesda, MD with a Porphyria Navigator, Find a Specialist tool, UPA One to One peer ambassadors and a drug safety page; 1-800-868-1292
UPA Connect UP peer groups
Small peer-led Zoom groups, including a weekly Acute Porphyrias Connect UP (Wednesdays 1:30 PM Eastern), a monthly evening group and a group for people who have had paralysis from AHP; sign up to be matched
Porphyria Together (UPA Facebook group)
The United Porphyrias Association's Facebook group for patients and families
American Porphyria Foundation on Facebook
The APF's page, with news, event announcements and member stories
American Porphyria Foundation on YouTube
Recorded patient meetings, expert talks and awareness videos
United Porphyrias Association on YouTube
Webinars with porphyria experts, including discussions of the 2026 acute porphyria guidelines
Porphyrias Consortium (American Porphyrias Expert Collaborative)
The NIH-founded network of 14 specialist centers that runs the Longitudinal Study of the Porphyrias and lists porphyria centers of expertise
Alnylam Assist for Givlaari
Alnylam's support program for people prescribed Givlaari: case managers, copay help for commercial insurance, foundation referrals and a Patient Assistance Program; 1-833-256-2748
Panhematin patient support (Recordati Rare Diseases)
RRD Patient Solutions case managers, copay and patient assistance programs, an AIP Attack Plan worksheet, a medical emergency letter and the Panhematin Locator; 1-866-209-7604
NORD Rare Disease Report
Reviewed plain-language medical overview of acute intermittent porphyria from the National Organization for Rare Disorders, written with porphyria specialists

Events

Peer meetings from the United Porphyrias Association: a weekly Zoom group for people with any acute hepatic porphyria, a monthly evening group, a monthly group for people who have had paralysis from an attack, and an in-person dinner in Denver on November 6, 2026. The American Porphyria Foundation's events feed listed nothing upcoming when this page was checked.

United Porphyrias Association
United Porphyrias AssociationFrom porphyria.org, checked October 18, 2026
Support Group

Acute Porphyrias Connect UP (weekly Zoom)

When
1:30 to 2:30 PM Eastern, October 28, 2026Weekly on Wednesdays
Where
Zoom
Cost
Free
For
People with AIP, HCP, VP or ADP and their caregivers

UPA's weekly peer group for people living with acute hepatic porphyria (AIP, HCP, VP and ADP), moderated by Mayra Martinez and Ginger Davis, who both live with acute intermittent porphyria. UPA describes it as a conversation among peers who bring lived experience, empathy and care.

Connect UP groups are small and peer-led. Fill in UPA's Connect UP sign-up form to be matched to the group; the Zoom link comes from UPA, not from a public page.

United Porphyrias Association
United Porphyrias AssociationFrom porphyria.org, checked October 18, 2026
Support Group

Acute Porphyrias Evening Connect UP (monthly Zoom)

When
6:00 to 7:00 PM Eastern, November 3, 2026Monthly (see the UPA calendar)
Where
Zoom
Cost
Free
For
People with AIP, HCP, VP or ADP and their caregivers

An evening or late-afternoon version of UPA's acute porphyria peer group, for people who cannot make the weekday afternoon meeting. UPA calls it a supportive and understanding space to connect, share and feel seen.

Sign up through UPA's Connect UP form to be matched; UPA sends the Zoom details.

United Porphyrias Association
United Porphyrias AssociationFrom porphyria.org, checked October 18, 2026
Support Group

Acute Porphyrias and Paralysis Connect UP (monthly Zoom)

When
1:30 to 2:30 PM Eastern, November 5, 2026Monthly (see the UPA calendar)
Where
Zoom
Cost
Free
For
People who have had paralysis from an acute porphyria attack

A special Connect UP for anyone who has experienced paralysis because of their acute hepatic porphyria, one of the most serious complications of a severe attack and one that can take months to recover from.

Sign up through UPA's Connect UP form to be matched; UPA sends the Zoom details.

Denver, CO (venue not yet posted)
United Porphyrias Association
United Porphyrias AssociationFrom porphyria.org, checked October 18, 2026
In-Person Meetup

Link UP in Denver, CO (UPA community dinner)

When
7:00 PM Mountain, November 6, 2026
Where
Denver, CO (venue not posted)
Cost
Not posted
For
People with any porphyria and their families in the Denver area

A dinner and patient meet-up for porphyria community members in the Denver region, for all types of porphyria (the event is tagged AIP, HCP, VP, EPP, XLP, PCT, CEP and HEP). UPA frames it as a chance to enjoy a meal and connect with others nearby who understand what you are going through.

UPA's page gives the date and a 7 PM start and says more details and registration are coming soon; the venue was not posted when this page was checked.

Details come from the United Porphyrias Association's own event pages (porphyria.org/connect-up and porphyria.org/events), checked October 18, 2026. Connect UP groups require signing up through UPA's form to be matched to a group; times are shown in Eastern time as UPA's calendar links give them. The Denver dinner page says registration details are coming, so confirm with UPA before traveling.

Related Conditions

Conditions that share genes, symptoms or treatments with Acute Hepatic Porphyria, followed by others in the metabolic & lysosomal category. Patients with Acute Hepatic Porphyria may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Acute Hepatic Porphyria Treatments

2 pharmaceutical companies have Acute Hepatic Porphyria in their rare disease portfolio

Frequently Asked Questions About Acute Hepatic Porphyria