AAV Gene Therapy Study for Subjects With PKU

  • STATUS
    Recruiting
  • End date
    Dec 5, 2026
  • participants needed
    100
  • sponsor
    BioMarin Pharmaceutical
Send
Updated on 19 February 2024
See if I qualify
deficiency
gene therapy

Summary

This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.

Details
Condition Phenylketonuria, Phenylketonuria
Age 15years - 100years
Treatment BMN 307
Clinical Study IdentifierNCT04480567
SponsorBioMarin Pharmaceutical
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
Ability and willingness to maintain dietary protein intake consistent with baseline intake
Willingness to abstain from hepatotoxic substances post-BMN 307 administration
Willingness and capable per investigator opinion to comply with study procedures and requirements
Willingness to use effective methods of contraception
Plasma Phe levels > 600 mol/L

Exclusion Criteria

Subjects with primary BH4 deficiency or other forms of BH4 metabolism deficiency
Clinically significant liver dysfunction or disease
Prior treatment with gene therapy
Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study
History of malignancy
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center

Select a site

Enter your location to find study centers near you

Entire World

study centers available

    loading...

Send a message

Enter your contact details to connect with study team

First name*
Last name*
Email*
Phone number*
  • United States+1
  • United Kingdom+44
  • Afghanistan (‫افغانستان‬‎)+93
  • Albania (Shqipëri)+355
  • Algeria (‫الجزائر‬‎)+213
  • American Samoa+1
  • Andorra+376
  • Angola+244
  • Anguilla+1
  • Antigua and Barbuda+1
  • Argentina+54
  • Armenia (Հայաստան)+374
  • Aruba+297
  • Ascension Island+247
  • Australia+61
  • Austria (Österreich)+43
  • Azerbaijan (Azərbaycan)+994
  • Bahamas+1
  • Bahrain (‫البحرين‬‎)+973
  • Bangladesh (বাংলাদেশ)+880
  • Barbados+1
  • Belarus (Беларусь)+375
  • Belgium (België)+32
  • Belize+501
  • Benin (Bénin)+229
  • Bermuda+1
  • Bhutan (འབྲུག)+975
  • Bolivia+591
  • Bosnia and Herzegovina (Босна и Херцеговина)+387
  • Botswana+267
  • Brazil (Brasil)+55
  • British Indian Ocean Territory+246
  • British Virgin Islands+1
  • Brunei+673
  • Bulgaria (България)+359
  • Burkina Faso+226
  • Burundi (Uburundi)+257
  • Cambodia (កម្ពុជា)+855
  • Cameroon (Cameroun)+237
  • Canada+1
  • Cape Verde (Kabu Verdi)+238
  • Caribbean Netherlands+599
  • Cayman Islands+1
  • Central African Republic (République centrafricaine)+236
  • Chad (Tchad)+235
  • Chile+56
  • China (中国)+86
  • Christmas Island+61
  • Cocos (Keeling) Islands+61
  • Colombia+57
  • Comoros (‫جزر القمر‬‎)+269
  • Congo (DRC) (Jamhuri ya Kidemokrasia ya Kongo)+243
  • Congo (Republic) (Congo-Brazzaville)+242
  • Cook Islands+682
  • Costa Rica+506
  • Côte d’Ivoire+225
  • Croatia (Hrvatska)+385
  • Cuba+53
  • Curaçao+599
  • Cyprus (Κύπρος)+357
  • Czech Republic (Česká republika)+420
  • Denmark (Danmark)+45
  • Djibouti+253
  • Dominica+1
  • Dominican Republic (República Dominicana)+1
  • Ecuador+593
  • Egypt (‫مصر‬‎)+20
  • El Salvador+503
  • Equatorial Guinea (Guinea Ecuatorial)+240
  • Eritrea+291
  • Estonia (Eesti)+372
  • Eswatini+268
  • Ethiopia+251
  • Falkland Islands (Islas Malvinas)+500
  • Faroe Islands (Føroyar)+298
  • Fiji+679
  • Finland (Suomi)+358
  • France+33
  • French Guiana (Guyane française)+594
  • French Polynesia (Polynésie française)+689
  • Gabon+241
  • Gambia+220
  • Georgia (საქართველო)+995
  • Germany (Deutschland)+49
  • Ghana (Gaana)+233
  • Gibraltar+350
  • Greece (Ελλάδα)+30
  • Greenland (Kalaallit Nunaat)+299
  • Grenada+1
  • Guadeloupe+590
  • Guam+1
  • Guatemala+502
  • Guernsey+44
  • Guinea (Guinée)+224
  • Guinea-Bissau (Guiné Bissau)+245
  • Guyana+592
  • Haiti+509
  • Honduras+504
  • Hong Kong (香港)+852
  • Hungary (Magyarország)+36
  • Iceland (Ísland)+354
  • India (भारत)+91
  • Indonesia+62
  • Iran (‫ایران‬‎)+98
  • Iraq (‫العراق‬‎)+964
  • Ireland+353
  • Isle of Man+44
  • Israel (‫ישראל‬‎)+972
  • Italy (Italia)+39
  • Jamaica+1
  • Japan (日本)+81
  • Jersey+44
  • Jordan (‫الأردن‬‎)+962
  • Kazakhstan (Казахстан)+7
  • Kenya+254
  • Kiribati+686
  • Kosovo+383
  • Kuwait (‫الكويت‬‎)+965
  • Kyrgyzstan (Кыргызстан)+996
  • Laos (ລາວ)+856
  • Latvia (Latvija)+371
  • Lebanon (‫لبنان‬‎)+961
  • Lesotho+266
  • Liberia+231
  • Libya (‫ليبيا‬‎)+218
  • Liechtenstein+423
  • Lithuania (Lietuva)+370
  • Luxembourg+352
  • Macau (澳門)+853
  • Madagascar (Madagasikara)+261
  • Malawi+265
  • Malaysia+60
  • Maldives+960
  • Mali+223
  • Malta+356
  • Marshall Islands+692
  • Martinique+596
  • Mauritania (‫موريتانيا‬‎)+222
  • Mauritius (Moris)+230
  • Mayotte+262
  • Mexico (México)+52
  • Micronesia+691
  • Moldova (Republica Moldova)+373
  • Monaco+377
  • Mongolia (Монгол)+976
  • Montenegro (Crna Gora)+382
  • Montserrat+1
  • Morocco (‫المغرب‬‎)+212
  • Mozambique (Moçambique)+258
  • Myanmar (Burma) (မြန်မာ)+95
  • Namibia (Namibië)+264
  • Nauru+674
  • Nepal (नेपाल)+977
  • Netherlands (Nederland)+31
  • New Caledonia (Nouvelle-Calédonie)+687
  • New Zealand+64
  • Nicaragua+505
  • Niger (Nijar)+227
  • Nigeria+234
  • Niue+683
  • Norfolk Island+672
  • North Korea (조선 민주주의 인민 공화국)+850
  • North Macedonia (Северна Македонија)+389
  • Northern Mariana Islands+1
  • Norway (Norge)+47
  • Oman (‫عُمان‬‎)+968
  • Pakistan (‫پاکستان‬‎)+92
  • Palau+680
  • Palestine (‫فلسطين‬‎)+970
  • Panama (Panamá)+507
  • Papua New Guinea+675
  • Paraguay+595
  • Peru (Perú)+51
  • Philippines+63
  • Poland (Polska)+48
  • Portugal+351
  • Puerto Rico+1
  • Qatar (‫قطر‬‎)+974
  • Réunion (La Réunion)+262
  • Romania (România)+40
  • Russia (Россия)+7
  • Rwanda+250
  • Saint Barthélemy+590
  • Saint Helena+290
  • Saint Kitts and Nevis+1
  • Saint Lucia+1
  • Saint Martin (Saint-Martin (partie française))+590
  • Saint Pierre and Miquelon (Saint-Pierre-et-Miquelon)+508
  • Saint Vincent and the Grenadines+1
  • Samoa+685
  • San Marino+378
  • São Tomé and Príncipe (São Tomé e Príncipe)+239
  • Saudi Arabia (‫المملكة العربية السعودية‬‎)+966
  • Senegal (Sénégal)+221
  • Serbia (Србија)+381
  • Seychelles+248
  • Sierra Leone+232
  • Singapore+65
  • Sint Maarten+1
  • Slovakia (Slovensko)+421
  • Slovenia (Slovenija)+386
  • Solomon Islands+677
  • Somalia (Soomaaliya)+252
  • South Africa+27
  • South Korea (대한민국)+82
  • South Sudan (‫جنوب السودان‬‎)+211
  • Spain (España)+34
  • Sri Lanka (ශ්‍රී ලංකාව)+94
  • Sudan (‫السودان‬‎)+249
  • Suriname+597
  • Svalbard and Jan Mayen+47
  • Sweden (Sverige)+46
  • Switzerland (Schweiz)+41
  • Syria (‫سوريا‬‎)+963
  • Taiwan (台灣)+886
  • Tajikistan+992
  • Tanzania+255
  • Thailand (ไทย)+66
  • Timor-Leste+670
  • Togo+228
  • Tokelau+690
  • Tonga+676
  • Trinidad and Tobago+1
  • Tunisia (‫تونس‬‎)+216
  • Turkey (Türkiye)+90
  • Turkmenistan+993
  • Turks and Caicos Islands+1
  • Tuvalu+688
  • U.S. Virgin Islands+1
  • Uganda+256
  • Ukraine (Україна)+380
  • United Arab Emirates (‫الإمارات العربية المتحدة‬‎)+971
  • United Kingdom+44
  • United States+1
  • Uruguay+598
  • Uzbekistan (Oʻzbekiston)+998
  • Vanuatu+678
  • Vatican City (Città del Vaticano)+39
  • Venezuela+58
  • Vietnam (Việt Nam)+84
  • Wallis and Futuna (Wallis-et-Futuna)+681
  • Western Sahara (‫الصحراء الغربية‬‎)+212
  • Yemen (‫اليمن‬‎)+967
  • Zambia+260
  • Zimbabwe+263
  • Åland Islands+358

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Browse trials for

deficiency
gene therapy

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.