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PhenylketonuriaPhase 2SynPheny-1NCT04534842

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

A Phase 2 study testing SYNB1618 and SYNB1934 in people with Phenylketonuria, run by Synlogic at 11 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This Phase 2 study in patients with phenylketonuria (PKU) will be an open-label, dual-arm study of either a SYNB1618 or SYNB1934 dose-ramp regimen. All evaluations and assessments throughout this study may be conducted either at the clinical site or by a home healthcare professional at an alternative location (e.g.… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Phenylketonuria
What is being tested?
SYNB1618 and SYNB1934
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
11 sitesUnited States
Study dates
25 Aug 2020to 7 Oct 2022
Phase
Phase 2
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥ 18 years.
  • Able and willing to voluntarily complete the informed consent process.
  • Diagnosis of classic PKU based on medical history as assessed by the investigator (e.g., Phe concentration of >1200 µmol/L at any time, low dietary Phe tolerance, or genetic diagnosis).
  • Blood Phe ≥ 600 µmol/L at Screening.
  • Stable diet including stable medical formula regimen (if used) for at least 1 month prior to Screening.
  • Available for and agree to all study procedures, including urine and blood collection, adherence to diet control, follow-up visits, and IMP ingestion compliance.
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  • Male patients who are sexually abstinent or surgically sterilized (vasectomy), or those who are sexually active with a female partner(s) and agree to use an effective method of contraception (such as condom with spermicide) combined with an acceptable method of contraception for their non-pregnant female partner(s) (as defined in Inclusion Criterion #8 below) after informed consent, throughout the study, and for a minimum of 3 months after the last dose of IMP, and who do not intend to donate sperm in the period from Screening until 3 months following administration of the IMP.
  • Female patients who meet one of the following criteria:
  • Women of childbearing potential (WOCBP) must have a negative serum pregnancy test (human chorionic gonadotropin) at Screening and a negative urine pregnancy test at Baseline prior to the start of IMP and must agree to use acceptable method(s) of contraception, combined with an acceptable method of contraception for their male partner(s) (as defined in Inclusion Criterion #7 above) after informed consent, throughout the study, and for a minimum of 3 months after the last dose of IMP. Acceptable methods of contraception include hormonal contraception, hormonal or non-hormonal intrauterine device, bilateral tubal occlusion, complete abstinence, vasectomized partner with documented azoospermia 3 months after procedure, diaphragm with spermicide, cervical cap with spermicide, vaginal sponge with spermicide, or male or female condom with or without spermicide.
  • Premenopausal woman with one of the following:
  • Documented hysterectomy, ii. Documented bilateral salpingectomy, iii. Documented bilateral oophorectomy, iv. Documented tubal ligation/occlusion, v. Sexual abstinence is preferred or usual lifestyle of the patient c. Postmenopausal women (12 months or more amenorrhea verified by follicle stimulating hormone [FSH] assessment and over 45 years of age, in the absence of other biological or physiological causes).
  • Screening laboratory evaluations (e.g., chemistry panel, complete blood count [CBC] with differential, urinalysis, creatinine clearance, CRP) within normal limits or judged to be not clinically significant by the investigator.

Who cannot join

  • Currently taking Palynziq® (pegvaliase-pqpz) (within 1 month of Screening).
  • Inflammatory or irritable bowel disorder of any grade.
  • History of or current immunodeficiency disorder.
  • Intolerance of or allergic reaction to E. coli Nissle or any of the ingredients in SYNB1618 formulation.
  • Any condition (e.g., celiac disease, gastrectomy, bypass surgery, ileostomy) or receiving prescription medication or an over-the-counter product that may possibly affect absorption of medications or nutrients.
  • Currently taking or plans to take any type of systemic (e.g., oral or intravenous) antibiotic within 28 days prior to the first dose of IMP through final safety assessment, including planned surgery, hospitalizations, dental procedures, or interventional studies that are expected to require antibiotics. Exception: topical antibiotics are allowed.
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  • Within the 3 months prior to anticipated first dose, major surgery (an operation upon an organ within the cranium, chest, abdomen, or pelvic cavity) or inpatient hospital stay.
  • Dependence on alcohol or drugs of abuse.
  • Administration or ingestion of an investigational drug within the 30 days or 5 half-lives before Screening, whichever is longer, or cell/gene therapy prior to the Screening visit, or current enrollment in an investigational drug study.
  • Acute or chronic medical, surgical, psychiatric, or social condition or laboratory abnormality that may increase patient risk associated with study participation, compromise adherence to study procedures and requirements, or may confound interpretation of study safety or PD results and, in the judgment of the investigator, would make the patient inappropriate for enrollment.

Trial locations and contacts

11 sites in 1 country.

11 sites in 1 country

United States11 sites

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Excel Medical Clinical Trials, LLC

    Boca Raton, Florida, 33434

  • University of Florida College of Medicine

    Gainesville, Florida, 32608

  • University of Texas Health Science Center at Houston

    Houston, Texas, 77030

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15224

  • Oregon Health and Science University

    Portland, Oregon, 97201

  • PRA Health Sciences

    Salt Lake City, Utah, 84124

  • University Hospital Adult Services

    Salt Lake City, Utah, 84132

  • Washington University in St Louis

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: An Open-label Study of the Efficacy and Safety of SYNB1618 and SYNB1934 in Patients With Phenylketonuria (SynPheny-1)

This Phase 2 study in patients with phenylketonuria (PKU) will be an open-label, dual-arm study of either a SYNB1618 or SYNB1934 dose-ramp regimen. All evaluations and assessments throughout this study may be conducted either at the clinical site or by a home healthcare professional at an alternative location (e.g., patient's home, hotel).

Read the detailed description

This is an open-label, dual-arm Phase 2 study; all participating PKU patients will receive active study drug, SYNB1618 or SYNB1934. This study is evaluating a dose-ramp regimen consisting of 4 dose levels of SYNB1618 or SYNB1934 over 15 days of treatment.

This study has been designed with the flexibility of being able to be conducted either at the clinical site or by a home healthcare professional at an alternative location (e.g., patient's home, hotel).

Patients will be screened for eligibility and complete a customized diet run-in period prior to starting the 15-day dose ramp ('treatment period'). During the treatment period patients will be required to adhere to the same customized diet menus as in the diet run-in period.

The efficacy of SYNB1618 SYNB1934 will be assessed in this study by measuring the reduction of the area under the curve (AUC) for plasma D5-phenylalanine (D5-Phe) as well as the reduction of plasma Phe levels.

Safety will be monitored by documentation of adverse events (AEs), clinical laboratory measurements, vital signs, and physical examinations.

Conditions
Phenylketonuria
Being tested
SYNB1618, SYNB1934
Medical subject headings
Phenylketonurias

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SYNB1618Drug

    15-day dose-ramp regimen (4 dose levels) of orally administered SYNB1618

  • SYNB1934Drug

    15-day dose-ramp regimen (4 dose levels) of orally administered SYNB1934

Study groups in NCT04534842
Study groupRoleWhat they receive
SYNB1618ExperimentalDose ramp of SYNB1618
Receives: Drug: SYNB1618
SYNB1934ExperimentalDose ramp of SYNB1934
Receives: Drug: SYNB1934

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Changes from baseline in labeled Phe (D5-Phe) in plasma

    The primary efficacy outcome will be assessed by measuring change from baseline in plasma D5-Phe AUC over 24 hours after D5-Phe administration

    Measured: Day 14

Secondary outcomes (2)

  1. Changes from baseline in fasting levels of plasma Phe

    The secondary efficacy outcome will be assessed by measuring fasting levels of plasma Phe as compared to baseline

    Measured: Day 14

  2. Incidence of Treatment-Emergent Adverse Events

    Will be measured by assessing nature and frequency of AEs

    Measured: Day 43

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Sep 2020
  2. Study start25 Aug 2020
  3. Primary completion7 Oct 2022
  4. Study completion7 Oct 2022

Resources

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04534842
Sponsor's protocol ID
SYNB1618-CP-003
Acronym
SynPheny-1
Status last verified
Oct 2022
First submitted
24 Aug 2020
Last updated
17 Oct 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Vockley J, Sondheimer N, Puurunen M, Diaz GA, Ginevic I, Grange DK, Harding C, Northrup H, Phillips JA 3rd, Searle S, Thomas JA, Zori R, Denney WS, Ernst SL, Humphreys K, McWhorter N, Kurtz C, Brennan AM. Efficacy and safety of a synthetic biotic for treatment of phenylketonuria: a phase 2 clinical trial. Nat Metab. 2023 Oct;5(10):1685-1690. doi: 10.1038/s42255-023-00897-6. Epub 2023 Sep 28.

    PubMed 37770764

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing SYNB1618 and SYNB1934 in people with Phenylketonuria, run by Synlogic at 11 sites 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 12 inclusion and 10 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 17 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites 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. 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?

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?

Changes from baseline in labeled Phe (D5-Phe) in plasma (time frame: Day 14). 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 NCT04534842, 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 17 Oct 2022; AzTrials last refreshed its copy on 8 Nov 2026.

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