Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria
Information source: The University of Texas Medical Branch, Galveston
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Acute Porphyrias
Intervention: Panhematin (Biological); Placebo (Other)
Phase: Phase 2
Status: Recruiting
Sponsored by: The University of Texas Medical Branch, Galveston Official(s) and/or principal investigator(s): Karl E Anderson, MD, Principal Investigator, Affiliation: UT, Galveston
Overall contact: Pamela J Kiani, Phone: 409-772-1976, Email: pjkiani@utmb.edu
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).
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)
Clinical Details
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
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Pain
Secondary outcome: Biochemical effects of Panhematin
Eligibility
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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
Locations and Contacts
Pamela J Kiani, Phone: 409-772-1976, Email: pjkiani@utmb.edu
University of Texas Medical Branch, Galveston, Texas 77555, United States; Recruiting Karl E Anderson, MD, Phone: 409-772-4661, Email: kanderso@utmb.edu Csilla Hallberg, MD, Phone: 409-772-4661, Email: challberg@utmb.edu Karl E Anderson, MD, Principal Investigator
Additional Information
Starting date: April 2014
Last updated: January 15, 2015
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