BridgeMD
New treatment option for Phenylketonurias
Official title AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
ClinicalTrials.gov ID: NCT06332807
What this study is testing
What is NGGT002?
NGGT002 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for phenylketonurias.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 55
You may be able to join if
- Is willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related...
- Male and female people with diagnosis of classic PKU, a condition characterized by severe PAH deficiency with confirmed PAH mutations predicted with...
- Adults aged 18-55 at the time of informed consent
- people intolerant or unresponsive to available medical therapies, such as Kuvan, Playnzip, etc.
- people who have been on medications, such as Kuvan, Palynziq, etc but have come off for medical reasons or the patient's decision at least 28 days...
You likely can't join if
- people with PKU that is not due to PAH mutation
- Presence of anti-AAV8 neutralizing antibodies
- Prior to dosing, people exceed the limit of any of the following liver function and hematology tests in two consecutive blood laboratory tests:
- Alanine aminotransferase (ALT) \>1.5×ULN and/or aspartate aminotransferase (AST) \>1.5×ULN
- Alkaline phosphatase (ALP) \>1.5×ULN
- Total bilirubin (TBil) \>1.5×ULN, direct bilirubin \>1.5×ULN
See the full eligibility criteria
- Is willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related procedures; a legally authorized representative may provide written...
- Male and female people with diagnosis of classic PKU, a condition characterized by severe PAH deficiency with confirmed PAH mutations predicted with no residual enzyme activity. A list of PAH mutations for classic PKU...
- Adults aged 18-55 at the time of informed consent
- people intolerant or unresponsive to available medical therapies, such as Kuvan, Playnzip, etc.
- people who have been on medications, such as Kuvan, Palynziq, etc but have come off for medical reasons or the patient's decision at least 28 days prior to signing the consent form (people who have good disease control...
- At least 1 documented measurements of Phe ≥ 600 μmol/L while on usual diet in the preceding 6 months.
- people are willing to record their diet and follow the instruction of dietitians during the trial.
- Willingness and capable per Investigator opinion to comply with study procedures and requirements.
- Women of child bearing potential must be confirmed as negative non pregnant people by blood pregnancy test from day -28 to day 0. people must agree to use a highly effective form of contraception from the time of...
- documented vasectomy or permanent sterilization
- condom
- combined (estrogen and progestogen-containing) hormonal contraception (oral, intravaginal or transdermal)
- progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable)
- intrauterine device
- intrauterine hormone-releasing system
- sexual abstinence is acceptable only as true abstinence and when in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, hypothermal, post-ovulation) is not...
- people with PKU that is not due to PAH mutation
- Presence of anti-AAV8 neutralizing antibodies
- Prior to dosing, people exceed the limit of any of the following liver function and hematology tests in two consecutive blood laboratory tests:
- Alanine aminotransferase (ALT) \>1.5×ULN and/or aspartate aminotransferase (AST) \>1.5×ULN
- Alkaline phosphatase (ALP) \>1.5×ULN
- Total bilirubin (TBil) \>1.5×ULN, direct bilirubin \>1.5×ULN
- International normalized ratio (INR) \> 1.5
- Blood creatinine (Scr) \>1.5×ULN
- Hematology values outside of the normal range (Hemoglobin \<110 g/L (male), \<100 g/L (female), white blood cell \<3.0×10\^9/L, neutrophil \<1.5×10\^9/L, platelet \<100×10\^9/L)
- Hemoglobin A1c \>6% or fasting glucose \>6.1 mmol/L
- At the time of screening, abnormal vital signs (i.e. Temperature\ 37.4°C; Blood pressure\ 130/80 mmHg; heart rate \ 100/min; respiratory rate \ 18/min; oxygen saturation\<95%), physical examination, laboratory tests, or...
- Contraindications to corticosteroid use or possible deterioration of corticosteroid use assessed and determined by the Investigator.
- Active infection with hepatitis A virus (HAV ribonucleic acid [RNA] positive), active or occult hepatitis B virus infection (positive HBV-DNA or anti-HBc positive with negative hBsAg, HBV surface antigen), active...
- people with history of liver disease such as clinically significant steatosis, fibrosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, biliary disease within 6 months of informed consent; except for Gilbert's...
- All types of past and current malignancy
- Imaging (liver ultrasound) proved the existence of Liver fibrosis, liver cirrhosis and other serious liver diseases
- Severe diseases in the cardiovascular, respiratory, digestive tract, endocrine, kidney, blood, nervous, mental and other systems before screening.
- History of allergy to Albumin (Human)
- The people who have Substance Use Disorder (for example alcohol, heroin, amphetamine, etc)
- The people who have received any gene therapy in the past, regardless of when it was administered.
- The people who have received any investigational treatment and took drugs within 3 months before screening (or 5 half-lives, if longer)
- people with elevated circulating serum alpha-fetoprotein (AFP)
- Other conditions that the Investigators deemed inappropriate for enrollment, such as PKU severe comorbidities and conditions (i.e. renal insufficiency or kidney failure, osteoporosis, anemia, acid reflux or...
- people who are presently on available medications for the treatment of PKU, such as Kuvan, Palynziq, etc.
- people who weight over 120 Kg
- people who consume too much natural protein (\>2 g/Kg body weight/day) in their daily diet
- Breastfeeding people will not be included in the study
The study team makes the final eligibility decision.
Where it's taking place
- Orange, California, United States
- Minneapolis, Minnesota, United States
- Morristown, New Jersey, United States
- Pittsburgh, Pennsylvania, United States
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 55 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Orange, California, United States; Minneapolis, Minnesota, United States; Morristown, New Jersey, United States; Pittsburgh, Pennsylvania, United States; Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.